Study of CAD-1883 for Spinocerebellar Ataxia (Synchrony-1)

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)

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.

Study Overview

• Status: Withdrawn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

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 Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Long Beach, California, United States, 90806
      • Collaborative Neuroscience Network
    • Los Angeles, California, United States, 90095
      • UCLA Medical Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Florida
    • Gainesville, Florida, United States, 32608
      • University of Florida
    • Tampa, Florida, United States, 33612
      • University of South Florida: Ataxia Research Center
  • Texas
    • Houston, Texas, United States, 77030
      • Houston Methodist Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: CAD-1883; 150 mg filled capsules
Placebo Comparator: 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.	Drug: Placebos; capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Cadent Therapeutics

Study record dates

Study Major Dates

• Study Start (Anticipated): June 1, 2021
• Primary Completion (Anticipated): November 1, 2022
• Study Completion (Anticipated): December 1, 2022

Study Registration Dates

• First Submitted: March 6, 2020
• First Submitted That Met QC Criteria: March 9, 2020
• First Posted (Actual): March 10, 2020

Study Record Updates

• Last Update Posted (Actual): April 8, 2021
• Last Update Submitted That Met QC Criteria: April 5, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Keywords: SCA; Ataxia
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Machado-Joseph Disease
• Other Study ID Numbers: CAD1883-211

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No