A Phase 2 Study of CX-8998 in Adults With Tremor Associated With Parkinson's Disease

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of CX-8998 for Tremor Associated With Parkinson's Disease

Patrocinadores

Patrocinador principal: Jazz Pharmaceuticals

Fuente Jazz Pharmaceuticals
Resumen breve

This is a Phase 2, multicenter, double-blind, placebo-controlled, parallel-group study consisting of a screening period of up to 4 weeks, a 4 week randomized double-blind, dose-titration treatment period, followed by a 1 week safety follow-up period after the last dose of study medication, and a scheduled follow-up safety telephone call one week later.

Descripción detallada

This is a Phase 2, multicenter, double-blind, placebo-controlled, parallel-group study consisting of a screening period of up to 4 weeks, a 4 week randomized double-blind, dose-titration treatment period, followed by a 1 week safety follow-up period after the last dose of study medication, and a scheduled follow-up safety telephone call one week later.

Subjects will be randomized 1:1 to one of two treatment groups. Group A will receive titrating doses of CX-8998 up to 10 mg BID and Group B will receive placebo.

Subjects will participate for a total of up to 12 weeks, including screening, the 4-week treatment period and follow-up.

Estado general Not yet recruiting
Fecha de inicio December 2019
Fecha de Terminación December 2020
Fecha de finalización primaria September 2020
Fase Phase 2
Tipo de estudio Interventional
Resultado primario
Medida Periodo de tiempo
Change from Baseline to Day 28 on the MDS-UPDRS Tremor Score as scored by the central rater Baseline through completion of study treatment period, an average of 28 days
Resultado secundario
Medida Periodo de tiempo
Change from Baseline to Day 28 on the TETRAS Activity of Daily Living subscale Baseline through completion of study treatment period, an average of 28 days
Change from Baseline to Day 28 in accelerometry score Baseline through completion of study treatment period, an average of 28 days
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] as assessed by CTCAE v4.0 Through study completion, an average of 12 weeks
Changes from baseline in QTcF Baseline through study completion, an average of 5 weeks
Percentage of subjects who did not complete the study due to Treatment Emergent Adverse Events as assessed by CTCAE v4.0 Duration of study, an average of 12 weeks
Percentage of subjects with Serious Adverse Events as assessed by CTCAE v4.0 Duration of study, an average of 12 weeks
Percentage of subjects with Adverse Events of Special Interest as assessed by CTCAE v4.0 Duration of study, an average of 12 weeks
Columbia-Suicide Severity Rating Scale (C-SSRS) Screening through study completion, an average of 8 weeks
Epworth Sleepiness Scale Baseline through completion of study treatment period, an average of 28 days
Questionnaire for Impulsive-Compulsive Disorders in Parkinson's Disease-Rating Scale (QUIP-RS) Screening through study completion, an average of 8 weeks
University of Miami Parkinson's disease Hallucinations Questionnaire (UM-PDHQ) Screening, Baseline and Day 28, an average of 28 days
Hospital Anxiety and Depression Scale (HADS) Screening and Day 28, an average of 28 days
Inscripción 60
Condición
Intervención

Tipo de intervención: Drug

Nombre de intervención: CX-8998

Descripción: T-type calcium channel blocker

Etiqueta de grupo de brazo: CX-8998 T-type calcium channel blocker

Tipo de intervención: Drug

Nombre de intervención: Placebo

Descripción: Placebo comparator

Etiqueta de grupo de brazo: Comparator

Elegibilidad

Criterios:

Inclusion Criteria:

- Men or non-pregnant, non-breastfeeding women 40 to 80 years-of-age who are able to read and understand English.

- Mini Mental State Exam (MMSE) score ≥ 24.

- Clinical diagnosis of idiopathic Parkinson's disease and presence of at least 2 out of 3 cardinal characteristics (tremor, rigidity, and/or bradykinesia).

