Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

NCT ID: NCT05573698

Last Updated: 2024-02-29

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

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

32 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-27

Study Completion Date

2023-08-25

Brief Summary

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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.

Detailed Description

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Conditions

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Friedreich Ataxia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Multiple Dose: DT-216

Participants will be administered multiple doses of DT-216

Group Type EXPERIMENTAL

DT-216

Intervention Type DRUG

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

Multiple Dose: DT-216 matching placebo

Participants will be administered multiple doses of placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching Placebo will be administered by intravenous (IV) injection

Interventions

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DT-216

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

Intervention Type DRUG

Placebo

Matching Placebo will be administered by intravenous (IV) injection

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Design Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinilabs

Eatontown, New Jersey, United States

Site Status

Countries

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United States

Other Identifiers

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DTX-216-102

Identifier Type: -

Identifier Source: org_study_id

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