Inhibidor de MEK mirdametinib (PD-0325901) en pacientes con neurofibromatosis plexiforme asociada a neurofibromatosis tipo 1 (ReNeu)
Un ensayo de fase 2b del inhibidor de MEK 1/2 (MEKi) PD-0325901 en pacientes adultos y pediátricos con neurofibromatosis tipo 1 (NF1) asociada a neurofibromas plexiformes inoperables (PN) que están causando una morbilidad significativa
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
Descripción general del estudio
Estado
Intervención / Tratamiento
Descripción detallada
Neurofibromas are non-malignant peripheral nerve sheath tumors, which are classified as plexiform neurofibromas (PNs) if they extend longitudinally along a nerve and involve multiple fascicles. PNs are a major cause of morbidity and disfigurement in individuals with NF1, and as the tumor growth progresses, can cause a multitude of clinical problems including pain and impaired physical function. PNs have the potential to undergo malignant transformation to Malignant Peripheral Nerve Sheet Tumors (MPNST).
Mirdametinib (PD-0325901) is an orally delivered, highly selective small-molecule inhibitor of the dual specificity kinases, MEK1 and MEK2 (MAPK/ERK Kinase) which prevents the phosphorylation and subsequent activation of mitogen-activated protein kinase (MAPK).
Previous studies of mirdametinib (PD-0325901) demonstrated PN shrinkage and sustained inhibition of pERK. Reduced tumor volume indicated that cell proliferation or cell death may be altered in PNs with administration of mirdametinib (PD-0325901).
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 2
Acceso ampliado
Contactos y Ubicaciones
Ubicaciones de estudio
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Alabama
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Birmingham, Alabama, Estados Unidos, 35233
- University of Alabama at Birmingham (UAB) - Children's of Alabama
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Arizona
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Phoenix, Arizona, Estados Unidos, 85054
- Mayo Clinic Arizona
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Arkansas
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Little Rock, Arkansas, Estados Unidos, 72202
- Arkansas Children's Hospital - University of Arkansas
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California
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Los Angeles, California, Estados Unidos, 90027
- Children's Hospital Los Angeles
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Los Angeles, California, Estados Unidos, 90095
- University of California, Los Angeles (UCLA)
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Orange, California, Estados Unidos, 92868
- Children's Hospital of Orange County
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Orange, California, Estados Unidos, 92868
- University of California - Irvine Health
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Palo Alto, California, Estados Unidos, 94304
- Lucile Packard Children's Hospital Stanford University
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Sacramento, California, Estados Unidos, 95817
- University of California - Davis
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Colorado
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Aurora, Colorado, Estados Unidos, 80045
- Children's Hospital Colorado
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Delaware
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Wilmington, Delaware, Estados Unidos, 19803
- Nemours A. I. duPont Hospital for Children
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District of Columbia
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Washington D.C., District of Columbia, Estados Unidos, 20010
- Children's National Hospital
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Florida
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Gainesville, Florida, Estados Unidos, 32610
- University of Florida (UF) - Gainesville
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Miami, Florida, Estados Unidos, 33155
- Nicklaus Children's Hospital
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Orlando, Florida, Estados Unidos, 32804
- Florida Hospital Cancer Institute
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Orlando, Florida, Estados Unidos, 32806
- Arnold Palmer Hospital for Children
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St. Petersburg, Florida, Estados Unidos, 33701
- Johns Hopkins All Children's Hospital
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Tampa, Florida, Estados Unidos, 33612
- Moffitt
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Illinois
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Chicago, Illinois, Estados Unidos, 60612
- University of Illinois Hospital
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Indiana
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Indianapolis, Indiana, Estados Unidos, 46202
- Indiana University
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Iowa
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Iowa City, Iowa, Estados Unidos, 52242
- University of Iowa Health Care
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109
- University of Michigan Comprehensive Cancer Center
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Detroit, Michigan, Estados Unidos, 48202
- Henry Ford Hospital
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Minnesota
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Minneapolis, Minnesota, Estados Unidos, 55455
- University of Minnesota Masonic Cancer Center
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Rochester, Minnesota, Estados Unidos, 55905
- Mayo Clinic
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Missouri
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St Louis, Missouri, Estados Unidos, 63110
- Washington University
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New Jersey
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Paterson, New Jersey, Estados Unidos, 07503
- Saint Joseph's Regional Medical Center
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New York
