German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.

NCT03135197 | Completed

• 1 other identifier: Fabry_Migalastat
• Study Type: observational
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of the study is to document long term data on treatment with Migalastat under "real world" conditions. The selection of patients is based on the SmPC/Fachinformation. The study duration/patient will be 2 years.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• LVMI

  Primary endpoint of the observational study is the change in left ventricular mass index (LVMI) over two years.

  two years

Secondary Outcomes (11)

• GFR

  24 months

• Cerebral ischemia or stroke.

  24 months

• Neuropathic Pain (GCPS)

  24 months

• Neuropathic Pain (NPSI)

  24 months

• Fabry Disease Severity (MSSI)

  24 months

Study Arms (1)

Migalastat

Migalastat administered according to SmPC

Eligibility Criteria

• Age: 16 Years - 74 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

At therapy initiation adult male and female patients with FD will be identified, whose GLA mutations are amenable for a stimulation with Migalastat-HCl, and will be treated with Migalastat-HCl at the recommended dose continuously for 24 months according to the manufacturers' instructions (SmPC).

You may qualify if:

• Males and females, 16 to 74 years, diagnosed with Fabry disease.
• Amenable GLA mutation.
• Treatment with Migalastat (initiation of therapy according to recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document. Biegstraaten et al, Orphanet J Rare Dis. 2015;10:36. AWMF-Leitlinien Morbus Fabry, Diagnose und Therapie, Registernummer 030-134).
• ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months).
• Estimated GFR (eGFR, CKD-EPI formula) at screening ≥30 ml/min/1.73 m2
• Subjects taking no ACE inhibitors, ARBs, or renin inhibitors or are on a stable dose for at least 4 weeks before screening.
• Subjects taking no analgesics/antidepressants or are on a stable dose for at least 4 weeks before screening.

You may not qualify if:

• Patient has a non-amenable GLA mutation or the mutation A143T or D313Y (for verification of amenable mutations please refer to: www.GalafoldAmenablityTable.com or to the "Fachinformation").
• Patient is unwilling to give informed consent.
• Patient is unable to comply with the clinical protocol.
• Patients on co-medication: Galafold plus Enzyme Replacement Therapy (ERT)
• Pregnant or breast feeding women.
• Patients on dialysis
• Patient has a clinically significant organ disease (e.g. cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial.
• Patients with a history of organ transplantation.

Sponsors & Collaborators

• University Hospital Muenster: lead
• Amicus Therapeutics: collaborator

Study Sites (1)

Universtiy Hospital Münster

Münster, 48149, Germany

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Eva Brand, Prof.

  University Hospital Muenster

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2017
• First Posted: May 1, 2017
• Study Start: June 8, 2017
• Primary Completion: June 30, 2020
• Study Completion: June 30, 2020
• Last Updated: January 5, 2021

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)