Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06772870




Registration number
NCT06772870
Ethics application status
Date submitted
6/01/2025
Date registered
14/01/2025
Date last updated
14/01/2025

Titles & IDs
Public title
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
Scientific title
A Phase 1, Double-Masked, Randomized, Placebo-Controlled, Single Ascending Dose Crossover Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Normal Healthy Participants
Secondary ID [1] 0 0
DTX-216P2-011
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - DT-216P2
Treatment: Drugs - Saline

Experimental: DT-216P2 -

Placebo comparator: Placebo -


Treatment: Drugs: DT-216P2
DT-216P2 will be administered as subcutaneous injection, subcutaneous infusion and intravenous infusion.

Treatment: Drugs: Saline
Normal saline solution will be used as placebo control.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of treatment emergent adverse events (TEAEs)
Timepoint [1] 0 0
From first dose to end of study, Day 30 post first dose administration.
Secondary outcome [1] 0 0
Maximum Plasma Concentration (Cmax) of DT-216P2
Timepoint [1] 0 0
Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Secondary outcome [2] 0 0
Time to Maximum Plasma Concentration (Tmax) of DT-216P2
Timepoint [2] 0 0
Pre-dose and up to 240 hours post first dose for both SC and IV infusion.
Secondary outcome [3] 0 0
Area Under the Concentration-time Curve (AUC) of DT-216P2
Timepoint [3] 0 0
Pre-dose and up to 240 hours post first dose for both SC and IV infusion.

Eligibility
Key inclusion criteria
* Participants must be 18-45 years of age inclusive, at the time of signing the informed consent.
* Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and ECG.
* Agree to abstain from strenuous physical activity outside of daily norm, until end of study.
* Body mass index between 16 and 32 kg/m2 (inclusive) at screening; weight should be <= 100 kg at screening.
* Male and/or female using protocol defined and regulatory approved contraception.
* Capable of giving signed informed consent.
Minimum age
18 Years
Maximum age
45 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
* Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol.
* Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator.
* Is not willing to comply with the contraceptive requirements during the study period, as per protocol.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/01/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2025
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network, Level 5, 89 Commercial Road - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Design Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants. Approximately 36 participants will be enrolled into this study.
Trial website
https://clinicaltrials.gov/study/NCT06772870
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ofer Gronen, MD
Address 0 0
Nucleus Network
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Uyen Nguyen, BS
Address 0 0
Country 0 0
Phone 0 0
+1 858-293-4902
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06772870