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

Friedreich Ataxia Clinical Trial

Official title:

A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05285540
• Other study ID #: DTX-216-101
• Status: Completed
• Phase: Phase 1
• Start date: March 11, 2022
• Est. completion date: December 19, 2022

Study information

• Verified date: March 2023
• Source: Design Therapeutics
• 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 intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 39
• Est. completion date: December 19, 2022
• Est. primary completion date: December 19, 2022
• 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

• 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

Intervention

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

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Frataxin expression	Frataxin mRNA and protein expression measured in peripheral blood monocytes	Up to approximately 30 days
Primary	Frequency of treatment adverse events (TEAEs)	Frequency of treatment emergent adverse events (TEAEs)	Up to approximately 30 days
Secondary	Maximum Plasma Concentration (Cmax) of DT-216	Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 30 days
Secondary	Time to Maximum Plasma Concentration (Tmax) of DT-216	Time to Maximum Plasma Concentration (Tmax) of DT-216	Up to approximately 30 days
Secondary	Area Under the Concentration-time Curve (AUC) of DT-216	Area Under the Concentration-time Curve (AUC) of DT-216	Up to approximately 30 days