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

Friedreich Ataxia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05573698
• Other study ID #: DTX-216-102
• Status: Completed
• Phase: Phase 1
• Start date: September 27, 2022
• Est. completion date: August 25, 2023

Study information

• Verified date: February 2024
• Source: Design Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: August 25, 2023
• Est. primary completion date: August 25, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

Intervention

Drug:
• DT-216
DT-216 will be administered by intravenous (IV) injection
• Placebo
Matching Placebo will be administered by intravenous (IV) injection

Locations

Country	Name	City	State
United States	Clinilabs	Eatontown	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Design Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Pharmacodynamic parameters	Frataxin expression	Up to approximately 60 days
Primary	Safety Measure	Frequency of treatment emergent adverse events (TEAEs)	Up to approximately 60 days
Secondary	Pharmacokinetic parameters	Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 60 days
Secondary	Pharmacokinetic parameters	Time to Maximum Plasma Concentration (Tmax) of DT-216	Up to approximately 60 days
Secondary	Pharmacokinetic parameters	Area Under the Concentration-time Curve (AUC) of DT-216	Up to approximately 60 days