Fabry DiseaseNCT01476163
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
A study testing migalastat HCl 150 mg and migalastat HCl 20 mg in people with Fabry Disease, run by Amicus Therapeutics.
Trial summary
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- migalastat HCl 150 mg and migalastat HCl 20 mg
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
- At least 2 years of age
- Strong clinical indication for treatment of Fabry disease
- No other treatment option including either unsuitable for ERT or unable to access ERT
- Appropriate female and male contraception
- Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data
Who cannot join
- Scheduled for renal or other organ transplant or replacement therapy
- Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa)
- Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol
- Treated with another investigational drug within 30 days of start of migalastat HCl treatment
- Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.
Trial locations and contacts
No sites are listed yet.
Amicus Therapeutics Patient Advocacy contact
About this study
Official title: Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
Read the detailed description
This Physician Initiated Request program allows physicians to request permission from Amicus to receive migalastat HCl for specific patients with Fabry disease who have a mutation amenable to this treatment, who do not have access to commercial Galafold or available treatment alternatives, or do not meet requirements for participation in an existing migalastat clinical study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive Amicus permission for participation. Key criteria for participation include: At least 2 years old; Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT.
- Conditions
- Fabry Disease
- Being tested
- migalastat HCl 150 mg, migalastat HCl 20 mg
- Keywords
- AT1001, migalastat, Fabry disease, Fabry, Amicus, lysosomal disease, lysosomal disorder
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
migalastat HCl 150 mgDrug
Also called AT1001, migalastat
150 mg capsule
migalastat HCl 20 mgDrug
Also called AT1001, migalastat
20 mg dispersible tablets; dosing will be based on body weight
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2011 (estimated)
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Clinical Research, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01476163
- Sponsor's protocol ID
- AT1001-188
- Status last verified
- Oct 2026
- First submitted
- 17 Nov 2011
- Last updated
- 27 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)
- ClinicalTrials.gov ID
- NCT01476163
- Acronym / sponsor ID
- AT1001-188
- Conditions
- Fabry Disease
- Keywords
- AT1001, migalastat, Fabry disease, Fabry, Amicus, lysosomal disease, lysosomal disorder
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- First posted
- 22 Nov 2011 (estimated)
- 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.
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
Detailed description
This Physician Initiated Request program allows physicians to request permission from Amicus to receive migalastat HCl for specific patients with Fabry disease who have a mutation amenable to this treatment, who do not have access to commercial Galafold or available treatment alternatives, or do not meet requirements for participation in an existing migalastat clinical study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive Amicus permission for participation. Key criteria for participation include: At least 2 years old; Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
- At least 2 years of age
- Strong clinical indication for treatment of Fabry disease
- No other treatment option including either unsuitable for ERT or unable to access ERT
- Appropriate female and male contraception
Key exclusion criteria (5 listed)
- Scheduled for renal or other organ transplant or replacement therapy
- Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa)
- Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol
- Treated with another investigational drug within 30 days of start of migalastat HCl treatment
- Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| migalastat HCl 150 mgAlso: AT1001, migalastat150 mg capsule | Drug | – |
| migalastat HCl 20 mgAlso: AT1001, migalastat20 mg dispersible tablets; dosing will be based on body weight | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
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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.
Amicus Therapeutics Patient Advocacy contact
Medical Monitor, Clinical Research study director
Amicus Therapeutics
Questions about this trial
What is this clinical trial studying?
A study testing migalastat HCl 150 mg and migalastat HCl 20 mg in people with Fabry Disease, run by Amicus Therapeutics.
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 6 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Amicus Therapeutics Patient Advocacy (patientadvocacy@amicusrx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
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 27 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.