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

Primary Purpose

Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2

Status
Withdrawn
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
CAD-1883
Placebos
Sponsored by
Cadent Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinocerebellar Ataxias focused on measuring Ataxia, SCA

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Collaborative Neuroscience Network
  • UCLA Medical Center
  • University of Colorado
  • University of Florida
  • University of South Florida: Ataxia Research Center
  • Houston Methodist Research Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

CAD-1883

Placebo

Arm 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.

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

First Posted
March 6, 2020
Last Updated
April 5, 2021
Sponsor
Cadent Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04301284
Brief Title
Study of CAD-1883 for Spinocerebellar Ataxia
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Withdrawn
Why Stopped
In January 2021 Novartis acquired Cadent Therapeutics. As part of a pipeline reassessment, the Synchrony-1 trial will not proceed as initially scheduled.
Study Start Date
June 2021 (Anticipated)
Primary Completion Date
November 2022 (Anticipated)
Study Completion Date
December 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cadent Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
Keywords
Ataxia, SCA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
CAD-1883
Arm Type
Experimental
Arm 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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
CAD-1883
Intervention Description
150 mg filled capsules
Intervention Type
Drug
Intervention Name(s)
Placebos
Intervention Description
capsules
Primary Outcome Measure Information:
Title
To evaluate the safety and tolerability of oral administration of CAD-1883 versus placebo: Incidence of Adverse Events
Description
Incidence of Adverse Events
Time Frame
12 week treatment period

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Collaborative Neuroscience Network
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
University of South Florida: Ataxia Research Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Houston Methodist Research Institute
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Study of CAD-1883 for Spinocerebellar Ataxia

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