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The Study of Brave-LC LDN Treatment Trial

Brave-LC: A Phase 3 Randomized Double-blind, Placebo-controlled Trial to Evaluate the Efficacy of a 16-week Course of Low-dose Naltrexone to Reduce Fatigue Severity in Persons Aged 6 Through 25 Years With Long COVID

This is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating the efficacy and safety of a 16-week course of low-dose naltrexone (LDN) in approximately 1,300 participants aged 6 to 25 years with fatigue associated with Long COVID. Participants will be enrolled across three age cohorts (6-11, 12-17, and 18-25 years) and randomized 1:1 to receive LDN capsules or matching placebo capsules. LDN will be initiated at 1.5 mg daily and titrated weekly to a maximum tolerated dose of up to 4.5 mg daily. Randomization will be stratified by age cohort, and participants will also be randomized to a pharmacokinetic (PK) sampling window. Primary objective is to compare the change in fatigue severity from baseline to 16 weeks between participants aged 6 to 25 years with Long COVID randomized to a 16-week course of LDN or placebo.

Visão geral do estudo

Status

Ainda não está recrutando

Condições

Intervenção / Tratamento

Descrição detalhada

This is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating the efficacy and safety of a 16-week course of low-dose naltrexone (LDN) in approximately 1,300 participants aged 6 to 25 years with fatigue associated with Long COVID. Participants will be enrolled across three age cohorts (6-11, 12-17, and 18-25 years) and randomized 1:1 to receive LDN capsules or matching placebo capsules. LDN will be initiated at 1.5 mg daily and titrated weekly to a maximum tolerated dose of up to 4.5 mg daily. Randomization will be stratified by age cohort, and participants will also be randomized to a pharmacokinetic (PK) sampling window. Primary objective is to compare the change in fatigue severity from baseline to 16 weeks between participants aged 6 to 25 years with Long COVID randomized to a 16-week course of LDN or placebo. Secondary objectives: To compare the change in fatigue severity from baseline to 16 weeks between participants with Long COVID in age groups: 6 to 11 years, 12 to 17 years and 18-25 years. Each participant will be randomized to a 16-week course of LDN or placebo.

Tipo de estudo

Intervencional

Inscrição (Estimado)

1300

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Locais de estudo

    • Alabama
      • Birmingham, Alabama, Estados Unidos, 35233-0011
        • Children's of Alabama Child Health Research Unit (CHRU)
    • California
      • Chula Vista, California, Estados Unidos, 91910
        • Exalt Clinical Research Inc.
    • Colorado
      • Denver, Colorado, Estados Unidos, 80204-4506
        • Denver Health
    • Florida
      • Gainesville, Florida, Estados Unidos, 32610
        • University of Florida Health
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60612
        • Rush University Medical Center
    • Maryland
      • Baltimore, Maryland, Estados Unidos, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02115-6110
        • Brigham and Women's Hospital
      • Worcester, Massachusetts, Estados Unidos, 01655
        • University of Massachusetts Chan Medical School
    • New Hampshire
      • Lebanon, New Hampshire, Estados Unidos, 03756-1000
        • Dartmouth Health
    • New Jersey
      • New Brunswick, New Jersey, Estados Unidos, 08901-1935
        • Rutgers Robert Wood Johnson Medical School
    • New York
      • Brooklyn, New York, Estados Unidos, 11220
        • NYU Langone Hospital - Brooklyn
      • New York, New York, Estados Unidos, 10032
        • Columbia University
    • Texas
      • Houston, Texas, Estados Unidos, 77030
        • Baylor College of Medicine

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

6 anos a 25 anos (Filho, Adulto)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

Inclusion Criteria:

  1. Aged 6 to 25 years, inclusive, at the time of randomization.

  2. Fluent in English and/or Spanish.

  3. History of SARS CoV 2 infection shown by (1) a positive nucleic acid amplification test (NAAT) or antigen test, OR (2) meeting clinical criteria* AND an epidemiologic link** to a confirmed case.

    *Clinical criteria is defined as either (1) acute onset of fever AND cough; OR (2) acute altered/loss of taste or smell + one additional symptom including: fever, cough, general weakness/fatigue, headache, myalgia, sore throat, coryza, dyspnea, nausea, diarrhea, or anorexia; OR (3) three or more symptoms, including fever, cough, general weakness/fatigue, headache, myalgia, sore throat, coryza, dyspnea, nausea, diarrhea, or anorexia.

    • Epidemiologic link is defined as having contact (within 6 feet for at least 15 minutes) with someone sick with SARS CoV 2 test-positive COVID 19 illness.
  4. Clinical evidence of Long COVID* and Long COVID symptoms that cannot be attributed to another cause upon investigator review of medical history and workup participant received prior to study entry.

    *Participants must meet the 2024 National Academies of Sciences, Engineering, and Medicine definition for Long COVID.

  5. Has new fatigue*, ** that began either during or in the six months following the onset of the qualifying SARS CoV 2 infection or COVID 19 illness (see inclusion Criterion #3):

    *New fatigue symptoms can fluctuate but the total duration of fatigue must be at least 3 months at time of randomization and fatigue must be present for more days than not in the 30 days prior to randomization.

    **For this trial, fatigue is defined as having physical fatigue and/or cognitive fatigue (e.g., has trouble with memory, focus/attention, or decision-making, or is slow to process information, and is sometimes referred to as having "brain fog"). Fatigue is a persistent state of physical and/or cognitive exhaustion that is not substantially alleviated by sleep or rest, and that interferes with normal functioning, is often profound, and is not the result of ongoing or unusual excessive exertion.

  6. Has at least one new or worsening symptom*, ** that began either during or in the six months following the onset of the qualifying SARS CoV 2 infection or COVID 19 illness:

    *List of qualifying symptoms include: systemic symptoms (chills, Post-Exertional Malaise [PEM]); neurological symptoms (headache, dysautonomia/ Postural Orthostatic Tachycardia Syndrome [POTS], dizziness, unsteadiness, or neuropathy); sleep disturbance resulting in unrefreshing sleep; cardiopulmonary symptoms (chest pain, palpitations, shortness of breath, cough, fainting spells); musculoskeletal symptoms (muscle pain/aches, neck pain, back pain, joint pain); or gastrointestinal symptoms (nausea, diarrhea).

    **While the qualifying symptom(s) may fluctuate, the total duration of at least one qualifying symptom must be at least 3 months at time of randomization. In addition, that symptom must be present for more days than not in the 30 days prior to randomization.

  7. Participant scores <70 on the Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL-MFS) at Visit 01A.

  8. Willing and able to provide informed consent and assent (as applicable) before initiation of any study procedures.

  9. Able to understand and agree to comply with all planned study procedures and be available for all study visits.

    *Participants still may enroll if they decline genetic testing or if they are unable or unwilling to complete blood draw(s) for biomarkers. An initial blood draw for baseline clinical laboratory assessments is required to be completed prior to randomization.

  10. Participants of childbearing potential* must agree to use highly effective contraception** or practice true abstinence*** throughout the study (Visit 08).

    *Persons of childbearing potential is defined as post-menarchal biological females who have not reached menopause or undergone surgical sterilization. Menopause is defined as having no menses for at least 12 months without an alternative medical cause for amenorrhea.

    **Highly effective contraception includes hormonal contraception (oral, implant, injection, transdermal patch, vaginal ring), intrauterine device (IUD), bilateral tubal ligation, or partner vasectomy.

    ***True abstinence is defined as no heterosexual vaginal/penile intercourse at any time during the study

  11. Participants capable of fathering a child* and having sex with women of childbearing potential must agree to use contraception (i.e., condoms) throughout the study (Visit 08).

    *Persons capable of fathering a child are biological males who are post-pubertal and not permanently sterile by bilateral orchidectomy or vasectomy

  12. Agrees to utilize electronic data capture for the e-Memory Aid and electronic PROs and have access to a device connected to the internet for completion.

  13. Agrees to adhere to all lifestyle considerations.

Exclusion Criteria:

  1. Known acute SARS CoV 2 infection </= 30 days before randomization.

  2. Current or recent opioid use in the 7 days before randomization, as reported by the participant or their parent/legal guardian.

