A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Chiesi Farmaceutici S.p.A.
Study ID
NCT06328608
Phase
PHASE2/PHASE3
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • PRX-102 1 mg/kg every two weeks — DRUG
    Drug: PRX-102 1 mg/kg every two weeks

Study Details

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

Key Dates

Start date
Jul 29, 2025
Status verified
Mar 2026
Primary completion
Oct 31, 2028
Completion
Apr 30, 2031

Study Design

Enrollment
22 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Single Arm - Pegunigalsidase alfa (PRX-102)
    For Cohort C, PXR-102 administered every two weeks at 1.0 mg/kg is believed to be the minimum effective dose. For Cohorts A and B, the starting dose will be 1.0 mg/kg every two weeks but it may be adjusted on the outcomes of Stage I, with the support of the Data Safety Monitoring Board.

Primary Outcome Measure

Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 12 Months ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Phoenix Children'sPhoenixArizona85016
Jasmine Knoll
[email protected]
602-933-4363
Emory Genetics Clinical Trials CenterAtlantaGeorgia30322
William Wilcox
[email protected]
404-727-2931
University of IowaIowa CityIowa52242
John Bernat
[email protected]
319-356-2675
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Robert Hopkin
[email protected]
513-636-4760
University of UtahSalt Lake CityUtah84108
Julie M Porter
[email protected]
801-587-3605
Lysosomal and Rare Disorders Research and Treatment Center IncFairfaxVirginia22030
Ozlem Goker-Alpan
[email protected]
240-643-6003

Find similar trials in Phoenix, AZ

By research site
Phoenix Children's· Phoenix, AZEmory Genetics Clinical Trials Center· Atlanta, GAUniversity of Iowa· Iowa City, IACincinnati Children's Hospital Medical Center· Cincinnati, OHUniversity of Utah· Salt Lake City, UTLysosomal and Rare Disorders Research and Treatment Center Inc· Fairfax, VA

Related Studies