Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE1/PHASE2
10 participants
INTERVENTIONAL
2019-06-10
2023-08-01
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
NCT05222178
Natural History Clinical Study in Adult PKU
NCT04768348
AAV Gene Therapy Study for Subjects with PKU
NCT04480567
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II
NCT05238324
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
NCT06332807
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
In Part 2 dose expansion, evaluation of up to 2 dose levels is planned. Subjects will be randomized to receive HMI-102 or a concurrent delayed treatment control arm. Subjects in the delayed treatment control will be eligible to receive HMI-102 after 28 weeks.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort 1
Dose Level 1 of HMI-102 delivered intravenously one time
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Cohort 2
Dose Level 2 of HMI-102 delivered intravenously one time
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Cohort 3
Dose Level 3 of HMI-102 delivered intravenously one time
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Delayed Treatment Control
Delayed Treatment Control Arm
HMI-102
Control subjects will generally have the same assessments as treated subjects.
Control subjects will undergo pre-baseline procedures to confirm that they are eligible to receive treatment with HMI-102. Once eligible control subjects are dosed with HMI-102, they will initiate the same post-dose procedures as subjects who received HMI-102.
Expansion Phase First Dose level
Expansion Phase First Dose Level of HMI-102 delivered intravenously one time
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Expansion Phase Second Dose level
Expansion Phase Second Dose Level of HMI-102 delivered intravenously one time
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
HMI-102
Control subjects will generally have the same assessments as treated subjects.
Control subjects will undergo pre-baseline procedures to confirm that they are eligible to receive treatment with HMI-102. Once eligible control subjects are dosed with HMI-102, they will initiate the same post-dose procedures as subjects who received HMI-102.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Diagnosis of phenylketonuria (PKU) due to PAH deficiency
* Two plasma Phe values with a concentration of ≥ 600 μmol/L drawn at least 72 hours apart during the screening period and at least one historical value ≥ 600 μmol/L in the preceding 24 months.
* Subject has the ability and willingness to maintain their baseline diet, whether Phe-restricted or unrestricted for the duration of the trial, unless otherwise directed
Exclusion Criteria
* Presence of anti-AAVHSC15 neutralizing antibodies
* ALT \> ULN and AST \> ULN
* Alkaline phosphatase \> ULN.
* Total bilirubin \> ULN, direct bilirubin \> ULN
* Serum creatinine \>1.5x ULN
* International normalized ratio (INR) \> 1.2
* Hematology values outside of the normal range (hemoglobin \<11.0 g/dL for males or \<10.0 g/dL for females; white blood cells (WBC) \<3,000/μL; absolute neutrophils \<1500/μL; platelets \<100,000/μL)
* Hemoglobin A1c \>6.5% or fasting glucose \>126 mg/dL
* Any clinically significant abnormal laboratory result at screening, in the opinion of the Investigator
* Contraindication to corticosteroid use or conditions that could worsen in the presence of corticosteroids, as assessed and determined by the investigator
* Previously received gene therapy for the treatment of any condition.
18 Years
55 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Homology Medicines, Inc
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Olaf A Bodamer, M.D.
Role: PRINCIPAL_INVESTIGATOR
Boston Children's Hospital
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
University of South Florida
Tampa, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
The University of North Carolina At Chapel Hill
Chapel Hill, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
HMI-102-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.