  3. Plans to have elective surgery or procedure which may involve use of opioid medication during the study intervention.

  4. Known allergy/sensitivity/hypersensitivity to naltrexone or its components.

  5. Current or recent use of naltrexone in the 14 days before randomization.

  6. History of use of LDN for >/= 30 days duration to treat Long COVID in the 6 months before randomization.

  7. Enrolled at the time of consent/assent or planned enrollment in another interventional clinical trial during the trial.

  8. Plans to become pregnant or to father a child during the trial or is pregnant or breastfeeding at randomization.

  9. Has a positive pregnancy test at Visit 01A.* *All participants of childbearing potential will have a urine pregnancy test at Visit 01A, with results confirmed as negative before randomization.

  10. Has known active acute hepatitis or a history of chronic liver disease (known Child-Pugh Class B or C) or known severe chronic kidney disease and/or kidney failure (Estimated Glomerular Filtration Rate [eGFR] < 30 ml/min/1.73 m^2).

  11. Has known active Hepatitis B (HBV) infection, active Hepatitis C (HCV) infection, or unstable Human Immunodeficiency Virus (HIV) (i.e., not on antiretroviral therapy (ART) or on ART but has detectable viral load).

  12. Has recent active malignancy (received chemotherapy or radiotherapy in the 12 months prior to randomization) or active malignancy at randomization*.

    • Long COVID patients with non-melanoma skin carcinoma (basal cell or squamous cell skin cancer), or low-grade cervical squamous intraepithelial lesions, which are considered cured and not requiring ongoing therapy, may be enrolled at the discretion of the investigator.
  13. Began taking a new medication*, supplement, or vitamin or changed doses of a medication, supplement, or vitamin the 14 days prior to randomization.

    *Topical and intranasal (non-opioid) medications are not exclusionary.

  14. Diagnosed in the 3 months prior to randomization with a new medical condition*, other than Long COVID, causing new or worsening fatigue that is unresolved at time of randomization.

    *Medical conditions that could cause new or worsening fatigue include but are not limited to iron deficiency with or without severe anemia, hypothyroidism, infectious mononucleosis (Epstein-Barr Virus or cytomegalovirus infection), celiac disease, type 1 diabetes mellitus, acute leukemia or hematologic malignancy, adrenal insufficiency, cardiac disease (e.g. myocarditis, arrythmia, or cardiomyopathy), metabolic disease, renal disease, or a primary sleep disorders (e.g. obstructive sleep apnea, restless legs syndrome, narcolepsy, or parasomnia).

  15. Has a clinically significant mental health condition defined by history of one or more of the following:

    a. History of prior suicide attempt. b. Hospitalized in the 6 months prior to randomization for mental health symptoms or worsening of a known mental health condition.

    c. Has moderate-risk or high-risk score on C SSRS at Visit 01A, as applicable. d. Has a diagnosis of severe conduct disorder*that, in the site investigator's judgment, may pose a safety risk or interfere with the ability to adequately assess clinical trial endpoints.

    *The American Psychiatric Association defines conduct disorder as a repetitive and persistent pattern of behavior in which the basic rights of others or major age-appropriate societal norms or rules are violated (e.g., history of recurrent aggressive behavior toward people or animals, and/or serious violations of rules or laws like running away, possessing or using weapons, vandalism). If the site Investigator is unsure if a participant has a diagnosis of severe conduct disorder, they may refer to the DSM-5-TR criteria.

  16. Has any medical condition*, ** that, in the site investigator's judgment, pose a safety risk or interfere with the ability to adequately assess clinical trial endpoints.

    • Medical conditions include but are not limited to severe esophagitis or a condition that precludes participant from taking study product; an active unstable or progressive major psychiatric or neurologic condition, including severe developmental disabilities, new or untreated psychosis or schizophrenia, new or untreated bipolar I disorder; and acute opioid withdrawal disorder to include participants maintained on opiate agonists (methadone) or partial agonists (buprenorphine) and those who has failed a naloxone challenge test.

