Higher Dose of Alglucosidase Alpha for Pompe Disease

Launched by TAIPEI VETERANS GENERAL HOSPITAL, TAIWAN · Aug 17, 2021

Keywords

ClinConnect Summary

This clinical trial is looking at whether a higher dose of a medication called Alglucosidase Alpha can help improve the health and safety of patients with Pompe disease, a condition that affects how the body processes a certain type of sugar. The researchers want to see if this increased dosage can lead to better long-term results for patients and help create new guidelines for treatment.

To be part of the study, participants must have a confirmed diagnosis of Pompe disease and currently be receiving enzyme replacement therapy (ERT), which is a common treatment for this condition. They also need to agree to participate by signing a consent form. However, individuals who have not attended their regular medical visits or have allergies to the treatment will not be eligible. The trial is still in the planning stages and has not started recruiting participants yet, but it will include individuals of all ages, from infants to adults. Participants in the trial can expect to receive close monitoring and care as part of their involvement.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Diagnosis of Pompe disease
• • 2. Currently receiving ERT with regular clinic visits
• • 3. Agree to sign informed consent
• Exclusion Criteria:
• • 1. Lost follow-up for clinical visits
• • 2. Allergy to Myozyme
• • 3. Refuse to sign informed consent