Clinical trial

A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

Name

DTX-216-102

Description

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.

Trial arms

Trial start

2022-09-27

Estimated PCD

2023-08-25

Trial end

2023-08-25

Status

Completed

Phase

Early phase I

Treatment

DT-216

DT-216 will be administered by intravenous (IV) injection

Arms:

Multiple Dose: DT-216

Placebo

Matching Placebo will be administered by intravenous (IV) injection

Arms:

Multiple Dose: DT-216 matching placebo

Size

Primary endpoint

Safety Measure

Up to approximately 60 days

Eligibility criteria

Inclusion Criteria: * Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions. * Able and willing to sign informed consent form prior to study enrollment. * Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening. Exclusion Criteria: * Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study. * Has clinically significant abnormal laboratory results. * Has significant cardiac disease. * Received an investigational drug within 3 months of screening.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 32, 'type': 'ACTUAL'}}

Updated at

2024-02-29

1 organization

2 products

1 indication

Product

Indication

Organization

Product