      • Of note, an abnormal vital sign alone is not exclusionary at baseline. However, the abnormal vital sign may be indicative of an exclusionary medical condition as described above.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Low-Dose Naltrexone

Participants will receive a low-dose naltrexone (LDN) capsule that will be administered orally once daily for 16 weeks. LDN will be initiated at 1.5 mg daily and titrated weekly to a maximum tolerated dose of up to 4.5 mg daily. N=650

Opioid receptor antagonist used to treat opioid and alcohol use disorders in adults. Formulated as naltrexone hydrochloride, a white, crystalline compound freely soluble in water.

Comparador de Placebo: Placebo

Participants will receive a matching placebo capsule that will be administered orally once daily for 16 weeks. The placebo arm will follow the same dosing schedule and titration scheme as the LDN arm. N=650

Matching placebo capsule that is visually identical to the active study product and is administered orally once daily according to the same dosing schedule and titration scheme for 16 weeks.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Change from baseline in Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) total score.
Prazo: Baseline to 16 weeks after randomization

The Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and is scored from 0 to 100; higher scores represent lower levels of fatigue.

Baseline to 16 weeks after randomization

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Change from baseline Delis-Kaplan Executive Function System (D-KEFS) Category Fluency t-score
Prazo: Baseline to 16 weeks after randomization

The D-KEFS Verbal Fluency Category Fluency condition measures verbal fluency and executive function.

Participants generate as many words as possible beginning with a specified letter within a fixed time.

Performance score is scored by the number of correct words and interpreted using age-appropriate normative data.

This outcome applies to English-speaking participants aged 8 to 25 years.

Baseline to 16 weeks after randomization
Change from baseline Delis-Kaplan Executive Function System (D-KEFS) Letter Fluency t-score
Prazo: Baseline to 16 weeks after randomization

The D-KEFS Verbal Fluency Letter Fluency condition measures verbal fluency and executive function.

Participants generate as many words as possible beginning with a specified letter within a fixed time.

Performance score is scored by the number of correct words and interpreted using age-appropriate normative data.

This outcome applies to English-speaking participants aged 8 to 25 years.

Baseline to 16 weeks after randomization
Change from baseline in Behavior Rating Inventory of Executive Function-Second Edition (BRIEF-2) Global Executive Composite t-score
Prazo: Baseline to 16 weeks after randomization

The BRIEF-2 is a standardized questionnaire assessing executive function and self-regulation in participants aged 6 to 17 years.

Scores are interpreted using age- and sex-appropriate normative data. Higher scores indicate greater impairment in executive functioning.

Baseline to 16 weeks after randomization
Change from baseline in Behavior Rating Inventory of Executive Function-Second Edition (BRIEF-2A) Global Executive Composite t-score
Prazo: Baseline to 16 weeks after randomization

The BRIEF-2A is a standardized 75-item self-report assessment of executive functioning in participants aged 18 years and older.

Scores are interpreted using age- and sex-appropriate normative data. Higher scores indicate greater impairment in executive functioning.

Baseline to 16 weeks after randomization
Change from baseline in Conners Continuous Performance Test-Third Edition (CPT-3) Detectability t-score.
Prazo: Baseline to 16 weeks after randomization

The CPT-3 is a computerized assessment of sustained attention and response control for participants aged 8 years and older.

Performance is automatically recorded and interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Conners Continuous Performance Test-Third Edition (CPT-3) Hit Reaction Time t-score
Prazo: Baseline to 16 weeks after randomization

The CPT-3 is a computerized assessment of sustained attention and response control for participants aged 8 years and older.

Performance is automatically recorded and interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Connors Kiddie Continuous Performance Test (K-CPT-2) Detectability t-score.
Prazo: Baseline to 16 weeks after randomization

The K-CPT-2 Computerized assessment of sustained attention and response control for participants aged 6 to 7 years. Performance is automatically recorded and interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Connors Kiddie Continuous Performance Test (K-CPT-2) Hit Reaction Time t-score
Prazo: Baseline to 16 weeks after randomization

The K-CPT-2 Computerized assessment of sustained attention and response control for participants aged 6 to 7 years. Performance is automatically recorded and interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Developmental Neuropsychological Assessment (NEPSY-II) Semantic Word Generation Scaled score.
Prazo: Baseline to 16 weeks after randomization

The NEPSY-II Word Generation subtest measures verbal fluency and executive function. Participants generate words within semantic category during 60-second time limit.

