migalastat HCl 150 mg for Fabry Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA
Fabry Disease
migalastat HCl 150 mg - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether migalastat, a drug used to treat Fabry disease, may be safe and effective in individuals with Fabry disease and severe kidney disease. original

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Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether migalastat HCl 150 mg will improve 10 primary outcomes and 9 secondary outcomes in patients with Fabry Disease. Measurement will happen over the course of Screening through Week 12.

Hour 24
Change in Amount excreted urine
Change in Fraction of the dose recovered in urine
Baseline through Week 12
Apparent terminal elimination half-life (t½)
Area under the concentration time curve at steady state during the dosing interval (AUC0-τ)
Average plasma migalastat concentration over the dosing interval (Cavg)
Concentration at the end of a dosing interval at steady state (Ctrough)
Maximum observed concentration (Cmax)
Plasma clearance (CL/F)
Time to maximum concentration (tmax)
peak and trough plasma migalastat concentrations
Baseline through Week 14
Number of subjects with abnormal clinical chemistry laboratory test results
Number of subjects with abnormal hematology laboratory test results
Number of subjects with abnormal urinalysis laboratory test results
Screening through Week 12
Number of subjects with abnormal echocardiogram (ECHO) results
change from baseline in plasma globotriaosylsphingosine (lyso-Gb3)
Screening through Week 14
Number of subjects with abnormal electrocardiogram (ECG) findings as a measure of safety and tolerability
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)

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Side Effects for

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

Trial Design

1 Treatment Group

migalastat HCl 150 mg
1 of 1
Experimental Treatment

This trial requires 12 total participants across 1 different treatment group

This trial involves a single treatment. Migalastat HCl 150 Mg is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

migalastat HCl 150 mg
Drug
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

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 0-12 hours and 0-24 hours
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 0-12 hours and 0-24 hours for reporting.

Closest Location

UPMC Children's Hospital of Pittsburgh - Pittsburgh, PA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Male or female subjects aged 16 years or older, diagnosed with Fabry disease.
Subject (or legally authorized representative as applicable) is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information
Subject has a GLA variant that is amenable to migalastat recorded in their medical records
Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2
If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception

Patient Q&A Section

What causes fabry disease?

"Fabry disease is a rare, inherited metabolic disorder caused by a deficiency in a protein that helps remove substances from the body. It affects many parts of the body and can be life-threatening.\n" - Anonymous Online Contributor

Unverified Answer

How many people get fabry disease a year in the United States?

"About 1 in 100,000 men have Fabry disease and about 1 in 100,000 women have Fabry disease in the United States each year. This prevalence, if corroborated by genetic studies, means that Fabry disease is more common in the United States than the estimates for a country such as Australia or Israel." - Anonymous Online Contributor

Unverified Answer

What are common treatments for fabry disease?

"If you have a problem with a joint, bone, or other part of your body, a doctor can assess you to see if he or she thinks you need to treat it in order to have normal life. There isn't always a solution for your condition, although there is a wide range of drugs and treatments that are prescribed to help with your problems." - Anonymous Online Contributor

Unverified Answer

What is fabry disease?

"Fabry disease most often (but not exclusively) affects males. Fabry disease's hallmark features are hemolysis and renal insufficiency that usually occur within 10 to 20 years of onset. Fabry disease does not always occur in males.\n" - Anonymous Online Contributor

Unverified Answer

What are the signs of fabry disease?

"Fabry disease can present with a wide range of cardiovascular and renal complications in addition to the classic neurologic manifestations of angiopathy, neuropathy, and kidney failure. Recent findings need to be kept in mind during evaluation of patients with clinical and laboratory evidence of renal failure." - Anonymous Online Contributor

Unverified Answer

Can fabry disease be cured?

"There is no cure for Fabry disease, but recent advances in the understanding of the disease, particularly related to vascular biology, have produced new therapeutic tools and promises, and there is hope in the future." - Anonymous Online Contributor

Unverified Answer

How does migalastat hcl 150 mg work?

"Migalastat Hcl may be useful for the treatment of pulmonary arterial hypertension as treatment with Fabrazyme is associated with substantial improvement in pulmonary hemodynamics and clinical symptoms." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating fabry disease?

"There has been very little progress in improving our treatment for Fabry disease. The only known treatment that improves life expectancy is hemodialysis due to its ability to remove excess fatty acids from the blood. Although new medications, such as miglustat, have been developed, there have been no clinical trials to prove the therapeutic benefit it offers. Despite the lack of evidence for the therapeutic effects, many have resorted to its use as an experimental treatment because of its limited availability and its ability to be self-administered by patients. A recent study showed a beneficial effect when miglustat was administered as a prophylactic treatment in patients before renal transplantation." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving migalastat hcl 150 mg?

"Migalastat was well tolerated and produced statistically significant reductions in glycating proteins by reducing both glycated and total hemoglobin A(1)(HbA1c) in individuals with diabetes and renal disease." - Anonymous Online Contributor

Unverified Answer

Has migalastat hcl 150 mg proven to be more effective than a placebo?

"An H2-receptor antagonist does not seem as effective as ursodeoxycholic acid in the treatment of fabry disease. There were no significant differences between migalastat and a placebo both in terms of clinical and biochemical response. Clinical trial number: UMIN000003056. [Bupropion was not evaluated in this study. The primary study was published in Journal of Nutrition. See citation for full discussion and references.]." - Anonymous Online Contributor

Unverified Answer

What is the latest research for fabry disease?

"Fabry disease is a rare disease requiring a well-defined clinical trials database. A 2013 study found that patients treated with Alglucerase who began receiving treatment before they grew big, or those who were smaller at the start of treatment, appeared to have better outcomes than those that were normal-size at the start of their treatment. Fabry disease is a condition that may develop throughout life. You can find the most up-to-date information, and clinical trials for Fabry disease using [Power](http://www.withpower." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of migalastat hcl 150 mg?

"Common side effects (>10%) were nausea, headache, and diarrhea. There was no clinically significant change in QTc intervals or serum potassium levels or no clinically meaningful changes in hepatic enzyme activity when migalastat was given at the recommended dose. These data suggest that migalastat has acceptable safety when taken at the recommended dose in patients with fibromyalgia." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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