Recruiting NCT06874010
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: DT-216P2.
- Who it may be relevant to
- Registry conditions: Friedreich Ataxia. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia
Overview
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
Interventions
- Drug DT-216P2
Active
Primary outcome measures
- Frequency of treatment-emergent adverse events (TEAE) [Time frame: Up to Week 12]
Secondary outcome measures (5)
- Area under the curve (AUClast, AUCinf) [Time frame: Up to Week 12]
- Maximum concentration (Cmax) [Time frame: Up to Week 12]
- Time to Cmax (Tmax) [Time frame: Up to Week 12]
- Half-life (t1/2) [Time frame: Up to Week 12]
- Frataxin expression at baseline and after treatment will be measured [Time frame: Up to Week 12]
Eligibility criteria
Inclusion criteria
- Participants must be 18-65 years of age inclusive, at the time of signing the informed consent.
- Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene.
- Stage 5.5 or less on the FSA at screening.
- BMI between 16 and 32 kg/m2 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.
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.
- Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months.
- Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Australia · 3 centers
- Scientia Clinical Research Ltd — Sydney
- Doherty Clinical Trials — East Melbourne
- Nucleus Network — Melbourne
Identifiers
NCT: NCT06874010 · DTX-216P2-101