A Phase 1/2 Open-Label, Randomized, Concurrently-Controlled, Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-102 in Adult PKU Subjects With PAH Deficiency

Who is this study for? Patients with Phenylketonurias, PAH Deficiency
What treatments are being studied? HMI-102
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 55
Healthy Volunteers: No
View:

• Adults 18-55 years of age at the time of informed consent

• 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

Locations
United States
California
Kaiser Permanente Los Angeles Medical Center
Recruiting
Los Angeles
Children's Hospital of Orange County
Recruiting
Orange
Florida
University of South Florida
Recruiting
Tampa
Georgia
Emory University Hospital
Recruiting
Atlanta
Illinois
Lurie Children's Hospital of Chicago
Recruiting
Chicago
Massachusetts
Boston Children's Hospital
Recruiting
Boston
North Carolina
The University of North Carolina At Chapel Hill
Recruiting
Chapel Hill
New York
Icahn School of Medicine at Mount Sinai
Recruiting
New York
Ohio
Nationwide Children's Hospital
Recruiting
Columbus
Pennsylvania
Children's Hospital of Philadelphia
Recruiting
Philadelphia
UPMC Children's Hospital of Pittsburgh
Recruiting
Pittsburgh
Texas
University of Texas Southwestern Medical Center
Recruiting
Dallas
Utah
University of Utah
Recruiting
Salt Lake City
Contact Information
Primary
Julie Jordan, M.D.
clinicaltrials@homologymedicines.com
781-819-0967
Time Frame
Start Date: June 10, 2019
Estimated Completion Date: September 2023
Participants
Target number of participants: 28
Treatments
Experimental: Cohort 1
Dose Level 1 of HMI-102 delivered intravenously one time
Experimental: Cohort 2
Dose Level 2 of HMI-102 delivered intravenously one time
Experimental: Cohort 3
Dose Level 3 of HMI-102 delivered intravenously one time
Experimental: Delayed Treatment Control
Delayed Treatment Control Arm
Experimental: Expansion Phase First Dose level
Expansion Phase First Dose Level of HMI-102 delivered intravenously one time
Experimental: Expansion Phase Second Dose level
Expansion Phase Second Dose Level of HMI-102 delivered intravenously one time
Authors
Olaf A Bodamer
Sponsors
Leads: Homology Medicines, Inc

This content was sourced from clinicaltrials.gov

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