Study to Evaluate Multiple Ascending Dose and Multi-Dose... | NCT05573698

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

NCT05573698

Sponsor

Design Therapeutics, Inc.

Status

Completed

Last Update Posted

Feb 29, 2024Actual

Enrollment

32Actual

Phase

Phase 1

Conditions

Friedreich Ataxia

Interventions

DT-216
Placebo

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2024

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 27, 2022Actual

Primary Completion Date

Aug 25, 2023Actual

Completion Date

Aug 25, 2023Actual

First Submitted Date

Oct 6, 2022

First Submission Date that Met QC Criteria

Oct 6, 2022

First Posted Date

Oct 10, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 27, 2024

Last Update Posted Date

Feb 29, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

32Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
DT-216	Drug	DT-216 will be administered by intravenous (IV) injection	Multiple Dose: DT-216

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety Measure	Frequency of treatment emergent adverse events (TEAEs)	Up to approximately 60 days

Secondary Outcomes

Measure	Description	Time Frame
Pharmacokinetic parameters	Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 60 days
Pharmacokinetic parameters	Time to Maximum Plasma Concentration (Tmax) of DT-216

Other Outcomes

Measure	Description	Time Frame
Pharmacodynamic parameters	Frataxin expression	Up to approximately 60 days

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

55 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Clinilabs		Eatontown	New Jersey	07724	United States

Condition Browse Module

MeSH Terms

ID	Term
D005621	Friedreich Ataxia

Ancestor Terms

ID	Term
D013132	Spinocerebellar Degenerations
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided