Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Pompe Disease Infantile-Onset Clinical Trial

Official title:

Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT04327973
• Other study ID #: ATB200-15
• Status: Available

Study information

• Verified date: July 2023
• Source: Amicus Therapeutics
• Contact: For Site
• Phone: 609-662-2000
• Email: PompeSiteInfo[@]amicusrx.com
• Is FDA regulated: No
• 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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 0 Years to 17 Years

Eligibility

Inclusion Criteria:

1. Patient is male or female and 0 to < 18 years old.

2. Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.

3. Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.

4. Patient is currently being treated with an approved ERT and is declining (eg, worsening in motor function, respiratory function/parameters, cardiac function, etc) based on treating physician's clinical judgement.

5. The patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering ATB200/AT2221 in a manner consistent with all national requirements.

6. If of reproductive potential and sexually active, female subjects agree to use a highly effective method of contraception throughout the duration of the treatment and for up to 90 days after their last dose of ATB200/AT2221.

Exclusion Criteria:

1. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221

2. Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator and/or Amicus, pose an undue safety risk to the patient.

3. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease Infantile-Onset

Intervention

Biological:
• ATB200
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Drug:
• AT2221
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Locations

Country	Name	City	State
Italy	University Hospital of Padova	Padova
Taiwan	National Taiwan University Hospital	Taipei
United States	UF Health Shands Hospital	Gainesville	Florida
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Italy,  Taiwan,