Study of CAD-1883 for Spinocerebellar Ataxia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-30

Study Completion Date

2022-12-31

Brief Summary

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This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA). This study offers the opportunity to understand the safety, tolerability, and efficacy of CAD-1883 in the SCA patient population.

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Detailed Description

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This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of SCA using multiple dose levels. The study will include multiple cohorts of 16 patients each where 12 patients will be randomized to CAD-1883 and 4 to matching placebo.

Potential subjects will undergo a screening period (14 to 28 days), a baseline (Day 1) visit, and a 12-week treatment period. A follow-up visit will occur 4 weeks after the end of the treatment period. The total duration of individual subject participation may be up to 20 weeks, depending on the duration of the screening period.

The study will assess safety by adverse events, vital signs, laboratory parameters (including chemistry, hematology and urinalysis); pharmacokinetics of CAD-1883; and efficacy measures by Scale for the Assessment and Rating of Ataxia (SARA), clinician/patient rating of ataxia and symptoms, patient quality of life measures and wearable sensors to capture falls and gait measurements.

For planning purposes, the anticipated study completion date assumes evaluation of 3 cohorts.

Conditions

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Spinocerebellar Ataxias Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia Type 2 Spinocerebellar Ataxia Type 3 Spinocerebellar Ataxia Type 6 Spinocerebellar Ataxia Type 7 Spinocerebellar Ataxia Type 8 Spinocerebellar Ataxia Type 10 Spinocerebellar Ataxia Type 17 ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CAD-1883

Capsules of 150 mg of CAD-1883 will be administered orally, twice daily (BID). The second daily dose will be taken 8 hours (+/- 2 hours) after the first daily dose.

The initial dose regimen evaluated will be 150 mg BID. Additional dose regimens up to 600 mg BID will be determined based on forthcoming clinical data.

Group Type EXPERIMENTAL

CAD-1883

Intervention Type DRUG

150 mg filled capsules

Placebo

Matching placebo will be provided in capsules, to be administered orally, twice daily. The second daily dose will be taken 8 hours (+/- 2 hours) after the first daily dose.

Group Type PLACEBO_COMPARATOR

Placebos

Intervention Type DRUG

capsules

Interventions

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CAD-1883

150 mg filled capsules

Intervention Type DRUG

Placebos

capsules

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Prior Spinocerebellar Ataxia (SCA) diagnosis of the following SCA genotypes: SCA1, 2, 3, 6, 7, 8, 10, 17, or Autosomal Recessive Cerebellar Ataxia Type 1 (ARCA1).
* Scale for the Assessment and Rating of Ataxia (SARA) Total score ≥8 and \<30 at Screening
* SARA item #1 (gait) score of ≥2 at Screening
* Ability to ambulate 8 meters at Screening without assistance from another person

Exclusion Criteria

* Neurological condition other than SCA that could predominantly explain or contribute significantly to the subjects' symptoms of ataxia or that could confound the assessment of ataxia symptoms (e.g., chronic alcoholism, vitamin deficiencies, multiple sclerosis, Parkinson's disease, Friedreich's ataxia, vascular disease, tumors, paraneoplastic disease, head injury, idiopathic late onset ataxia, multisystem atrophy, stroke, arthritis, cerebral palsy, spasticity of unknown origin).
* Moderate or severe scores on the following Inventory of Non-Ataxia Signs (INAS) items at Screening: Dystonia: at least 3 of 5 items; Spasticity: at least 2 of 3 items; Rigidity: at least 2 of 3 items

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No