TRIPOD
Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg/kg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)
Sponsor study identifier
Secondary identifiers
CTIS
interventional
10
0 countries
N/A
Study Start
October 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Primary Completion
December 31, 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 22, 2024
Actual (reported)August 28, 2024
Actual (reported)October 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
December 31, 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Changes in muscle strength and function by manual muscle testing with Medical Research Council (MRC) grading scale
Muscle strength will be determined by manual muscle testing using the Medical Research Council (MRC) grading scale. Muscle strength is graded from 0-5 by physical examination, in which grade 5 represents normal muscle strength and grade 0 means paralysis of the muscle group tested. Muscle groups tests will include neck flexors/extensors, shoulder abductors, elbow extensors/flexors, wrist extensors/flexors, hip extension, hip flexors, hip adductors/abductors, knee extensors/flexors, and foot extensors/flexors.
At baseline and every 3 months for a duration of 21 months.
Changes in muscle strength and function by testing hand-held dynamometry (HHD)
Muscle strength will be measured with the CITEC hand-held dynamometer (HHD). Thirteen muscle groups will be tested: neck extension, neck flexion, and bilateral shoulder abduction, elbow flexion, elbow extension, wrist extension, squeezing, hip flexion, hip extension, hip abduction, knee flexion, knee extension, ankle dorsiflexion, and ankle plantar flexion. A mean value in Newton (N) is calculated from three consecutive measurements per muscle group.
At baseline and every 3 months for a duration of 21 months.
Changes in muscle strength and function by quick motor function test (QMFT)
Changes in lean body mass assessed by DEXA-scans
At baseline, after 9 months and at the end of the study (21 months).
Changes in lean body mass assessed by DEXA-scans
At baseline, after 9 months and at the end of the study (21 months).
Bone density assessed by DEXA-scans
At baseline, after 9 months and at the end of the study (21 months).
Need for walking devices and artificial ventilation (yes/no)
At baseline and every 3 months for a duration of 21 months.
Changes in alglucosidase alfa activity in plasma
At 3 months and 9 months.
Alglucosidase alfa will be administered by intravenous infusion at a dose of 20 mg/kg once every 4 weeks instead of once every 2 weeks for a duration of 9 months. After 9 months of treatment with the extended interval, it will be determined for each patient whether it is considered safe to discontinue enzyme replacement therapy (ERT). Both, patients who stop ERT after 9 months and those who continue with either the new or the previous dosing schedule, will be closely followed for an additional 12 months, leading to a total study duration of 21 months.
The interval of ERT with alglucosidase alfa will be extended from once every 2 weeks to once every 4 weeks. The dose of 20 mg/kg per infusion remains the same.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Pieter A. van Doorn, Prof. dr.
Erasmus Medical Center
Nadine A.M.E. van der Beek, Dr.
Erasmus Medical Center
Tim Preijers, Dr.
Erasmus Medical Center
First Submitted
August 22, 2024
First Posted
August 28, 2024
Study Start
October 1, 2024
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
The QMFT will be administered at the time points specified. This 16-item functional test has been developed to evaluate the function of the muscle groups that are specifically affected in patients with Pompe disease. The patient is asked to perform multiple activities in supine, sitting and standing position. The performance of the patient is scored on a 5-point ordinal scale (range 0-4). This standardized test can be performed within 5-10 minutes and can be performed in children and adults with varying disease severity.
At baseline and every 3 months for a duration of 21 months.
Changes in muscle strength and function by 6-minute walk test (6MWT)
The 6MWT is a timed test that measures functional endurance. The primary measurement is the distance walked in 6 minutes, measured in meters. The percent of predicted distance and the amount of time walked (to quantify endurance, as not all patients may complete the full 6-minute walk) will also be recorded. The patient is instructed to walk the length of a pre-measured hallway for six minutes. It is widely regarded as an objective measure, which is easy to perform and reflects the performance in ADL of the patient.
Every 3 months for a duration of 21 months.
Changes in pulmonary function by testing forced vital capacity (FVC) in sitting and supine positions
Pulmonary function testing will be performed at the time points specified. Forced vital capacity will be measured during sitting and supine positions as a measure of lung volume (and indirect measure of the strength of the diaphragm and abdominal muscles). The pulmonary testing protocol is standardized in accordance with ATS/ERS Guidelines36. Three repeated reproducible flow volume curves will be made. The best effort will be used for further analyses. Values will be expressed as a percentage of the predicted normal values or as z-score, based on age, gender, race, and height. Global Lung Initiative (GLI) 2012 reference equations will be used.
At baseline and every 3 months for a duration of 21 months.
Changes in pulmonary function by testing maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP)
In addition to the vital capacity, inspiratory (MIP) and expiratory (MEP) mouth pressures will be measured in upright seated position to determine the maximum force that can be exerted by the respiratory muscles. MIP will be measured at residual volume after maximal expiration, while MEP is registered at total lung capacity after maximal inspiration. Pressures must be maintained at least 1 second. Maneuvers will be repeated until three reproducible methods are obtained. The highest value will be taken for analyses.
At baseline and every 3 months for a duration of 21 months.
Changes in patient reported outcome measures by filling in the Rasch-built Pompe-specific activity (R-PAct) scale
The Rasch-built Pompe-specific activity (R-PAct) scale is a self-reported, 18-items questionnaire designed specifically for use in patients with Pompe disease, based upon experiences from patients about their most important and limiting aspects in daily life. All items have three response options: \[0\] unable to perform; \[1\] able to perform, but with difficulty or \[2\] able to perform without difficulty. If all items are answered, an appropriate centile metric score (range 0-100) will be calculated.
At baseline and every 3 months for a duration of 21 months.
Changes in patient reported outcome measures by filling in the SF-36 questionnaire
The SF-36 is a health-related quality of life questionnaire, consisting of 36 items. The items are assigned to the domains of physical functioning, role functioning-physical, role functioning-emotional, social functioning, body pain, mental health, vitality, general health perception and change in health.
At baseline, after 9 months and at the end of the study (21 months).
Body weight
Body weight will be measured in kilograms (kg).
At baseline and every 3 months for a duration of 21 months.
Heart rate
Heart rate will be measured in beats per minute (bpm).
At baseline and every 4 weeks at the start and at the end of the aglucosidase alfa infusion for a duration of 9 months. If alglucosidase alfa treatment is not stopped after 9 months, the measurement will take place for a total duration of 21 months.
Systolic and diastolic blood pressure
Systolic and diastolic blood pressure will be measured in millimeter of mercury (mmHg).
At baseline and every 4 weeks at the start and at the end of the aglucosidase alfa infusion for a duration of 9 months. If alglucosidase alfa treatment is not stopped after 9 months, the measurement will take place for a total duration of 21 months.
Respiratory rate
Respiratory rate will be measured in breaths per minute.
At baseline and every 4 weeks at the start of the aglucosidase alfa infusion for a duration of 9 months. If alglucosidase alfa treatment is not stopped after 9 months, the measurement will take place for a total duration of 21 months.
Contact the study team to confirm eligibility.
Primary Completion
December 31, 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
August 28, 2024
Record last verified: 2024-08
All data will be shared upon a reasonable request to the PI of the study.
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Source: ClinicalTrials.gov.
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