ATB200/AT2221 Expanded Access Study in Adult Pompe disease Subject

JRCT ID: jRCT2061210056

Registered date:04/12/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Pompe disease
• Date of first enrollment: 22/12/2021
• Target sample size: 10
• Study type: Interventional
• Intervention(s): ATB200 IV infusion over a 4-hour duration 1 hour after AT2221 administration every 2 weeks

Outcome(s)

• Primary Outcome: To evaluate the safety of ATB200/AT2221 co-administration

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Male or female, 18 years old and above
2. A diagnosis of LOPD based on documented deficiency of acid alpha-glucosidase (GAA) enzyme activity or GAA genotyping
3. Female subjects of childbearing potential and male subjects if they are using appropriate, reliable contraception

Exclude criteria

1. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat
2. Subject has a medical condition or any other extenuating circumstance that may pose an undue
safety risk to the patient
3. Subject, if female, is pregnant or breastfeeding
4. Subject, whether male or female, is planning to conceive a child during the study
5. Subject has received treatment with prohibited medications within 30 days of screening

Related Information

• Primary Sponsor: Wright Jacquelyn