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Nutritional Therapy in Late-onset Pompe Disease

NCT06130228 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2023-11-14

No results posted yet for this study

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-glucosidase. The adult/late onset form (LOPD) leads to glycogen accumulation and autophagic buildup, causing progressive muscle weakness that leads to wheelchair dependence, reduced quality of life and premature death due to cardiorespiratory insufficiency. While nutritional strategies, such as the low carbohydrate/high protein and ketogenic diets, have been used clinically, they are difficult to maintain and have limited benefits. Multi-ingredient supplementation (MIS) allows for targeting of several underlying pathogenic pathways and may be more convenient than traditional dietary strategies, thereby improving both adherence and LOPD pathology.

Conditions

  • Pompe Disease
  • Muscle Loss
  • Obesity
  • Nutrition Poor
  • Lysosomal Storage Diseases
  • Glycogen Storage Disease Type II
  • Glycogen Storage Disease Type II Late Onset
  • Glycogen Storage Disease Type II, Adult

Interventions

DIETARY_SUPPLEMENT

Multi-ingredient supplement (PDT-MIS)

Supplementation with active PDT-MIS daily

DIETARY_SUPPLEMENT

Placebo (PLA)

Supplementation with inactive placebo

Sponsors & Collaborators

Principal Investigators

  • Mark Tarnopololsky, MD/PhD · McMaster University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
21 Years
Max Age
90 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-01
Primary Completion
2024-09-01
Completion
2025-04-01

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06130228 on ClinicalTrials.gov