NCT06130228|UnknownPhase 2DMC
Nutritional Therapy in Late-onset Pompe Disease
PDT-MIS
Multi-ingredient Supplementation as an Adjunctive Therapy in Late-onset Pompe Disease
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Sponsor study identifier
interventional
28
0 countries
N/A
Study Start
April 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Primary Completion
September 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
April 1, 2025
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
April 1, 2025
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
November 8, 2023
Actual (reported)November 14, 2023
Actual (reported)April 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
September 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
April 1, 2025
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Percent change in the body composition index by DEXA analyses
Body composition index (lean mass/fat mass ratio)
Baseline to 4 months
Percent change in seated pulmonary function by spirometry
Seated forced expiratory volume/forced vital capacity ratio (FEV1/FVC)
Baseline to 4 months
Percent change in supine pulmonary function by spirometry
Supine forced expiratory volume/forced vital capacity ratio (FEV1/FVC)
Baseline to 4 months
Multi-ingredient supplementation (PDT-MIS) consists of daily intake of high-quality proteins, creatine, vitamin D, calcium, plant extracts (green coffee bean, green tea, beet root, and forskolin), and Omega-3 fatty acids. Concurrent with supplementation, patients will do mixed rehabilitative exercise (cardio and strength) and respiratory muscle training four days a week.
Placebo (PLA) consists of daily intake of collagen, safflower, and microcrystalline cellulose. Concurrent with supplementation, patients will do mixed rehabilitative exercise (cardio and strength) and respiratory muscle training four days a week.
Supplementation with active PDT-MIS daily
Supplementation with inactive placebo
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Mark Tarnopololsky, MD/PhD
McMaster University
First Submitted
November 8, 2023
First Posted
November 14, 2023
Percent change in 6-minute walking test distance
6-minute walking test distance (meters)
Baseline to 4 months
Percent change in health-related quality of life by SF-36 Survey
Baseline to 4 months
Percent change in health-related quality of life by Rotterdam Handicap Score
Baseline to 4 months
Percent change in health-related quality of life by the R-Pact Questionnaire
Baseline to 4 months
Percent change in maximal grip strength by dynamometry
Baseline to 4 months
Percent change in isometric leg strength by Biodex
Baseline to 4 months
Percent change in malondialdehyde levels in blood
Baseline to 4 months
Percent change in Oxygen Radical Absorbance Capacity in blood
Baseline to 4 months
Percent change in interleukin 6 levels in blood
Baseline to 4 months
Contact the study team to confirm eligibility.
Study Start
April 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Primary Completion
September 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
April 1, 2025
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Last Updated
November 14, 2023
Record last verified: 2023-11
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Source: ClinicalTrials.gov.
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