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Completed

NCT Number: NCT05285540

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

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.

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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinilabs

Eatontown, New Jersey, 07724, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

DT-216

Drug

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

DT-216 matching Placebo

Drug

Placebo will be administered by intravenous (IV) injection

Primary outcomes

  1. Frequency of treatment adverse events (TEAEs)

    Time frame: Up to approximately 30 days

    Frequency of treatment emergent adverse events (TEAEs)

Secondary outcomes

  1. Maximum Plasma Concentration (Cmax) of DT-216

    Time frame: Up to approximately 30 days

    Maximum Plasma Concentration (Cmax) of DT-216

  2. Time to Maximum Plasma Concentration (Tmax) of DT-216

    Time frame: Up to approximately 30 days

    Time to Maximum Plasma Concentration (Tmax) of DT-216

  3. Area Under the Concentration-time Curve (AUC) of DT-216

    Time frame: Up to approximately 30 days

    Area Under the Concentration-time Curve (AUC) of DT-216

Other outcomes

  1. Frataxin expression

    Time frame: Up to approximately 30 days

    Frataxin mRNA and protein expression measured in peripheral blood monocytes

Sponsors and collaborators

Lead sponsor

Design Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2022
Study completion
2022
First posted
Mar 17, 2022
Registry last updated
Mar 28, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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