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Albany, New York, Estados Unidos, 12208
- Albany Medical Center
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The Bronx, New York, Estados Unidos, 10467
- Children's Hospital at Montefiore
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North Carolina
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Chapel Hill, North Carolina, Estados Unidos, 27599
- University of North Carolina (UNC) Hospitals
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Durham, North Carolina, Estados Unidos, 27710
- Duke University Medical Center
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45229
- UC Health
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Columbus, Ohio, Estados Unidos, 43205
- Nationwide Children's Hospital
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Oklahoma
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Oklahoma City, Oklahoma, Estados Unidos, 73104
- University of Oklahoma - Health Sciences Center
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Oregon
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Portland, Oregon, Estados Unidos, 97239
- Oregon Health and Science University
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Pennsylvania
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Pittsburgh, Pennsylvania, Estados Unidos, 15219-2739
- UPMC Children's Hospital of Pittsburgh
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Tennessee
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Nashville, Tennessee, Estados Unidos, 37232
- Vanderbilt Children's Hospital
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Texas
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Dallas, Texas, Estados Unidos, 75244
- University of Texas Southwestern
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Fort Worth, Texas, Estados Unidos, 76104
- Cook Children's Medical Center
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Houston, Texas, Estados Unidos, 77030
- MD Anderson Cancer Center
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Utah
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Salt Lake City, Utah, Estados Unidos, 84108
- The University of Utah Health Sciences Center
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Virginia
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Charlottesville, Virginia, Estados Unidos, 22903
- University of Virginia - Emily Couric Cancer Center
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Norfolk, Virginia, Estados Unidos, 23510
- Children's Hospital of The King's Daughters
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Key Inclusion Criteria:
- Participant has documented NF1 mutation or a diagnosis of neurofibromatosis type 1 (NF1) using National Institute of Health (NIH) Consensus Conference criteria inclusive of the presence of a plexiform neurofibroma (PN).
- Participant has a PN that is causing significant morbidity.
- Participant has a PN that cannot be completely surgically removed.
- Participant has a target tumor that is amenable to volumetric MRI analysis.
- Participant is willing to undergo a tumor biopsy pre and post treatment if ≥ 18 years of age.
- Participant has adequate organ and bone marrow function.
Key Exclusion Criteria:
- Participant has abnormal liver function or history of liver disease.
- Participant has lymphoma, leukemia or any malignancy within the past 5 years (except for resected basal/squamous skin carcinomas without metastases within 3 years).
- Participant has breast cancer within 10 years.
- Participant has active optic glioma or other low-grade glioma requiring treatment.
- Participant has abnormal QT interval corrected or other heart disease within 6 months.
- Participant has a history of retinal pathology, risk factors for retinal vein occlusion or has a history of glaucoma.
- Participant has known malabsorption syndrome or gastrointestinal conditions that would impair absorption of mirdametinib (PD-0325901).
- Participant has received NF1 PN-targeted therapy within 45 days.
- Participant previously received or is currently receiving therapy with mirdametinib (PD-0325901) or any other MEK1/2 inhibitor.
- Participant has received radiation therapy within 6 months or has received radiation to the orbit at any time.
- Participant is unable to undergo or tolerate MRI.
- Participant has active bacterial, fungal or viral infection.
- Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: Mirdametinib (PD-0325901)
Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m^2 (maximum dose of 4 mg) by mouth twice daily |
Mirdametinib (PD-0325901) capsule or dispersible tablet
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Confirmed Objective Response Rate at the End of the Treatment Phase.
Periodo de tiempo: Up to 24 months
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Response will be determined by a blinded centralized review of volumetric MRI. The confirmed objective response rate (complete or partial response) by the end of Treatment Phase (i.e., Cycle 24) is defined as the proportion of participants who have a confirmed ≥ 20% reduction in target tumor volume as compared to baseline as assessed by a BICR, and the response needs to be confirmed by BICR in a consecutive tumor assessment within 2 - 6 months. Partial response is defined as a ≥ 20% reduction in target tumor volume from baseline. Complete response is defined as the complete resolution of the target tumor. |
Up to 24 months
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Percentage of Patients With Treatment-Emergent Adverse Events.
Periodo de tiempo: All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months.
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All adverse events were coded using MedDRA Version 24.0. Adverse events will be assessed according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. |
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months.
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Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response.
