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Chaperone Therapy

Migalastat for Fabry Disease

Phase 3

Recruiting

Research Sponsored by Amicus Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has a GLA variant that is amenable to migalastat recorded in their medical records

Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session

Must not have

Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)

Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through month 12

Awards & highlights

No Placebo-Only Group

Pivotal Trial

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This trial will test the safety and how well the drug migalastat HCl works in people with Fabry disease and severe kidney impairment.

Who is the study for?

This trial is for adults with Fabry disease and severe kidney impairment, including those on stable hemodialysis. Participants must have a specific GLA gene variant treatable by migalastat and agree to use contraception if of reproductive potential. Exclusions include pregnancy, breastfeeding, unstable heart conditions, recent other investigational drugs or gene therapy, allergy to migalastat or similar drugs.

What is being tested?

The study tests the safety and how the body processes Migalastat HCl in individuals with Fabry disease who also have severe renal impairment (SRI) or are at the end stage of renal disease (ESRD). It's an open-label study where all participants know they're receiving Migalastat.

What are the potential side effects?

While not specified here, common side effects of Migalastat may include headache, nasal and throat irritation, stomach pain, fever; serious side effects could involve allergic reactions or worsening kidney function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My records show a GLA variant treatable with migalastat.

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I have end-stage renal disease and will complete all my dialysis sessions as prescribed.

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I am 18 or older and have been diagnosed with Fabry disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am allergic to migalastat or similar medications.

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I am currently taking or have taken Zavesca® (miglustat).

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I have never had gene therapy and don't plan to during the study.

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I have had a stroke, heart attack, or severe chest pain.

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I have a serious heart condition that is not stable.

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I am currently undergoing peritoneal dialysis.

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I need treatment with Glyset, Replagal, or Fabrazyme.

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I have had a kidney transplant.

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I am currently pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through month 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Amount recovered in dialysate (AeD)

Apparent plasma clearance (CL/F)

Apparent terminal elimination half-life (t½)

+19 more

Secondary study objectives

Adverse events (AEs)

Change from baseline eGFR based on the Chronic Kidney Disease Epidemiology Collaboration equation (eGFRCKD-EPI)

Change from baseline in estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease equation (eGFR MDRD)

+5 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%

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

Muscle spasms

Abdominal pain

Vertigo

Tinnitus

Tendonitis

Oropharyngeal pain

Muscular weakness

Bronchitis

Constipation

Insomnia

Albumin urine present

Palpitations

Abdominal pain upper

Blood uric acid increased

Protein urine present

Asthma

Cough

Dyspnoea

Dyspnoea exertional

Hypothyroidism

Gastrooesophageal reflux disease

Dyspepsia

Vomiting

Pain

Migraine

Neuralgia

Blood creatinine increased

Glomerular filtration rate decreased

Hypercholesterolaemia

Hypotension

Atrial fibrillation

Dehydration

Breast cancer

Ventricular tachycardia

Abdominal wall haematoma

Visual impairment

Left ventricular hypertrophy

Renal failure

Barrett's oesophagus

Atrioventricular block complete

Embolic stroke

Device malfunction

Uterine prolapse

Large intestine perforation

Invasive lobular breast carcinoma

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

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.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2: End-Stage Renal DiseaseExperimental Treatment1 Intervention

All hemodialysis subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat). Subjects will take 1 migalastat capsule orally with water every other week.

Group II: Cohort 1: Severe Renal ImpairmentExperimental Treatment1 Intervention

All subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat) at a dose regimen based on their eGFRMDRD result at Visit 1. Subjects will take 1 migalastat capsule orally with water either every 4 or 7 days.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Migalastat

FDA approved

0 / 12Eligibility criteria met