Summary

Eligibility
for people ages 18-75 (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

Summary

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.

Official Title

Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of CAD-1883 in Adults With Spinocerebellar Ataxia (Synchrony-1)

Details

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.

Keywords

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 Ataxia SCA Cerebellar Ataxia Spinocerebellar Degenerations Machado-Joseph Disease CAD-1883

Eligibility

You can join if…

Open to people ages 18-75

  • 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

You CAN'T join if...

  • 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

Locations

  • UCLA Medical Center
    Los Angeles California 90095 United States
  • Collaborative Neuroscience Network
    Long Beach California 90806 United States

Details

Status
currently not accepting new patients, but might later
Start Date
Completion Date
(estimated)
Sponsor
Cadent Therapeutics
ID
NCT04301284
Phase
Phase 2
Study Type
Interventional
Last Updated