Periodo de tiempo: Starting on the onset of confirmed objective response in the Treatment Phase and afterwards on the 15th day of every 4 cycles (each cycle is 28 days) until disease progression or death, whichever comes first, assessed up to approximately 3 years
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Duration of response is defined as the time in months between the first instance of response that is subsequently confirmed, until the date of radiographic disease progression or death, whichever occurs first. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine duration of response. Participants without radiographic disease progression or death while on study will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. |
Starting on the onset of confirmed objective response in the Treatment Phase and afterwards on the 15th day of every 4 cycles (each cycle is 28 days) until disease progression or death, whichever comes first, assessed up to approximately 3 years
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Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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The PedsQL consists of a 23-item core measure of global QOL that can be completed in approximately 5 minutes. There is a total score and four subscales: physical functioning, emotional functioning, social functioning and school/work functioning. Participants ≥ 5 years of age complete an age-appropriate self-report; and parents/guardians of children ages 2-17 complete a parent proxy report of the age-specific QOL. The recall period is 7 days. PedsQL items are answered on a Likert scale with responses ranging from 0 to 4 (where 0 means it is never a problem and 4 means it is almost always a problem). These items are then reverse scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. On this scale, higher scores indicate better outcomes. Overall scale scores are calculated as the mean of the scores for the questions in said scale (or of all questions for the total score). The change from baseline is modelled using a mixed-model for repeated measures. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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The NRS-11 is a self-reported 11-point numerical scale that assesses pain severity. Participants ≥ 8 years of age are asked to select a number from 0 (no pain) to 10 (worst pain you can imagine) that best describes their worst pain. The recall period is 24 hours. The change from baseline is modelled using a mixed-model for repeated measures. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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The PII assesses relevant aspects of one's life, including pain interference with activities, spending time with family/friends, mood, sleep and attention. Participants ≥ 6 years of age complete a self-report, and parents/guardians of children aged 6-17 complete a parent proxy report. The recall period is 24 hours. The PII consists of 6 questions, each asking the responder to select one number from 0 to 6 that best describes how their/their proxy's pain has impacted various items over the past 24 hours with 0 representing "Not at all" and 6 representing "Completely". The mean of the completed items is taken as the PII score for a single assessment. The PII score presented each visit will be taken as the average of the PII scores over the 7 consecutive days up to and including visit day, with no transformation applied. The change from baseline is modelled using a mixed-model for repeated measures. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Otras medidas de resultado
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Change From Baseline in Localized Strength (Dynamometer) at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Assessment of strength will be conducted only in the area affected by the target tumor. Measurements will be obtained using Medical Research Council (MRC) grading followed by quantitative assessments using the sponsor provided MicroFET2 dynamometer. Dynamometer assessment (in lbs) must be conducted in accordance with the study reference manual. For each participant, the sum of the dynamometer scores for the muscle groups tested will be taken as an overall score for each visit with higher scores indicating better localized strength. To assess the change from baseline at a visit, a participant will need assessments in which all the following categories, as assessed at baseline, match at that visit: side affected, position during assessment (sitting/supine/lateral decubitus), muscle group assessed. The change from baseline in localized strength measured by the sum of the dynamometer scores for the muscle groups tested will be assessed at Cycle 13. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Range of Motion of PN-Associated Functional Impairment at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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If motor dysfunction or weakness is evident, range of motion of the affected areas and/or joints will be measured by a goniometer. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Endurance at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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If airway or lower extremity motor dysfunction is evident, endurance will be measured by completion of a 6-minute walking test. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Time to Response Defined as the Time Between First Dose and the First Date of Objective Response That is Subsequently Confirmed.
Periodo de tiempo: Up to 24 months
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Response will be determined by a blinded centralized review of volumetric MRI. Time to response is defined as the time in months from the start of treatment to the date of first response that was subsequently confirmed. |
Up to 24 months
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Time to Progression, From the First Dose to the First Date of a ≥ 20% Increase in Tumor Volume From Baseline.
Periodo de tiempo: On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years
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Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Time to progression is defined as the time in months between the first treatment date until the date of radiographic disease progression. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine time to progression. Participants without radiographic disease progression will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. |
On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years
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Progression Free Survival, Defined as the Time in Months From the First Dose to the Date of the First ≥ 20% Increase in Tumor Volume From Baseline or Death.
Periodo de tiempo: On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 3.5 years
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Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Progression free survival is defined as the time in months between the first treatment date, until the date of radiographic disease progression or death, whichever occurs first. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine progression free survival. Participants without radiographic disease progression or death while on study will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. |
On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 3.5 years
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Change From Baseline in PN-Associated Disfigurement Using Standardized Photography, Centrally Reviewed.
Periodo de tiempo: Up to 24 months
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For participants with a PN that is visible and amenable to photography, changes in visible tumor aspects will be evaluated by a centralized reviewer. |
Up to 24 months
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Comparison of Tumor Response to Levels of pERK and Biomarkers Indicative of Inhibition of Downstream Targets of MEK (eg, ERK Phosphorylation).
