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

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

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05285540

Enrollment

Registered

2022-03-17

Start date

2022-03-11

Completion date

2022-12-19

Last updated

2023-03-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Friedreich Ataxia

Brief 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.

Interventions

DRUGDT-216

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

DRUGDT-216 matching Placebo

Placebo will be administered by intravenous (IV) injection

Study design

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 55 Years

Healthy volunteers

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/

Design outcomes

Primary

Measure	Time frame	Description
Frequency of treatment adverse events (TEAEs)	Up to approximately 30 days	Frequency of treatment emergent adverse events (TEAEs)

Secondary

Measure	Time frame	Description
Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 30 days	Maximum Plasma Concentration (Cmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216	Up to approximately 30 days	Time to Maximum Plasma Concentration (Tmax) of DT-216
Area Under the Concentration-time Curve (AUC) of DT-216	Up to approximately 30 days	Area Under the Concentration-time Curve (AUC) of DT-216

Other

Measure	Time frame	Description
Frataxin expression	Up to approximately 30 days	Frataxin mRNA and protein expression measured in peripheral blood monocytes

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026