Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

NCT ID: NCT05285540

Last Updated: 2023-03-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-11

Study Completion Date

2022-12-19

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Friedreich Ataxia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Single Dose: DT-216

DT-216 will be administered once

Group Type EXPERIMENTAL

DT-216

Intervention Type DRUG

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

Single Dose: DT-216 matching placebo

Placebo will be administered once

Group Type EXPERIMENTAL

DT-216 matching Placebo

Intervention Type DRUG

Placebo will be administered by intravenous (IV) injection

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

DT-216

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

Intervention Type DRUG

DT-216 matching Placebo

Placebo will be administered by intravenous (IV) injection

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions
* Body mass index (BMI) between 17 and 32 kg/m2
* Stage 5.5 or less on the Functional Staging for Ataxia (FSA)

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Design Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Clinilabs

Eatontown, New Jersey, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

DTX-216-101

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Idebenone to Treat Friedreich's Ataxia
NCT00229632 COMPLETED PHASE2
FHND1002 for ALS Treatment: Phase 2
NCT07138014 NOT_YET_RECRUITING PHASE2
Methylprednisolone Treatment of Friedreich Ataxia
NCT02424435 COMPLETED EARLY_PHASE1