Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
UniQure Biopharma B.V.
Study ID
NCT06270316
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
18 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • AMT-191 — DRUG
    A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease.

Study Details

The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.

Key Dates

Start date
Jun 5, 2024
Status verified
Oct 2025
Primary completion
Dec 1, 2027
Completion
Apr 30, 2031

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Dose Ranging Cohort 1
  • Experimental: Dose Ranging Cohort 2
  • Experimental: Dose Ranging Cohort 3

Primary Outcome Measure

Evaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 in Participants with FD [ Time Frame: 60 Months ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
The Kirklin Clinic Of university of Alabama Birmingham HospitalBirminghamAlabama35233
Christina Desruisseau, BSN
[email protected]
205-975-2935
Eric Wallace, MD (PRINCIPAL_INVESTIGATOR)
Emory University School of MedicineAtlantaGeorgia30322
Dawn Laney
[email protected]
404-778-8518
William Wilcox, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Carolyn Rasmussen, MS Genetic Counseling
[email protected]
312.227.6763
Carolyn Raski, MS Genetic Counselor
[email protected]
Carlos E Prada, MD (PRINCIPAL_INVESTIGATOR)
MHealth Fairview University of Minnesota Medical Center East BankMinneapolisMinnesota55455
Melissa Fetterley, BSN
[email protected]
612-672-5151
Brenda Diethelm-Okita, Masters
[email protected]
Chester B Whitley, MD (PRINCIPAL_INVESTIGATOR)
NYC Health + Hospitals/MetropolitanNew YorkNew York10029
Beena Thomas
[email protected]
(843) 297- 5656
Maryam Banikazemi (PRINCIPAL_INVESTIGATOR)
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Jennifer Baker
[email protected]
412-692-6378
Damara Ortiz, MD (PRINCIPAL_INVESTIGATOR)
University of Utah, Clinical and Translational Sciences InstituteSalt Lake CityUtah84108
Carrie Bailey, Bachelor of Science
[email protected]
801-587-3605
Jenny Billy
[email protected]
Brian Shayota, MD, MPH (PRINCIPAL_INVESTIGATOR)
Lysosomal & Rare Disorders Research and Treatment Center, IncFairfaxVirginia22030
Lauren Noll
[email protected]
(571) 732-4655
Ozlem Goker-Alpan (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By research site
The Kirklin Clinic Of university of Alabama Birmingham Hospital· Birmingham, ALEmory University School of Medicine· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILMHealth Fairview University of Minnesota Medical Center East Bank· Minneapolis, MNNYC Health + Hospitals/Metropolitan· New York, NYUPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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