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Gene Therapy

Gene Therapy for Phenylketonuria

Phase 1 & 2
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
Must not have
Prior treatment with gene therapy
Clinically significant liver dysfunction or disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug, BMN 307, to see if it is safe and effective in treating adults with PKU who have PAH deficiency.

Who is the study for?
This trial is for adults with Phenylketonuria (PKU), a condition where the body can't break down an amino acid called phenylalanine due to PAH deficiency. Participants must have high levels of phenylalanine in their blood and be willing to maintain their current diet, avoid liver-damaging substances, use contraception, and follow study rules.
What is being tested?
The trial tests BMN 307, a gene therapy intended to correct the genetic defect causing PKU. It's given once to see if it's safe and effective at lowering phenylalanine levels. The study gradually increases doses among participants who are closely monitored over time.
What are the potential side effects?
Potential side effects aren't specified but may include reactions related to gene therapy such as immune responses, discomfort at injection site or general symptoms like fever or fatigue. Long-term safety monitoring is part of this early-phase trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with PKU, a condition where my body can't process a certain protein.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have previously undergone gene therapy.
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I have a serious liver condition.
Select...
I have a BH4 deficiency.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Dose 3 of BMN 307Experimental Treatment1 Intervention
Group II: Dose 2 of BMN 307Experimental Treatment1 Intervention
Group III: Dose 1 of BMN 307Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
159 Previous Clinical Trials
114,974 Total Patients Enrolled
Medical Director, MDStudy DirectorBioMarin Pharmaceutical
80 Previous Clinical Trials
16,416 Total Patients Enrolled

Media Library

BMN 307 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04480567 — Phase 1 & 2
PKU Research Study Groups: Dose 2 of BMN 307, Dose 3 of BMN 307, Dose 1 of BMN 307
PKU Clinical Trial 2023: BMN 307 Highlights & Side Effects. Trial Name: NCT04480567 — Phase 1 & 2
BMN 307 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04480567 — Phase 1 & 2
~42 spots leftby Dec 2027
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