Study of CAD-1883 for Spinocerebellar Ataxia

Phase 2

Withdrawn

• Conditions: Spinocerebellar Ataxias; Spinocerebellar Ataxia Type 3; Spinocerebellar Ataxia Type 7; ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1; Spinocerebellar Ataxia Type 2; Spinocerebellar Ataxia Type 8; Spinocerebellar Ataxia Type 1; Spinocerebellar Ataxia Type 6; Spinocerebellar Ataxia Type 10; Spinocerebellar Ataxia Type 17
• Interventions: Drug: Placebos; Drug: CAD-1883

• Registration Number: NCT04301284
• Lead Sponsor: Cadent Therapeutics

Brief 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.

Detailed Description

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.

Study Timeline

• Study First Submitted: 2020-03-06
• Study First Submitted QC: 2020-03-09
• Study First Posted: 2020-03-10
• Status Verified: 2021-04
• Last Update Submitted: 2021-04-05
• Last Update Posted: 2021-04-08
• Study Start: 2021-06
• Primary Completion: 2022-11
• Study Completion: 2022-12

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Key

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebos	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.
CAD-1883	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.

Primary Outcome Measures

Name	Time	Method
To evaluate the safety and tolerability of oral administration of CAD-1883 versus placebo: Incidence of Adverse Events	12 week treatment period	Incidence of Adverse Events

Trial Locations

Locations (6)

Collaborative Neuroscience Network; 🇺🇸 Long Beach, California, United States

Houston Methodist Research Institute; 🇺🇸 Houston, Texas, United States

UCLA Medical Center; 🇺🇸 Los Angeles, California, United States

University of Colorado; 🇺🇸 Aurora, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of South Florida: Ataxia Research Center; 🇺🇸 Tampa, Florida, United States