Clinical trials: Anderson Fabry Disease
Fabry Disease
Migalastat Food Effect Study
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects.
- Trial name
- NCT01489995
- Testing
- A (migalastat), B (migalastat), C (migalastat)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Fabry Disease
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years.
- Trial name
- NCT02921620
- Testing
- PRX-102
- Sponsor
- Protalix
Fabry Disease
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks.
- Trial name
- NCT04974749
- Testing
- REPLAGAL
- Where
- 6 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Fabry Disease
Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion.
- Trial name
- NCT02995993
- Testing
- Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
- Where
- 2 sites in Germany
- Volunteers
- 6 people
- Sponsor
- Greenovation Biotech GmbH
Metabolic Disease, Fabry Disease
Getting Global Rare Disease Insights Through Technology Study
This project is a randomized controlled trial to use a mobile health journal, called Zamplo (formerly known as MyHealthJournal or ZoeInsights), to record patient reported outcomes (PROM) in patients with metabolic disorders.
- Conditions
- Metabolic Disease, Fabry Disease, Gaucher Disease, Pompe Disease
- Trial name
- GRIT (NCT04758130)
- Testing
- Zamplo Digital Health Platform
- Where
- Canada
- Volunteers
- 150 people
- Sponsor
- M.A.G.I.C. Clinic LTD
Fabry Disease
PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
Pharmacokinetic/Pharmacodynamic Study of 2 agalsidase beta Formulations in Single Dose Administered to Healthy Volunteers as intravenous infusion, at a concentration of 1 mg/kg
- Trial name
- NCT05343715
- Testing
- Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg
- Where
- Argentina
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Bio Sidus SA
Fabry Disease
Early Detection of Fabry Disease
The main aim of this study is early detection of FD using real-world data for the development of advanced natural language processing methods and to develop a predictive algorithm and to measure the performance of the algorithm…
- Trial name
- NCT05106764
- Testing
- No intervention
- Where
- 3 sites in Germany
- Volunteers
- 50 people
- Sponsor
- Takeda
Multiple Sclerosis
Prevalence of Fabry Disease in a Defined Population at Risk - Patients Formerly Diagnosed With Multiple Sclerosis
The association of Multiple Sclerosis (MS) and Fabry disease is known from own clinical experiences as well as from case reports in the literature, where symptoms and suspicious results in the brain MRI led to the misdiagnosis of…
- Trial name
- NCT01271699
- Volunteers
- 250 people
- Sponsor
- CENTOGENE GmbH Rostock
Fabry Disease
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and…
- Trial name
- NCT04281537
- Testing
- Enzyme Replacement Therapy (ERT)
- Where
- 9 sites in United States, Brazil and 3 more
- Volunteers
- 82 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2…
- Trial name
- NCT01650779
- Testing
- Agalsidase beta
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201…
- Trial name
- NCT04999059
- Testing
- Safety and Efficacy Assessments
- Where
- 3 sites in Australia, Brazil
- Volunteers
- 5 people
- Sponsor
- AVROBIO
Fabry Disease, Fabry Disease, Cardiac Variant
Long-Term Follow-up of Subjects Who Were Treated With ST-920
Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol.
- Trial name
- NCT05039866
- Testing
- ST-920
- Where
- 13 sites in United States, Australia and 3 more
- Volunteers
- 32 people
- Sponsor
- Sangamo Therapeutics
Fabry Disease
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of…
- Trial name
- NCT04840667
- Testing
- REPLAGAL
- Where
- 28 sites in Canada, Finland and 6 more
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female…
- Trial name
- NCT06052800
- Where
- 5 sites in Taiwan
- Volunteers
- 78 people
- Sponsor
- Sanofi
Fabry Disease
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires…
- Trial name
- NCT04552691
- Testing
- Pegunigalsidase Alfa
- Where
- 11 sites in United States
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease
Fabry Disease (FD) is a rare X-linked lysosomal storage disorder (LSD) caused by mutations in the GLA gene which translates into decreased activity or lack of function of the enzyme alpha-galactosidase A (α-GAL A) and…
- Trial name
- NCT03228940
- Testing
- RVX000222
- Where
- Canada
- Sponsor
- Resverlogix Corp
Fabry Disease
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A.
- Trial name
- NCT04046224
- Testing
- ST-920
- Where
- 18 sites in United States, Australia and 5 more
- Volunteers
- 36 people
- Sponsor
- Sangamo Therapeutics
Fabry Disease
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
This is a Phase II, open-label study designed to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of AL01211 in male subjects with classic Fabry disease who have never received any treatment (eg.
- Trial name
- NCT06114329
- Testing
- AL01211
- Where
- 6 sites in China
- Volunteers
- 16 people
- Sponsor
- AceLink Therapeutics, Inc.
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00304512
- Testing
- migalastat HCl
- Where
- 6 sites in United States, Australia and 4 more
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire