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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 5 years
Awards & highlights

Study 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.Check my eligibility
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.See study design
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
Select...
I have been diagnosed with PKU, a condition where my body can't process a certain protein.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Secondary outcome measures
Change from baseline in dietary protein intake from intact food
Change from baseline in mean Plasma Phe levels
Number of participants with treatment-emergent adverse event

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
156 Previous Clinical Trials
190,625 Total Patients Enrolled
Medical Director, MDStudy DirectorBioMarin Pharmaceutical
76 Previous Clinical Trials
16,144 Total Patients Enrolled

Media Library

BMN 307 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04480567 — Phase 1 & 2
Phenylketonuria Research Study Groups: Dose 2 of BMN 307, Dose 3 of BMN 307, Dose 1 of BMN 307
Phenylketonuria 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can individuals enroll in this experiment presently?

"Clinicaltrials.gov's listings state that this medical trial, which first opened on September 24th 2020 and last had an update October 6th 2021, is no longer actively recruiting participants. Fortunately however, 19 other research programs are currently in search of volunteers."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
AAV Gene Therapy Study for Subjects With PKU
2020. "AAV Gene Therapy Study for Subjects With PKU". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04480567.
All > Phenylketonuria
~50 spots leftby Dec 2027
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