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Chaperone Therapy

Migalastat for Fabry Disease

Phase 3
Waitlist Available
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
Be younger than 18 years old
Must not have
History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline over time; up to 5 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a drug for Fabry disease, a rare genetic disorder. The drug is given to patients who are over 12 years old and have a certain type of mutation. The trial will measure how well the drug works and how safe it is.

Who is the study for?
This trial is for children over 12 years old with Fabry Disease who finished Study AT1001-020. They need consent from a parent or guardian and must not have severe kidney disease, be pregnant, breastfeeding, or have allergies to the study medication. They can't join if they've had gene therapy or certain treatments for Fabry Disease recently.
What is being tested?
The trial tests Migalastat HCl 150 mg in pediatric patients with Fabry Disease and specific GLA variants. It's an open-label study, meaning everyone knows what treatment is being given, focusing on its safety, how it works in the body (pharmacodynamics), and effectiveness.
What are the potential side effects?
While specific side effects are not listed here, common ones may include allergic reactions to the medication components or other iminosugars like miglustat. Participants will be monitored for any adverse effects throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am over 12 and have completed Study AT1001-020 for Fabry disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I am allergic to the study medication or similar drugs.
Select...
My kidney function is reduced.
Select...
I have never had gene therapy and don't plan to start it during the study.
Select...
I need to take Glyset or Zavesca for my condition.
Select...
I need dialysis or a kidney transplant due to advanced kidney disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline over time; up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline over time; up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
change in Tanner stage
changes in Electrocardiogram (ECG) results
incidence of changes in echocardiogram results
Secondary study objectives
change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores
change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores

Side effects data

From 2019 Phase 3 trial • 84 Patients • NCT02194985
20%
Nasopharyngitis
19%
Fatigue
19%
Arthralgia
17%
Pain in extremity
15%
Paraesthesia
14%
Oedema peripheral
14%
Proteinuria
14%
Overdose
14%
Urinary tract infection
14%
Headache
14%
Upper respiratory tract infection
12%
Nausea
12%
Influenza
12%
Musculoskeletal pain
12%
Dizziness
12%
Hypoaesthesia
11%
Diarrhoea
11%
Sinusitis
11%
Back pain
11%
Pyrexia
10%
Depression
8%
Muscle spasms
8%
Abdominal pain
8%
Vertigo
8%
Tinnitus
8%
Tendonitis
8%
Oropharyngeal pain
8%
Muscular weakness
7%
Bronchitis
7%
Constipation
7%
Insomnia
7%
Albumin urine present
7%
Palpitations
7%
Abdominal pain upper
7%
Blood uric acid increased
7%
Protein urine present
7%
Asthma
7%
Cough
7%
Dyspnoea
7%
Dyspnoea exertional
6%
Hypothyroidism
6%
Gastrooesophageal reflux disease
6%
Dyspepsia
6%
Vomiting
6%
Pain
6%
Migraine
6%
Neuralgia
6%
Blood creatinine increased
6%
Glomerular filtration rate decreased
6%
Hypercholesterolaemia
6%
Hypotension
2%
Atrial fibrillation
2%
Dehydration
2%
Breast cancer
1%
Ventricular tachycardia
1%
Abdominal wall haematoma
1%
Visual impairment
1%
Left ventricular hypertrophy
1%
Renal failure
1%
Barrett's oesophagus
1%
Atrioventricular block complete
1%
Embolic stroke
1%
Device malfunction
1%
Uterine prolapse
1%
Large intestine perforation
1%
Invasive lobular breast carcinoma
1%
Biliary dyskinesia
1%
Drug hypersensitivity
1%
Appendicitis perforated
1%
Biliary sepsis
1%
Diverticulitis
1%
Infective exacerbation of bronchiectasis
1%
Pneumonia
1%
Rib fracture
1%
Heart rate increased
1%
Diabetes mellitus
1%
Lipomatosis
1%
Arthritis
1%
Osteoarthritis
1%
Periarthritis
1%
Tendon calcification
1%
Convulsion
1%
Syncope
1%
Transient ischaemic attack
1%
Suicidal ideation
1%
Pneumothorax
1%
Subcutaneous emphysema
1%
Air embolism
1%
Hypertension
100%
80%
60%
40%
20%
0%
Study treatment Arm
Migalastat HCl 150 mg

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: migalastat HCl 150 mgExperimental Treatment1 Intervention
One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
migalastat HCl 150 mg
2015
Completed Phase 3
~90

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,712 Total Patients Enrolled
25 Trials studying Fabry Disease
1,067 Patients Enrolled for Fabry Disease

Media Library

Migalastat HCl (Chaperone Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04049760 — Phase 3
Fabry Disease Research Study Groups: migalastat HCl 150 mg
Fabry Disease Clinical Trial 2023: Migalastat HCl Highlights & Side Effects. Trial Name: NCT04049760 — Phase 3
Migalastat HCl (Chaperone Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04049760 — Phase 3
~2 spots leftby Dec 2025
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