Status:

UNKNOWN

Nutritional Therapy in Late-onset Pompe Disease

Lead Sponsor:

McMaster University

Conditions:

Pompe Disease

Muscle Loss

Eligibility:

All Genders

21-90 years

Phase:

PHASE2

Brief Summary

RATIONALE: Pompe disease (PD) is a recessive genetic disorder wherein the body cannot break down glycogen due to a mutation in the acid alpha glucosidase (GAA) gene, which encodes for acid alpha-gluco...

Detailed Description

DESIGN AND INTERVENTION: The present study is a 4-month randomized, double-blind, placebo-controlled clinical trial (RCT) with sampling pre and post intervention in late onset Pompe disease patients u...

Eligibility Criteria

Inclusion

  • Genetically confirmed LOPD
  • Have undergone enzyme replacement therapy for at least three months.
  • Physically capable of doing rehabilitative exercise, respiratory muscle training, and the clinical tests described herein.

Exclusion

  • Dairy protein allergy
  • Renal disease (creatinine \> 140)
  • Attempting pregnancy or currently pregnant
  • Current supplementation

Key Trial Info

Start Date :

April 1 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

April 1 2025

Estimated Enrollment :

28 Patients enrolled

Trial Details

Trial ID

NCT06130228

Start Date

April 1 2024

End Date

April 1 2025

Last Update

November 14 2023

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