Study of CAD-1883 for Spinocerebellar Ataxia

Brief Title

Study of CAD-1883 for Spinocerebellar Ataxia

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)

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

To evaluate the safety and tolerability of oral administration of CAD-1883 versus placebo: Incidence of Adverse Events


Condition

Spinocerebellar Ataxias

Intervention

CAD-1883

Study Arms / Comparison Groups

 CAD-1883
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

48

Start Date

August 2020

Completion Date

December 2021

Primary Completion Date

November 2021

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04301284

Organization ID

CAD1883-211


Responsible Party

Sponsor

Study Sponsor

Cadent Therapeutics


Study Sponsor

, , 


Verification Date

April 2020