ASPIRE Pediatric Fabry Study

Phase 1

• Conditions: Fabry disease and with amenable GLA variants; MedDRA version: 20.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-000146-21-ES
• Lead Sponsor: Amicus Therapeutics, UK Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-04-11
• Study Completion: 2021-02-02
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1.have a parent or legally-authorized representative who is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
2.male or female, diagnosed with Fabry disease aged between 12 and < 18 years at baseline, and who might benefit from specific treatment for their condition, in the opinion of the investigator
3.confirmed amenable GLA variant determined using the migalastat amenability assay
4.weight of = 45 kg (99 pounds) at screening
5.treatment-naïve or discontinued ERT treatment at least 14 days prior to screening
6.have at least one complication (ie, historical or current laboratory abnormality and/or sign/symptom) of Fabry disease
7.able to swallow capsules
8.if of reproductive potential, agree to use medically accepted methods of contraception throughout the duration of the study and for up to 30 days after last dose of study medication
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at screening)
2.advanced kidney disease requiring dialysis or kidney transplantation
3.history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
4.subject has received any gene therapy at any time or anticipates starting gene therapy during the study period
5.requires treatment with Glyset® (miglitol) or Zavesca® (miglustat), within 6 months before screening or throughout the study
6.requires treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta) within 14 days before screening or throughout the study
7.received any investigational/experimental drug, biologic or device within 30 days before screening
8.any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
9.is pregnant or breast-feeding, or is planning to become pregnant during the study period
10.in the opinion of the investigator, the subject and/or parent or legally authorized representative is unlikely or unable to comply with the study requirements

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified