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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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7 August 2023 |
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Main ID: |
NCT03865836 |
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Date of registration:
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05/03/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
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Scientific title:
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Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease |
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Date of first enrolment:
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June 12, 2019 |
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Target sample size:
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Recruitment status: |
Available |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03865836 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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Contacts
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Name:
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For Site |
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Address:
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Telephone:
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609-662-2000 |
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Email:
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PompeSiteInfo@amicusrx.com |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Subject must have a diagnosis of Pompe disease based on documentation of one of the
following:
1. deficiency of GAA enzyme
2. GAA genotyping
2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or
is declining on currently approved ERT (e.g. Myozyme)
3. The patient must be willing to receive treatment with ATB200/AT2221 via this program,
which includes signing an authorization form for sharing clinical data with Amicus
Therapeutics, and its agent Early Access Care LLC.
Exclusion Criteria:
1. Patient, whether male or female, is planning to conceive a child during the treatment
program.
2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa,
or AT2221
3. Patient has a medical or any other extenuating condition or circumstance that may, in
the opinion of the investigator, pose an undue safety risk to the subject or
compromise his/her ability to comply with or adversely impact protocol requirements.
4. Patient has received any gene therapy at any time.
5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca),
acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Biological: ATB200
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Drug: AT2221
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Secondary ID(s)
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ATB200-11
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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