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Gene Therapy

HMI-102 for Pyridoxine-Dependent Epilepsy (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

pheNIX Trial Summary

This trial is testing a new drug for PKU patients with PAH deficiency. The safety and effectiveness of the drug will be followed for one year.

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

pheNIX Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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 outcome measures
Phenylketonuria Quality of Life Questionnaire (PKU-QOL)

pheNIX 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

Frequently Asked Questions

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

What is the aggregate amount of individuals participating in this medical trial?

"Affirmative. Clinicaltrials.gov's records demonstrate that this clinical study, first published on June 10th 2019, is actively seeking participants. 28 individuals need to be recruited from 13 distinct medical locations."

Answered by AI

Is this trial currently welcoming new participants?

"Affirmative. The clinicaltrials.gov registry states that this experiment is currently recruiting volunteers, with the initial posting dating back to June 10th 2019 and most recent update being October 8th 2021. Across 13 centres, 28 patients need to be recruited for the trial."

Answered by AI

What qualifications must a prospective participant possess to be considered for enrollment in this medical research?

"This medical trial necessitates 28 phenylketonuria patients aged between 18 and 55. Additionally, applicants must be able to stay consistent with their diet regimen for the duration of the experiment (unless otherwise directed)."

Answered by AI

Are individuals over the age of 70 being invited to join this research project?

"According to the qualifications listed, this clinical trial is open for individuals aged 18-55. Separately, there are 14 opportunities specifically tailored towards minors and 12 that cater towards senior citizens."

Answered by AI

What goals are the researchers hoping to accomplish with this experiment?

"This clinical trial aims to measure the emergence of treatment-related adverse events between Week 24 and 28 as its primary outcome (Dose Escalation Phase). Secondary objectives include assessing incidences of plasma Phe concentration thresholds up to Week 28 post administration of HMI-102, recording changes in total protein intake at week 52 post administration of HMI-102, and tracking patients who reach plasma Phe concentrations at Week 52."

Answered by AI

Are there numerous healthcare facilities conducting this experiment in the metropolitan area?

"The current enrolment of this clinical trial is taking place in 13 different cities, such as Chapel Hill, Philadelphia and Salt Lake City. To reduce travel costs and time, it may be beneficial to select the nearest site if you choose to participate."

Answered by AI

Who else is applying?

What state do they live in?
Michigan
How old are they?
< 18
What site did they apply to?
Lurie Children's Hospital of Chicago
What portion of applicants met pre-screening criteria?
Met criteria
~2 spots leftby Mar 2025