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Trial record 1 of 2 for:    hmi102
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Long-Term Follow Up Study of Subjects Previously Administered HMI 102

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04348708
Recruitment Status : Enrolling by invitation
First Posted : April 16, 2020
Last Update Posted : April 16, 2020
Sponsor:
Information provided by (Responsible Party):
Homology Medicines, Inc

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

Condition or disease Intervention/treatment
Phenylketonuria PAH Deficiency Genetic: HMI-102

Detailed Description:
This Long-Term Follow Up Study will evaluate the safety and efficacy of PKU Subjects with PAH Deficiency Previously Administered HMI 102. Subjects will have already received a single dose of HMI-102 administered intravenously

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Study Type : Observational
Estimated Enrollment : 21 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
Estimated Study Start Date : August 30, 2020
Estimated Primary Completion Date : January 30, 2026
Estimated Study Completion Date : December 1, 2026


Group/Cohort Intervention/treatment
Dose Level Cohort 1
Dose Level 1 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Dose Level Cohort 2
Dose Level 2 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Dose Level Cohort 3
Dose Level 3 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene




Primary Outcome Measures :
  1. Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102 [ Time Frame: Baseline to year 5 ]
    Subjects with at least one AESI or SAE


Secondary Outcome Measures :
  1. Plasma Phe Concentration [ Time Frame: Baseline to year 5 ]
    Plasma phenylalanine (Phe) concentration at each time point during the study

  2. Phe-restricted diet [ Time Frame: Baseline to year 5 ]
    Incidence of subjects with a Phe-restricted diet

  3. Phenylketonuria Quality of Life Questionnaire (PKU-QOL [ Time Frame: Baseline to year 5 ]
    Quality of life (QOL), as assessed using the Phenylketonuria-QOL (PKU-QOL) questionnaire

  4. Protein intake [ Time Frame: Baseline to year 5 ]
    Protein intake relative to Phe concentration at each time point during the study


Biospecimen Retention:   Samples Without DNA
Neutralizing AAV antibody, AAV antibody (IgG), Anti-PAH antibody, Vector DNA in blood, Vector DNA in body fluids, Serum Chemistry, Hematology


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects with PAH Deficiency who were previously administered HMI-102
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04348708


Locations
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United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, New York
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Sponsors and Collaborators
Homology Medicines, Inc
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Responsible Party: Homology Medicines, Inc
ClinicalTrials.gov Identifier: NCT04348708    
Other Study ID Numbers: HMI-102-102
First Posted: April 16, 2020    Key Record Dates
Last Update Posted: April 16, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Homology Medicines, Inc:
PKU
Phenylketonuria
PAH Deficiency
Phenylalanine
Adeno Associated Virus
AAVHSC15
Additional relevant MeSH terms:
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Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases