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Clinical Trials in the USA / NCT04348708
Enrolling by invitation Observational

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

NCT04348708 · tracked via the Priya Life Science USA tracker
Sponsor
Homology Medicines, Inc
Phase
Observational
Started
2020-08-19
Last updated
2022-06-30

Condition(s) studied

PhenylketonuriaPAH Deficiency

Investigational drug(s) / intervention(s)

HMI-102

HMI-102: HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Study summary

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102

Eligibility

Sex
ALL
Min age
18 Years
Max age
55 Years
Healthy volunteers
No
Inclusion Criteria: * Subject was previously administered HMI 102. * Subject is able to understand the purpose and risks of the study and is willing to provide informed consent. * Subject is able to comply with all study procedures and long-term follow-up. Exclusion Criteria: * Participation in the study is not in the subject's best interest, in the opinion of the Investigator.

Primary outcome measure(s)

  • Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102 — Baseline to year 5
    Subjects with at least one AESI or SAE

Trial sites (2)

FacilityCityRegionStatus
Boston Children's Hospital Boston Massachusetts
Icahn School of Medicine at Mount Sinai New York New York

Other trials for the same condition

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria Recruiting
A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria Active, not recruiting
A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function Recruiting
Immune Modulation During Palynziq® Treatment in Adults (IMPALA) Recruiting
A Study of JNT-517 in Participants With Phenylketonuria (PKU) Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04348708 on ClinicalTrials.gov ↗ ← All trials in the USA