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NCT03865836 Clinical Trial

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Pompe Disease Clinical Trial

Official title:
Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease

Clinical Trial Details — Status: Available

Administrative data
NCT number NCT03865836
Other study ID # ATB200-11
Secondary ID
Status Available
Phase
First received
Last updated

Study information
Verified date July 2023
Source Amicus Therapeutics
Contact For Site
Phone 609-662-2000
Email PompeSiteInfo@amicusrx.com
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial Summary

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Description:

This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.

Recruitment information / eligibility
Status Available
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group N/A and older
Eligibility 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)

Study Design

Related Conditions & MeSH terms

Intervention

Biological:
• ATB200

Drug:
• AT2221

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Amicus Therapeutics
See also
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Completed NCT02240407 - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease Phase 1
Completed NCT05073783 - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A

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