NCT05222178 is a Phase 1 clinical trial of HMI-103 in Phenylketonurias, sponsored by Homology Medicines, Inc.

Who is sponsoring NCT05222178?

NCT05222178 is sponsored by Homology Medicines, Inc.

What drugs are being tested in NCT05222178?

Interventions in NCT05222178: HMI-103.

What condition does NCT05222178 study?

NCT05222178 studies Phenylketonurias, PAH Deficiency, Phenylketonuria.

Is NCT05222178 still recruiting?

NCT05222178 is currently terminated. Last updated 10 October 2023.

Last reviewed 2023-10-10 · How we verify

NCT05222178

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Terminated Phase 1 Last updated 10 October 2023

What this trial tests

Phase 1 trial testing HMI-103 in Phenylketonurias in 3 participants. Terminated before completion.

Timeline

3 June 2022

Primary endpoint
14 September 2023

14 September 2023

Quick facts

Lead sponsor	Homology Medicines, Inc
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	3
Start date	3 June 2022
Primary completion	14 September 2023
Estimated completion	14 September 2023
Sites	2 locations across United States

Drugs / interventions tested

HMI-103 — full drug profile →

Conditions studied

Phenylketonurias — all drugs for Phenylketonurias →
PAH Deficiency — all drugs for PAH Deficiency →
Phenylketonuria — all drugs for Phenylketonuria →

Sponsor

Homology Medicines, Inc — full company profile →

Who can join

Adults 18 to 55, any sex, with Phenylketonurias or PAH Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

State-of-the-art 2023 on gene therapy for phenylketonuria.
Martinez M, Harding CO, Schwank G, Thöny B. · · 2024 · cited 22× · PMID 37401651 · DOI 10.1002/jimd.12651
Gene editing for dyslipidemias: New tools to "cut" lipids.
Stankov S, Cuchel M. · · 2023 · cited 22× · PMID 36725417 · DOI 10.1016/j.atherosclerosis.2023.01.010
Clinical, genetic, and experimental research of hyperphenylalaninemia.
Chen A, Pan Y, Chen J. · · 2022 · cited 12× · PMID 36685931 · DOI 10.3389/fgene.2022.1051153
Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders.
Ghasemzad M, Hashemi M, Lavasani ZM, Hossein-Khannazer N, et al · · 2022 · cited 10× · PMID 36004917 · DOI 10.3390/bioengineering9080392
Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy <i>Arsa</i> Knock-Out Mice.
St Martin T, Seabrook TA, Gall K, Newman J, et al · · 2023 · cited 6× · PMID 36977578 · DOI 10.1523/jneurosci.1829-22.2023
Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.
Raigani M, Eftekhari Z, Adeli A, Kazemi-Lomedasht F. · · 2025 · cited 5× · PMID 40896588 · DOI 10.1016/j.omtn.2025.102666
Progress in Gene Therapy for Hereditary Tyrosinemia Type 1.
Thomas H, Carlisle RC. · · 2025 · cited 1× · PMID 40143050 · DOI 10.3390/pharmaceutics17030387

Verify or expand the search:

Other recruiting trials for Phenylketonurias

Currently open trials in the same condition.

NCT06332807 — AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) · Phase 1, PHASE2 · recruiting
NCT06560736 — Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria · NA · recruiting
NCT06718842 — Walking Program in Fatty Liver Children With Phenylketonuria · NA · recruiting
NCT06687733 — Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects · Phase 1, PHASE2 · recruiting
NCT06061614 — Safety and Efficacy Study of NGGT002 in PKU Adult Subjects · EARLY_PHASE1 · recruiting

Other Homology Medicines, Inc trials

Trials by the same sponsor.

NCT04768348 — Natural History Clinical Study in Adult PKU · terminated
NCT04348708 — Long-Term Follow Up Study of Subjects Previously Administered HMI 102 · enrolling by invitation
NCT03952156 — Gene Therapy Clinical Study in Adult PKU · Phase 1, PHASE2 · terminated

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05222178 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Homology Medicines, Inc
Last refreshed: 10 October 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05222178.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing