Fabry DiseaseNCT03135197
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
An observational study of Fabry Disease, run by University Hospital Muenster at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 74 years
- Conditions the study is for
- Fabry Disease
- How many people
- 75 participants
- Where
- 1 siteGermany
- Study dates
- 8 Jun 2017to 30 Jun 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 74 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
Who may be able to join
- 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).
- The following Inclusion criteria refer to the time of Migalastat initiation (T0):
- 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.
Who cannot join
- 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.
- The following Exclusion criteria refer to the time of Migalastat initiation (T0):
Show all 9Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the detailed description
Phase 3 data should be confirmed in this study with long-term data.
- LVMI is expected to remain stable or to be ameliorated over an average of 24 months treatment duration. The LVMI reduction observed in patients followed up to 24 months is expected to be significantly reduced with a mean change of -6.6 g/m2 (-11.0, -2.1, 95% CI).
- eGFR [CKD-EPI] is expected to remain stable over an average of 24 months treatment duration. The long-term effect of Migalastat on eGFR is expected to be comparable to the decline over time in healthy adults. The annualized rate of change over this period is expected to be ≤1 mL/min/1.73 m2 in females and ≤3 mL/min/1.73 m2 in males.
- Significant reduction is expected in plasma lyso-Gb3 concentration at month 6, month 12 and month 24 following treatment with Migalastat.
- ERT-naïve patients treated with Migalastat are expected to show an improvement of GI symptoms (diarrhea) over 24 months.
- No progression of White Matter Lesions (WML) during treatment duration is expected.
- No higher frequency of stroke/transient cerebral ischemia during treatment duration is expected.
- Severity of neuropathic pain is expected to remain stable or to improve during treatment duration.
- Dosing/amount of symptomatic medications of neuropathic symptoms is expected to decrease during treatment duration.
- Conditions
- Fabry Disease
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Migalastat | Migalastat administered according to SmPC |
What is being measured
The primary outcome is the main question the study is designed to answer.
LVMI
Primary endpoint of the observational study is the change in left ventricular mass index (LVMI) over two years.
Measured: two years
Secondary outcomes (11)
GFR
Change in GFR over 24 months
Measured: 24 months
Cerebral ischemia or stroke.
Incidence of transient/manifest cerebral ischemia or stroke over 24 months.
Measured: 24 months
Neuropathic Pain (GCPS)
Change in severity of neuropathic pain measured by Graded Chronic Pain Scale (GCPS)
Measured: 24 months
Neuropathic Pain (NPSI)
Change in severity of neuropathic pain measured by Neuropathic Pain Symptom Inventory (NPSI) Score (items are quantified on a (0-10) numerical scale).
Measured: 24 months
Fabry Disease Severity (MSSI)
Change in disease severity measured by Mainz Severity Score Index (MSSI)
Measured: 24 months
Fabry Disease Severity (DS3)
Change in disease severity measured by the Disease Severity Scoring System (DS3)
Measured: 24 months
Lyso-Gb3
Change in Lyso-Gb3
Measured: 24 months
White Matter Lesion load
Change of White Matter Lesion load (quantified by WML volumetry [ml]).
Measured: 24 months
Cerebral microbleeds/hemorrhagic lesions.
Stabilization of cerebral microbleeds/hemorrhagic lesions.
Measured: 24 months
Gastrointestinal symptoms
Change in gastrointestinal symptoms (gastrointestinal symptoms rating scale, GSRS).
Measured: 24 months
Quality of life (SF-36)
Change in quality of life (Short Form (SF-36) Health Survey: 36-item, patient-reported survey of patient health).
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2017
- Study start8 Jun 2017
- Primary completion30 Jun 2020
- Study completion30 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Muenster
- Sponsor type
- University, hospital or other
- Collaborators
- Amicus Therapeutics
- Responsible party
- Sponsor
- Investigators
- Eva Brand, Prof., Principal investigator, University Hospital Muenster
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03135197
- Sponsor's protocol ID
- Fabry_Migalastat
- Status last verified
- Dec 2020
- First submitted
- 20 Apr 2017
- Last updated
- 5 Jan 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
- ClinicalTrials.gov ID
- NCT03135197
- Acronym / sponsor ID
- Fabry_Migalastat
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- University Hospital MuensterUniversity, hospital or other
- Collaborators
- Amicus Therapeutics
- Study start
- 8 Jun 2017 (actual)
- Primary completion
- 30 Jun 2020 (actual)
- Study completion
- 30 Jun 2020 (actual)
- First posted
- 1 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Phase 3 data should be confirmed in this study with long-term data.
- LVMI is expected to remain stable or to be ameliorated over an average of 24 months treatment duration. The LVMI reduction observed in patients followed up to 24 months is expected to be significantly reduced with a mean change of -6.6 g/m2 (-11.0, -2.1, 95% CI).
- eGFR [CKD-EPI] is expected to remain stable over an average of 24 months treatment duration. The long-term effect of Migalastat on eGFR is expected to be comparable to the decline over time in healthy adults. The annualized rate of change over this period is expected to be ≤1 mL/min/1.73 m2 in females and ≤3 mL/min/1.73 m2 in males.
- Significant reduction is expected in plasma lyso-Gb3 concentration at month 6, month 12 and month 24 following treatment with Migalastat.
- ERT-naïve patients treated with Migalastat are expected to show an improvement of GI symptoms (diarrhea) over 24 months.
- No progression of White Matter Lesions (WML) during treatment duration is expected.
- No higher frequency of stroke/transient cerebral ischemia during treatment duration is expected.
- Severity of neuropathic pain is expected to remain stable or to improve during treatment duration.
- Dosing/amount of symptomatic medications of neuropathic symptoms is expected to decrease during treatment duration.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Migalastat | – | –Migalastat administered according to SmPC |
Who may qualify
- Ages
- 16 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
LVMI
Time frame: two years
Primary endpoint of the observational study is the change in left ventricular mass index (LVMI) over two years.
11 secondary outcomes
GFR
Time frame: 24 months
Cerebral ischemia or stroke.
Time frame: 24 months
Neuropathic Pain (GCPS)
Time frame: 24 months
Neuropathic Pain (NPSI)
Time frame: 24 months
Fabry Disease Severity (MSSI)
Time frame: 24 months
Fabry Disease Severity (DS3)
Time frame: 24 months
Lyso-Gb3
Time frame: 24 months
White Matter Lesion load
Time frame: 24 months
Cerebral microbleeds/hemorrhagic lesions.
Time frame: 24 months
Gastrointestinal symptoms
Time frame: 24 months
Quality of life (SF-36)
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eva Brand, Prof. principal investigator
University Hospital Muenster
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by University Hospital Muenster at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
LVMI (time frame: two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.