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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up every year; up to 5 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ every year; up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~every year; up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and every year; up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

change in Tanner stage

change in body weight in kilograms

change in height in centimeters

+4 more

Secondary outcome measures

change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores

change in Left Ventricular Mass Index (LVMi)

change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores

+4 more

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%

Urinary tract infection

14%

Proteinuria

14%

Overdose

14%

Headache

14%

Upper respiratory tract infection

12%

Nausea

12%

Musculoskeletal pain

12%

Influenza

12%

Dizziness

12%

Hypoaesthesia

11%

Back pain

11%

Sinusitis

11%

Pyrexia

11%

Diarrhoea

10%

Depression

Vertigo

Tinnitus

Abdominal pain

Muscle spasms

Tendonitis

Oropharyngeal pain

Muscular weakness

Bronchitis

Albumin urine present

Abdominal pain upper

Insomnia

Palpitations

Constipation

Blood uric acid increased

Protein urine present

Asthma

Cough

Dyspnoea

Dyspnoea exertional

Pain

Vomiting

Gastrooesophageal reflux disease

Dyspepsia

Hypothyroidism

Migraine

Neuralgia

Blood creatinine increased

Glomerular filtration rate decreased

Hypercholesterolaemia

Hypotension

Atrial fibrillation

Dehydration

Breast cancer

Left ventricular hypertrophy

Renal failure

Ventricular tachycardia

Barrett's oesophagus

Uterine prolapse

Invasive lobular breast carcinoma

Abdominal wall haematoma

Large intestine perforation

Visual impairment

Device malfunction

Embolic stroke

Atrioventricular block complete

Biliary dyskinesia

Drug hypersensitivity

Appendicitis perforated

Biliary sepsis

Diverticulitis

Infective exacerbation of bronchiectasis

Pneumonia

Rib fracture

Heart rate increased

Diabetes mellitus

Lipomatosis

Arthritis

Osteoarthritis

Periarthritis

Tendon calcification

Convulsion

Syncope

Transient ischaemic attack

Suicidal ideation

Pneumothorax

Subcutaneous emphysema

Air embolism

Hypertension

100%

80%

60%

40%

20%

Study treatment Arm

Migalastat HCl 150 mg

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

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor

54 Previous Clinical Trials

2,709 Total Patients Enrolled

25 Trials studying Fabry Disease

1,061 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are elderly citizens welcome to participate in this experiment?

"To be eligible for this particular clinical trial, patients must fall between the ages of 12 and 17. There are a total of 6 similar studies available for children and another 19 trials open to seniors."

Answered by AI

Does this research represent a new direction in medicine?

"Since 2019, Amicus Therapeutics has been researching migalastat HCl 150 mg. They sponsored the first clinical trial in 2019 which only involved 15 patients. After this initial study, Phase 3 drug approval was given to migalastat HCl 150 mg in 2019. As of now, there are 2 active studies being conducted for migalastat HCl 150 mg across 11 cities and 8 countries."

Answered by AI

Could you please tell me how many different hospitals are running this research?

"There are several locations where patients can enroll in this study, which include but are not limited to: Cincinnati Children's Hospital (Cincinnati, OH), University of South Florida (Tampa, FL), and the Lysosomal & Rare Disorders Research & Treatment Center (Fairfax, VA)."

Answered by AI

Could you please tell me what other researchers have looked into with migalastat HCl 150 mg?

"Right now, there are two concurrent clinical trials researching migalastat HCl 150 mg. These investigations are both in Phase 3 and based out of Minneapolis, Minnesota; though, 23 different locations have active trials for migalastat HCl 150 mg."

Answered by AI

Does migalastat HCl 150 mg have any dangerous side effects?

"Migalastat HCl 150 mg is considered to be a safe medication, with a safety score of 3. This is due in part because this drug has undergone Phase 3 clinical trials, which provides data affirming both its efficacy and safety."

Answered by AI

How many individuals are enrolled in this clinical trial?

"At this moment, this particular clinical trial is not searching for new patients. However, if you are interested in other medical studies, there are 23 trials actively recruiting patients with fabry disease and 2 more that require participants to take migalastat HCl 150 mg."

Answered by AI

If I qualify, who can join this experiment?

"This particular clinical trial is searching for 15 individuals, all of whom must be between 12 and 17 years old. Furthermore, these patients must have a pre-existing diagnosis of Fabry disease and meet the following additional requirements: Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020."

Answered by AI

Are there any patients who can still join this clinical trial?

"The clinicaltrial.gov website indicates that this study is not presently recruiting patients for participation. The trial was inaugurated on October 14th, 2019 but the most recent edit occurred on July 18th, 2022. Although this particular study isn't looking for participants at the moment, there are 25 other trials which are actively recruiting individuals."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

2019. "Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04049760.

All > Fabry Disease