Performance is scored by the number of correct words[TS3.1] and interpreted using age-appropriate normative data.

This outcome applies to English-speaking participants aged 6 to 7 years.

Baseline to 16 weeks after randomization
Change from baseline in Digit Span-Wechsler Intelligence Scale for Children-Fifth Edition (WISC-V) t-score.
Prazo: Baseline to 16 weeks after randomization

The WISC-V Digit Span subtest assesses attention and working memory in participants aged 6 to 15 years.

Raw scores are based on Digit Span Forward, Backward, and Sequencing tasks and are interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Digit Span-Wechsler Intelligence Scale for Children-Fourth Edition (WISC-IV) t-score.
Prazo: Baseline to 16 weeks after randomization

The WISC-IV Digit Span subtest assesses attention and working memory in participants aged 16 years and older.

Raw scores are based on Digit Span Forward, Backward, and Sequencing tasks and are interpreted using age-appropriate normative data.

Baseline to 16 weeks after randomization
Change from baseline in Functional Disability Inventory (FDI) total score.
Prazo: Baseline to 16 weeks after randomization

The Functional Disability Inventory (FDI) contains 15 items rated from 0 ('no trouble') to 4 ('impossible').

Total scores range from 0 to 60. Higher scores represent greater functional disability and more impairment in daily functioning.

Baseline to 16 weeks after randomization
Change from baseline in Patient Global Impression of Severity (PGI-S)
Prazo: Baseline to 16 weeks after randomization

The Patient Global Impression of Severity (PGI-S) is a single-item questionnaire rated on a 5-point Likert scale from 1 ('None') to 5 ('Very severe').

Higher scores reflect greater perceived illness or symptom severity.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Adult Pain Intensity Scale t-score in participants aged 18 to 25 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity Scale asks about pain over the past 7 days.

Items are rated on a 5-point scale from 1 ('not at all') to 5 ('very much'). Results are reported as T-scores. Higher scores representing greater pain intensity.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Adult Pain Interference Scale t-score in participants aged 18 to 25 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity Scale asks how pain affects activities, mood, and social participation over the past 7 days.

Items are rated on a 5-point scale from 1 ('not at all') to 5 ('very much'). Results are reported as T-scores. Higher scores representing greater pain interference.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Parent-Proxy Pain Intensity Scale t-score in participants aged 6 to 7 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity scale asks about pain over the past 7 days.

Items are rated on a 5-point scale from 1 ('not at all') to 5 ('very much'). Results are reported as T-scores, with higher scores representing greater pain intensity.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Parent-Proxy Pain Interference Scale t-score in participants aged 6 to 7 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Parent-Proxy Pain Interference Scale asks how pain affects activities, mood, and social participation over the past 7 days.

Items are rated on a 5-point scale from 1('not at all') to 5('very much'). Results are reported as T-scores. Higher scores representing greater pain interference.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Intensity Scale t-score in participants aged 8 to 17 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Intensity Scale asks about pain over the past 7 days.

Items are rated on a 5-point scale from 1 ('not at all') to 5 ('very much'). Results are reported as T-scores. Higher scores representing greater pain intensity.

Baseline to 16 weeks after randomization
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference Scale t-score in participants aged 8 to 17 years.
Prazo: Baseline to 16 weeks after randomization

The Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference scale asks how pain affects activities, mood, and social participation over the past 7 days.

Items are rated on a 5-point scale from 1 ('not at all') to 5 ('very much'). Results are reported as T-scores. Higher scores representing greater pain interference.

Baseline to 16 weeks after randomization
Change from baseline in Pediatric Quality of Life (PedsQL) Generic Core Scales Short Form 15 total score.
Prazo: Baseline to 16 weeks after randomization

The PedsQL Generic Core Scales Short Form 15 contains 15 Likert-type items across physical, emotional, social, and school functioning and is scored from 0 to 100.

Higher scores represent better quality of life.

