Study of CAD-1883 for Spinocerebellar Ataxia

Spinocerebellar Ataxias Clinical Trial

— Synchrony-1  

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)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04301284
• Other study ID #: CAD1883-211
• Status: Withdrawn
• Phase: Phase 2
• Start date: June 2021
• Est. completion date: December 2022

Study information

• Verified date: April 2021
• Source: Cadent Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 2022
• Est. primary completion date: November 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Key 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

Related Conditions & MeSH terms

• ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1
• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 1
• Spinocerebellar Ataxia Type 10
• Spinocerebellar Ataxia Type 17
• Spinocerebellar Ataxia Type 2
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxia Type 6
• Spinocerebellar Ataxia Type 7
• Spinocerebellar Ataxia Type 8
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Drug:
• CAD-1883
150 mg filled capsules
• Placebos
capsules

Locations

Country	Name	City	State
United States	University of Colorado	Aurora	Colorado
United States	University of Florida	Gainesville	Florida
United States	Houston Methodist Research Institute	Houston	Texas
United States	Collaborative Neuroscience Network	Long Beach	California
United States	UCLA Medical Center	Los Angeles	California
United States	University of South Florida: Ataxia Research Center	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Cadent Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the safety and tolerability of oral administration of CAD-1883 versus placebo: Incidence of Adverse Events	Incidence of Adverse Events	12 week treatment period