NCT04301284

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.

Trial Health

30
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Jun 2021

Shorter than P25 for phase_2

Geographic Reach
1 country

6 active sites

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 6, 2020

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 10, 2020

Completed
1.2 years until next milestone

Study Start

First participant enrolled

June 1, 2021

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2022

Completed
Last Updated

April 8, 2021

Status Verified

April 1, 2021

Enrollment Period

1.4 years

First QC Date

March 6, 2020

Last Update Submit

April 5, 2021

Conditions

Spinocerebellar AtaxiasSpinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2Spinocerebellar Ataxia Type 3Spinocerebellar Ataxia Type 6Spinocerebellar Ataxia Type 7Spinocerebellar Ataxia Type 8Spinocerebellar Ataxia Type 10Spinocerebellar Ataxia Type 17ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1

Keywords

AtaxiaSCA

Outcome Measures

Primary Outcomes (1)

  • 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

Study Arms (2)

CAD-1883

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

CAD-1883DRUG

150 mg filled capsules

CAD-1883
PlacebosDRUG

capsules

Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Collaborative Neuroscience Network

Long Beach, California, 90806, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

University of South Florida: Ataxia Research Center

Tampa, Florida, 33612, United States

Location

Houston Methodist Research Institute

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Spinocerebellar AtaxiasMachado-Joseph DiseaseSpinocerebellar ataxia 8Spinocerebellar Ataxia 10Olivopontocerebellar Atrophy VAtaxia

Condition Hierarchy (Ancestors)

Cerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinocerebellar DegenerationsSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesDyskinesiasNeurologic ManifestationsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSigns and SymptomsPathological Conditions, Signs and Symptoms
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 6, 2020

First Posted

March 10, 2020

Study Start

June 1, 2021

Primary Completion

November 1, 2022

Study Completion

December 1, 2022

Last Updated

April 8, 2021

Record last verified: 2021-04

Data Sharing

IPD Sharing
Will not share

Locations

US(6)