Baseline to 16 weeks after randomization
Change from baseline in Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) subscale scores.
Prazo: Baseline to 16 weeks after randomization

The Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and includes general fatigue, sleep/rest fatigue, and cognitive fatigue subscales. Scores are reported on a 0 to 100 scale.

Higher scores represent lower levels of fatigue.

Baseline to 16 weeks after randomization
Change from baseline in Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) total score in participants aged 12 to 17 years.
Prazo: Baseline to 16 weeks after randomization

The Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and is scored from 0 to 100; higher scores represent lower levels of fatigue.

Baseline to 16 weeks after randomization
Change from baseline in Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) total score in participants aged 18 to 25 years.
Prazo: Baseline to 16 weeks after randomization

The Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and is scored from 0 to 100; higher scores represent lower levels of fatigue.

Baseline to 16 weeks after randomization
Change from baseline in Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) total score in participants aged 6 to 11 years.
Prazo: Baseline to 16 weeks after randomization

The Pediatric Quality of Life - Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and is scored from 0 to 100; higher scores represent lower levels of fatigue.

Baseline to 16 weeks after randomization
Change in Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL-MFS) total score from 16 to 26 weeks after randomization.
Prazo: 16 to 26 weeks after randomization

The Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL-MFS) contains 18 Likert-type items and is scored from 0 to 100.

Higher scores represent lower levels of fatigue.

16 to 26 weeks after randomization
Incidence of new infections.
Prazo: First administration of study product through 6 months after randomization

New infections will be summarized by frequency, percentage, and incidence rate. Severity and relatedness will also be summarized as applicable.

First administration of study product through 6 months after randomization
Incidence of new-onset chronic medical conditions (NOCMCs).
Prazo: First administration of study product through 6 months after randomization

NOCMCs will be assessed by number, severity, and relatedness and summarized by frequency, percentage, and incidence rate.

First administration of study product through 6 months after randomization
Incidence of solicited adverse events (AEs).
Prazo: First administration of study product through 8 weeks after randomization

Solicited AEs will be assessed by number, severity, and relatedness according to the DAIDS table for Grading the Severity of Adult and Pediatric Adverse Event, version 2.1.

First administration of study product through 8 weeks after randomization
Incidence of treatment emergent serious adverse events (SAEs).
Prazo: First administration of study product through 6 months after randomization

Treatment-emergent SAEs will be assessed by number, severity, and relatedness and summarized by frequency, percentage, and incidence rate.

First administration of study product through 6 months after randomization
Incidence of treatment emergent unsolicited adverse events (AEs).
Prazo: First administration of study product through 16 weeks after randomization

Treatment-emergent unsolicited AEs will be assessed by number, severity, and relatedness according to the DAIDS table for Grading the Severity of Adult and Pediatric Adverse Event, version 2.1.

First administration of study product through 16 weeks after randomization
Incidence of treatment-emergent medically attended adverse events (MAAEs).
Prazo: First administration of study product through 6 months after randomization

Treatment-emergent MAAEs will be assessed by number, severity, and relatedness and summarized by frequency, percentage, and incidence rate.

First administration of study product through 6 months after randomization
Patient Global Impression of Change (PGI-C) at 16 weeks after randomization
Prazo: 16 weeks after randomization

The PGI-C is a 7-point self-report scale ranging from 'very much improved'[TS4.1] to 'very much worse'[TS5.1] and measures overall improvement from treatment.

16 weeks after randomization

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Investigadores

  • Investigador principal: Laura Malone, MD PhD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

16 de novembro de 2026

Conclusão Primária (Estimado)

31 de dezembro de 2028

Conclusão do estudo (Estimado)

1 de janeiro de 2029

Datas de inscrição no estudo

Enviado pela primeira vez

29 de outubro de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

29 de outubro de 2026

Primeira postagem (Real)

4 de novembro de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

4 de novembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

29 de outubro de 2026

Última verificação

28 de outubro de 2026

Mais Informações

Termos relacionados a este estudo

Outros números de identificação do estudo

  • 25-0018
  • 1OT2HL156812 (Concessão/Contrato do NIH dos EUA)

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

produto fabricado e exportado dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .