AAV Gene Therapy Study for Subjects with PKU (NCT04480567) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
AAV Gene Therapy Study for Subjects with PKU
United States, United Kingdom100 participantsStarted 2020-09-24
Plain-language summary
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
Who can participate
Age range
15 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
* Ability and willingness to maintain dietary protein intake consistent with baseline intake
* Willingness to abstain from hepatotoxic substances post-BMN 307 administration
* Willingness and capable per investigator opinion to comply with study procedures and requirements
* Willingness to use effective methods of contraception
* Plasma Phe levels \> 600 µmol/L
Exclusion Criteria:
* Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency
* Clinically significant liver dysfunction or disease
* Prior treatment with gene therapy
* Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
* History of malignancy
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.