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Gene Therapy

Gene Therapy Clinical Study in Adult PKU (pheNIX Trial)

Phase 1 & 2
Waitlist Available
Led By Olaf A Bodamer, M.D.
Research Sponsored by Homology Medicines, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up weeks 24-28
Awards & highlights
No Placebo-Only Group

Summary

This trial tests a new gene therapy called HMI-102 in adults with PKU, a genetic condition. The therapy aims to help their bodies produce an enzyme they are missing. Participants will receive the treatment and be monitored over several months. HMI-102 is designed to treat PKU by delivering the PAH gene to produce the missing enzyme.

Eligible Conditions
  • Pyridoxine-Dependent Epilepsy (PDE)
  • Tetrahydrobiopterin Deficiency
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 24-28
This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 24-28 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in 12-lead electrocardiograms (ECGs), vital signs, physical examinations (Dose Escalation Phase)
Change from baseline in Plasma Phe Concentration (Dose Escalation Phase)
Change from baseline in clinical laboratory values (Dose Escalation Phase)
+3 more
Secondary study objectives
Change from baseline in total protein intake at Week 52 post-administration of HMI-102 (Dose Expansion Phase)
Incidence and severity of treatment-emergent adverse events (TEAEs) (Dose Expansion Phase)
Incidence of plasma Phe concentration thresholds up to Week 28 post administration of HMI-102 (Dose Expansion Phase)
+1 more
Other study objectives
Phenylketonuria Quality of Life Questionnaire (PKU-QOL)

Awards & Highlights

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

Trial Design

6Treatment groups
Experimental Treatment
Group I: Expansion Phase Second Dose levelExperimental Treatment1 Intervention
Expansion Phase Second Dose Level of HMI-102 delivered intravenously one time
Group II: Expansion Phase First Dose levelExperimental Treatment1 Intervention
Expansion Phase First Dose Level of HMI-102 delivered intravenously one time
Group III: Delayed Treatment ControlExperimental Treatment1 Intervention
Delayed Treatment Control Arm
Group IV: Cohort 3Experimental Treatment1 Intervention
Dose Level 3 of HMI-102 delivered intravenously one time
Group V: Cohort 2Experimental Treatment1 Intervention
Dose Level 2 of HMI-102 delivered intravenously one time
Group VI: Cohort 1Experimental Treatment1 Intervention
Dose Level 1 of HMI-102 delivered intravenously one time

Find a Location

Who is running the clinical trial?

Homology Medicines, IncLead Sponsor
5 Previous Clinical Trials
12,031 Total Patients Enrolled
Olaf A Bodamer, M.D.Principal InvestigatorBoston Children's Hospital
~2 spots leftby Dec 2025
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