- Hoehn & Yahr Stage I III (inclusive) if not experiencing motor fluctuations. If experiencing motor fluctuations, must be Hoehn & Yahr Stage I IV (inclusive) when OFF or I-III (inclusive) when ON.

- An MDS-UPDRS tremor score (sum of items 2.10, 3.15, 3.16, 3.17, 3.18) of a least 10 (during ON for subjects experiencing fluctuations) (centrally rated) (Forjaz et al., 2015). A limited number of subjects with an MDS-UPDRS of 8 or 9 may be included with Sponsor approval.

- Treated with a stable regimen of anti-parkinsonian and/or anti-tremor medication (with the exception of primidone) for at least 2 weeks prior to screening. Changes to anti-parkinsonian or anti-tremor medications after screening is not permitted.

Exclusion Criteria:

- Current diagnosis of: a. essential tremor / b. cerebellar disease

- Presence or known history of: a. significant visual hallucinations (in the opinion of the Investigator and/or Study Safety Representative) / b. significant impulse control disorder (ICD) (in the opinion of the Investigator and/or Study Safety Representative).

- History or clinical features consistent with an atypical parkinsonian syndrome.

- Dyskinesia or dystonia that would, in the opinion of the investigator, central rater, or Sponsor, interfere with the assessment of tremor.

- Exposure to tremorigenic drugs or drug withdrawal states within the 30 days prior to the first planned dose of study drug.

- Direct or indirect trauma to the nervous system within 3 months preceding the onset of tremor.

- History or clinical evidence of psychogenic tremor origin. Known history of other medical or neurological conditions that may cause or explain subject's tremor.

- Prior MR-guided Focused Ultrasound or surgical intervention (e.g., deep brain stimulation, ablative thalamotomy or gamma knife thalamotomy) for treatment of tremor or Parkinson's disease.

- Use of medication(s) in the past month that might produce tremor or interfere with the evaluation of tremor.

- Inability to refrain from use of medication/substance(s) that might produce tremor or interfere with the evaluation of tremor on study visit days.

- Positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.

- Regular use of more than two units of alcohol per day.

- Use of prescription or non-prescription drugs or other products (i.e. grapefruit juice) known to be strong inhibitors or inducers of CYP3A4 which cannot be discontinued 2 weeks prior to Day 1 of dosing and withheld throughout the study.

- Concurrent illnesses that would be a contraindication to trial participation.

- Psychological, social, familial, or geographical reasons that would hinder or prevent compliance with the requirements of the protocol or compromise the informed consent process.

- Any other condition and/or situation that causes the Investigator or Study Safety Representative to deem a subject unsuitable for the study (e.g., due to expected study medication non-compliance, inability to medically tolerate the study procedures, or a subject's unwillingness to comply with study-related procedures).

- Treatment with an investigational agent within 30 days prior to the first dose of CX-8998 or planning to receive an investigational agent during the study.

Género: All

Edad mínima: 40 Years

Edad máxima: 80 Years

Voluntarios Saludables: No

Oficial general
Apellido Papel Afiliación
Stacey Boyer, PhD Study Director Jazz Pharmaceuticals
Contacto general

Apellido: Ryan O'Mara

Teléfono: 1.585.766.2886

Email: [email protected]

Ubicación
Instalaciones:
Parkinson's Disease and Movement Disorders Center of Boca Raton | Boca Raton, Florida, 33486, United States
University of Kansas Medical Center | Kansas City, Kansas, 66160, United States
Ubicacion Paises

United States

Fecha de verificación

April 2019

Fiesta responsable

Tipo: Sponsor

Tiene acceso ampliado No
Condición Examinar
Número de brazos 2
Grupo de brazo

Etiqueta: CX-8998 T-type calcium channel blocker

Tipo: Experimental

Etiqueta: Comparator

Tipo: Placebo Comparator

Datos del paciente Undecided
Información de diseño del estudio

Asignación: Randomized

Modelo de intervención: Parallel Assignment

Propósito primario: Treatment

Enmascaramiento: Double (Participant, Investigator)

Fuente: ClinicalTrials.gov