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

NCT05573698 | Completed Phase 1 FDA Drug

• 1 other identifier: DTX-216-102
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• Safety Measure

  Frequency of treatment emergent adverse events (TEAEs)

  Up to approximately 60 days

Secondary Outcomes (3)

• Pharmacokinetic parameters

  Up to approximately 60 days

• Pharmacokinetic parameters

  Up to approximately 60 days

• Pharmacokinetic parameters

  Up to approximately 60 days

Other Outcomes (1)

• Pharmacodynamic parameters

  Up to approximately 60 days

Study Arms (2)

Multiple Dose: DT-216

EXPERIMENTAL

Participants will be administered multiple doses of DT-216

Drug: DT-216

Multiple Dose: DT-216 matching placebo

PLACEBO COMPARATOR

Participants will be administered multiple doses of placebo

Drug: Placebo

Interventions

DT-216 DRUG

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

Multiple Dose: DT-216

Placebo DRUG

Matching Placebo will be administered by intravenous (IV) injection

Multiple Dose: DT-216 matching placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Design Therapeutics, Inc.: lead

Study Sites (1)

Clinilabs

Eatontown, New Jersey, 07724, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2022
• First Posted: October 10, 2022
• Study Start: September 27, 2022
• Primary Completion: August 25, 2023
• Study Completion: August 25, 2023
• Last Updated: February 29, 2024

Record last verified: 2024-02

Locations

US (1)