Periodo de tiempo: Up to 24 months
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Measured in tumor biopsies in participants ≥ 18 years of age. |
Up to 24 months
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Acceptability of the Dispersible Tablet Formulation as Measured by the Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ)
Periodo de tiempo: Completed only once through study completion
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The Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ) uses a 5-point numerical rate scale to measure acceptability of use of the tablet formulation with questions related to taste, smell and administration of study medication. Participants ≥ 8 years of age complete a 12-item self-report; adult parents/caregivers responsible for oversight of study drug administration for participants ages 6 months to 17 years complete a 19-item caregiver report. Each questionnaire is provided with a "past 7 days" recall with all items using a 5-point Numerical Rate Scale (1 to 5) with higher item-scores reflecting greater oral treatment acceptability. The overall P-OMAQ-P (pediatric self-report) and P-OMAQ-C (adult caregiver) scores for a participant in the study will be taken as the mean of all answered questions on the respective questionnaire. |
Completed only once through study completion
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Change From Baseline in Localized Strength (Medical Research Council (MRC) Scale Grading) at Cycle 13.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
|
If motor dysfunction or weakness is evident, strength of the affected muscle groups will be measured by dynamometer and assessed by a muscle grading scale. Medical Research Council (MRC) Scale grading (0-5) must be conducted in accordance with the study reference manual. MRC Grading is assessed on a scale of 0 to 5, with 0 representing no movement being observed, and 5 representing the muscle contracting normally against full resistance. To assess the change from baseline at a visit, a participant will need assessments in which all the following categories, as assessed at baseline, match at that visit: side affected, position during assessment (sitting/supine/lateral decubitus), muscle group assessed. The MRC Grade visit score is calculated for each participant separately, and these scores are then averaged. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Mobility 8a.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
|
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported mobility. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 14 to 59 for self-report, and 14 to 56 for parent proxy with higher scores representing higher reported mobility and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Upper Extremities 8a.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
|
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher physical functioning in the upper extremities. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 10 to 57 for self-report, and 13 to 55 for parent proxy with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Adult v2.0 Physical Function 8b.
Periodo de tiempo: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
|
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants ≥ 18 years with a target PN impacting physical functioning complete a self-report of physical function. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported physical capability. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 20.3 to 60.1 with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10. |
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
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Colaboradores e Investigadores
Investigadores
- Director de estudio: Medical Responsible, SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Publicaciones y enlaces útiles
Publicaciones Generales
- Moertel CL, Hirbe AC, Shuhaiber HH, Bielamowicz K, Sidhu A, Viskochil D, Weber MD, Lokku A, Smith LM, Foreman NK, Hajjar FM, McNall-Knapp RY, Weintraub L, Antony R, Franson AT, Meade J, Schiff D, Walbert T, Ambady P, Bota DA, Campen CJ, Kaur G, Klesse LJ, Maraka S, Moots PL, Nevel K, Bornhorst M, Aguilar-Bonilla A, Chagnon S, Dalvi N, Gupta P, Khatib Z, Metrock LK, Nghiemphu PL, Roberts RD, Robison NJ, Sadighi Z, Stapleton S, Babovic-Vuksanovic D, Gershon TR; ReNeu Trial Investigators; ReNeu Study Investigators. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. J Clin Oncol. 2025 Feb 20;43(6):716-729. doi: 10.1200/JCO.24.01034. Epub 2024 Nov 8.
- Hirbe A, Moertel CL, Babovic-Vuksanovic D, Rohl C, Bischoff K, Kelts K, Langseth AJ, Weber MD, Oberlander B, Wunderlich A, Jared J, Gershon TR. Plain language summary of the ReNeu study: a study of mirdametinib for adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Future Oncol. 2026 Oct 29:1-21. doi: 10.1080/14796694.2026.2722495. Online ahead of print.
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades del Sistema Nervioso
- Neoplasias
- Enfermedades Neuromusculares
- Enfermedades Genéticas Congénitas
- Enfermedades del Sistema Nervioso Periférico
- Neoplasias por tipo histológico
- Enfermedades neurodegenerativas
- Neoplasias De Tejido Nervioso
- Neoplasias del Sistema Nervioso
- Trastornos Heredodegenerativos, Sistema Nervioso
- Neoplasias de la vaina nerviosa
- Síndromes Neoplásicos Hereditarios
- Síndromes neurocutáneos
- Neoplasias del Sistema Nervioso Periférico
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Neurofibromatosis
- Neurofibromatosis 1
- Neurofibroma
- Neurofibroma Plexiforme
- mirametinib
Otros números de identificación del estudio
- MEK-NF-201
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
IPD supporting publicly available results will be evaluated for sharing.
Marco de tiempo para compartir IPD
IPD from completed trials will be publicly available within 6 months after all of the following events:
- Approval of a product/new indication by major authorities (FDA, EMA, PMDA if requested) with no pending submissions
- Public results via primary manuscript or trial registry disclosure
- Legal authority to share data
- Privacy protections are in place If approval is not sought or development is globally discontinued, data will be publicly available within 18 months after global trial completion. Further information on how to request data can be found on our website bit.ly/IPD21
Criterios de acceso compartido de IPD
Qualified researchers may propose access to IPD from sponsored trials via https://vivli.org/members/ourmembers/
Tipo de información de apoyo para compartir IPD
- PROTOCOLO DE ESTUDIO
- SAVIA
- CÓDIGO_ANALÍTICO
- RSC
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .