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

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ClinicalTrials.gov Identifier: NCT04301284
Recruitment Status : Withdrawn (In January 2021 Novartis acquired Cadent Therapeutics. As part of a pipeline reassessment, the Synchrony-1 trial will not proceed as initially scheduled.)
First Posted : March 10, 2020
Last Update Posted : April 8, 2021
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
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 Drug: CAD-1883 Drug: Placebos Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Estimated Study Start Date : June 2021
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : December 2022


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




Primary Outcome Measures :
  1. To evaluate the safety and tolerability of oral administration of CAD-1883 versus placebo: Incidence of Adverse Events [ Time Frame: 12 week treatment period ]
    Incidence of Adverse Events



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04301284


Locations
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United States, California
Collaborative Neuroscience Network
Long Beach, California, United States, 90806
UCLA Medical Center
Los Angeles, California, United States, 90095
United States, Colorado
University of Colorado
Aurora, Colorado, United States, 80045
United States, Florida
University of Florida
Gainesville, Florida, United States, 32608
University of South Florida: Ataxia Research Center
Tampa, Florida, United States, 33612
United States, Texas
Houston Methodist Research Institute
Houston, Texas, United States, 77030
Sponsors and Collaborators
Cadent Therapeutics
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Responsible Party: Cadent Therapeutics
ClinicalTrials.gov Identifier: NCT04301284    
Other Study ID Numbers: CAD1883-211
First Posted: March 10, 2020    Key Record Dates
Last Update Posted: April 8, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cadent Therapeutics:
Ataxia
SCA
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn