A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.

• Register: EUCTR
• Last refreshed on: 11 April 2016
• Main ID: EUCTR2011-004800-40-IT
• Date of registration: 12/03/2012
• Prospective Registration: Yes
• Primary sponsor: GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
• Public title: A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.
• Scientific title: An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease.
• Date of first enrolment: 15/05/2012
• Target sample size: 100
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-004800-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 1

Countries of recruitment

Australia, Austria, Belgium, Brazil, Denmark, Egypt, Italy, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Clinical Trials Helpdesk
• Affiliation: GlaxoSmithKline Research & Development

• Name: Clinical Trials Helpdesk
• Affiliation: GlaxoSmithKline Research & Development

Key inclusion & exclusion criteria

Inclusion criteria:
1.Subject with Fabry disease who completed treatment in a previous study of migalastat HCl given as monotherapy. 2. Male or female subjects 16 years of age or older. Note: Subjects under the age of 18 will be enrolled only at sites with all required regulatory and ethics approvals to do so. 3. A female subject is eligible to participate if she is: A. Of non-childbearing potential, or B. Of childbearing potential and NOT pregnant or nursing, has a negative urine pregnancy test at the Baseline Visit (Visit 1), and agrees to one of the methods of avoiding pregnancy listed in Appendix 1 as per the study protocol from the time of first dose of study medication until 30 days after study completion. A female is considered ''Non-childbearing potential'' if she is status-post hysterectomy, status-post surgical removal of both ovaries, has current, documented tubal ligation, or is postmenopausal and >2 years without menses. Female subjects who are post-menopausal <2 years must be confirmed menopausal by Follicle Stimulating Hormone (FSH) and estradiol levels. A female is considered ''childbearingpotential'' if she has functional ovaries, ducts, and uterus with no impairment that would cause sterility. This includes women with oligomenorrhea (even severe), and women who are perimenopausal or who have just begun to menstruate. 4. Male subjects must agree to use one of the contraception methods listed in Appendix 1. This criterion must be followed from the time of the first dose of study medication until 30 days after study completion. 5. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI) or has a legally authorized representative who has given written informed consent. 6. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 96
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. The last available estimated glomerular filtration rate (eGFR) in the previous study was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of Baseline Visit (Visit 1), which is >30 mL/min/1.73m2. 2. The subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis. 3. The subject is treated or has been treated with another investigational drug (except migalastat HCl) within 30 days of study start. 4. Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a- galactosidase A.
MedDRA version: 14.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Intervention(s)

Product Name: Migalastat Hydrochloride
Product Code: GR181413
Pharmaceutical Form: Capsule
INN or Proposed INN: Migalastat Hydrochloride
CAS Number: 75172-81-5
Current Sponsor code: AT1001
Other descriptive name: GR181413A
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Primary end point(s): The safety assessments are: • Adverse events (AEs), Possible Suicidality Related AEs (PSRAEs) and serious adverse events (SAEs) • Withdrawal due to AEs • Vital signs (blood pressure and heart rate) and body weight • Hematology, chemistry, and urinalysis parameters • Echocardiography (ECHO) • Electrocardiograms (ECGs)

Secondary Objective: To explore long-term efficacy/pharmacodynamics of migalastat HCl in subjects with Fabry disease who have completed treatment in a previous study of migalastat HCl.

Timepoint(s) of evaluation of this end point: AEs will be collected from the start of study treatment (i.e., Visit 1) and until the follow-up contact.

Main Objective: To assess long-term safety of migalastat HCl in the treatment of subjects with Fabry disease who completed treatment in a previous study of migalastat HCl.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Change from baseline will be evaluated for each efficacy endpoint, where baseline is defined (as in Table 1 of the study protocol) as the value from the last treatment visit of the previous migalastat HCl study.

Secondary end point(s): The efficacy/pharmacodynamic assessments are: • Estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease (MDRD) equation. • Measurement of 24-hour urine protein • Evaluation of left ventricular mass index (LVMi ), as measured by echocardiography • Ejection fraction, as measured by echocardiography • Evaluation of urine globotriaosylceramide (GL-3) from 24-hour urine collection • Evaluation of leukocyte a-galactosidase A (a-Gal A) activity • Evaluation of patient reported assessment of pain as assessed by the Brief Pain Inventory (BPI) short form • Evaluation of patient reported Quality of Life as assessed by the Short Form -36 survey (SF-36).

Secondary ID(s)

MGM116041

2011-004800-40-GB

Source(s) of Monetary Support

GlaxoSmithKline Research & Development Limited