Clinical Trials
Our clinical trials units is dedicated to providing access to research therapies for rare metabolic and genetic diseases
Fabry Disease Studies
Peridot
A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults with Fabry Disease who are Treatment-naïve or Untreated for At least 6 Months
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Fabry HCM
Screening for Fabry Cardiomyopathy Using Cardiac Magnetic Resonance Imaging
Migalastat SWITCH
Evaluation of biomarkers for patients receiving Migalastat after switching from ERT
Gaucher, Fabry, MPS I, and Pompe Registries
Rare disease registries for lysosomal storage disorders
CFDI
Canadian Fabry Disease Initiative Registry
GRIT STUDY
M.A.G.I.C. Clinic, the largest metabolic diseases clinic in Alberta, Canada is looking to learn better ways of managing virtual care for rare disease patients worldwide.
Pompe Disease Studies
Gaucher, Fabry, MPS I, and Pompe Registries
Rare disease registries for lysosomal storage disorders
GRIT STUDY
M.A.G.I.C. Clinic, the largest metabolic diseases clinic in Alberta, Canada is looking to learn better ways of managing virtual care for rare disease patients worldwide.
Gaucher Disease Studies
Gaucher, Fabry, MPS I, and Pompe Registries
Rare disease registries for lysosomal storage disorders
GRIT STUDY
M.A.G.I.C. Clinic, the largest metabolic diseases clinic in Alberta, Canada is looking to learn better ways of managing virtual care for rare disease patients worldwide.
AVRO-RD-02-LTF01
A FOLLOW-UP STUDY OF SUBJECTS WITH TYPE 1 GAUCHER DISEASE WHO PREVIOUSLY RECEIVED AVR-RD-02 GENE THERAPY.
More Studies
CMVibe Trial
A Phase 1/2a Open-Label Dose-Ranging and Observer-Blind Placebo-Controlled, Safety and Immunogenicity Study of mRNA-1647 Cytomegalovirus Vaccine in Female and Male Participants 9 to 15 Years of Age and Participants 16 to 25 Years of Age.
PALomino
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
PTC923-MD-003-PKU
A PHASE 3 STUDY OF PTC923 IN SUBJECTS WITH
PHENYLKETONURIA
GRIT STUDY
M.A.G.I.C. Clinic, the largest metabolic diseases clinic in Alberta, Canada is looking to learn better ways of managing virtual care for rare disease patients worldwide.
REN001
A DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY TO EVALUATE THE
EFFICACY AND SAFETY OF 24 WEEKS TREATMENT WITH REN001 IN
PATIENTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)
Gene Therapy Trials
ST-920-201
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease
GTX-102
A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
juMPStart study
A Phase 1 Open-Label Dose Escalation Study to Evaluate the
Safety and Efficacy of HMI-203 in ERT-Treated Adults with
Mucopolysaccharidosis Type II (MPS II)
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FACTS
Phase I Gene Therapy for Fabry Disease
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