Pompe Disease (Late-onset)Phase 4TRIPODNCT06575829

Treatment Frequency Reduction in Pompe Disease

A Phase 4 study testing Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks in people with Pompe Disease (Late-onset) and GAA Deficiency, run by Erasmus Medical Center.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts

Official record

Search all trials

Trial summary

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. Read more about this study

Who is it for?: Ages 50 years and older
Conditions the study is for: Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II
What is being tested?: Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: No sites listed yet
Study dates: 1 Oct 2024to 31 Dec 2027
Phase: Phase 4
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 50 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confirmed disease-causing variants in the GAA gene)
Age ≥50 years
Current treatment with alglucosidase alfa at a standard dose of 20 mg/kg once every 2 weeks for ≥4 years
Relatively stable clinical condition over the past year
Able to walk ≥150 m within 6 minutes (6MWT)
(Forced) vital capacity (FVC) in sitting position: >55% of expected value and in supine position: >45% of expected value
Willing and able to adhere to the study procedures

Who cannot join

Rapidly progressive muscle weakness
Severely limited muscle strength almost requiring/requiring daily wheelchair use
Requiring respiratory support (non-invasive/invasive ventilation) or being at high risk to require respiratory support (ventilation) due to further deterioration of current pulmonary function. Using continuous positive airway pressure (CPAP) support only for obstructive sleep apnea syndrome (OSAS) is permitted.
Comorbidities which are expected to influence the primary outcome measures within the next 2 years

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

Ina Barzel, MSc contact

+31(0)107031182 |

i.barzel@erasmusmc.nl

Lianne H. Potters, MSc contact

+31(0)0633342010

l.potters@erasmusmc.nl

About this study

Official title: 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)

Read the detailed description

All eligible patients with late-onset Pompe disease will be treated with alglucosidase alfa 20 mg/kg once every 4 weeks for 9 months. During the study, patients will be monitored once every 3 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). The investigators consider it safe: 1] if the patient is stable compared to the year prior to reducing the ERT frequency, or, 2] if the patient previously deteriorated (slightly) despite standard ERT and this deterioration is not exaggerated by the alternative dosing regimen. If after 9 months there is no valid medical reason to switch back to standard dosing (once every 2 weeks) and the patient does not wish to discontinue treatment, the 4-week dosing regimen will be continued. If at any moment a patient shows an unexpectedly rapid decline in clinical outcome parameters (significantly higher than their own course at regular treatment dosage), treatment will be switched back to or be restarted with the standard dosing regimen of 20 mg/kg every 2 weeks.

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 to be able to take action (e.g., switch to a standard dosing regimen or restart ERT) if a more rapid clinical deterioration occurs than expected, or to investigate if muscle and pulmonary function regain when standard dosage has been re-instituted after signs of clinical deterioration during the 4-week treatment interval. After the end of the study (21 months), patients will be carefully followed according to the standard frequency (once every 6 months). If a patient shows an unexpectedly rapid decline in clinical outcome parameters henceforth, treatment will be switched back to or be restarted with the standard dosing regimen of 20 mg/kg eow.

Conditions: Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II, Acid Maltase Deficiency
Being tested: Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks
Medical subject headings: Glycogen Storage Disease Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeksDrug
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.

Study groups in NCT06575829
Study group	Role	What they receive
Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks	Experimental	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. Receives: Drug: Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: At baseline and every 3 months for a duration of 21 months.
Changes in muscle strength and function by quick motor function test (QMFT)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: At baseline, after 9 months and at the end of the study (21 months).
Body weight
Body weight will be measured in kilograms (kg).
Measured: At baseline and every 3 months for a duration of 21 months.
Heart rate
Heart rate will be measured in beats per minute (bpm).
Measured: 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).
Measured: 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.
Measured: 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.

Secondary outcomes (8)

Changes in lean body mass assessed by DEXA-scans
Body composition will be measured conform DEXA technology (currently at Erasmus MC: GE Healthcare Lunar iDXA equipment: densitometer and encore software). This is a low-cost technology and simultaneously accurate, easy to perform and involves very low radiation dose (8 µSv per scan) to the patient which brings no harm. This imaging technique is the gold standard technique for the assessment of body composition.
Body composition will be presented as mineral bone, fat and fatty free body mass in kilograms.
Measured: At baseline, after 9 months and at the end of the study (21 months).
Changes in lean body mass assessed by DEXA-scans
Percentages of total body fat mass, android and gynoid fat will be measured. Percentages of fat mass will also be measured at regional level (arms, trunk and legs).
Measured: At baseline, after 9 months and at the end of the study (21 months).
Bone density assessed by DEXA-scans
Bone mineral density is measured at total body in g/cm2.
Measured: At baseline, after 9 months and at the end of the study (21 months).
Need for walking devices and artificial ventilation (yes/no)
For the purpose of stabilization and if deemed necessary by the investigator, the patient may use a walking device during the assessment, such as cane or walker. If such a device is used during the assessment, this must be recorded in the CRF/HiX. It will also be determined whether patients may need non-invasive or invasive ventilation during the study, including the hours of ventilation per day.
Measured: At baseline and every 3 months for a duration of 21 months.
Changes in alglucosidase alfa activity in plasma
Blood samples will be collected before and after infusion of alglucosidase alfa at week 13 and week 39. Sampling times are as follows: pre-dose, 15 minutes before end of infusion, directly after end of infusion and 9 hours after start of infusion. The activity of alglucosidase alfa will be analyzed by the clinical chemistry laboratory of the Erasmus MC and expressed as nanomol 4-methylumbelliferyl-α-glucopyranoside (MU)/hour/10 microliter.
Measured: At 3 months and 9 months.
Serological testing of serum creatine kinase (CK) and antibodies against alglucosidase alfa
Blood samples will be drawn at baseline and at every 3 months for determination of serum creatine kinase (CK). Potential antibodies against alglucosidase alfa will be determined at baseline, after 9 months and at the end of the study (21 months).
Measured: At baseline and every 3 months for a duration of 21 months.
Urine tetraglucoside
Urine tetraglucoside (Glc4) will be measured in micromol/millimol kreatinine.
Measured: At baseline and every 3 months for a duration of 21 months.
Assessment of treatment-emergent adverse events (TEAEs), including infusion associated reactions (IARs)
At each study visit, patients will be evaluated for new TEAEs in regard to the intervention (extension of the dosing interval, possibly followed by discontinuation of therapy). Adverse events potentially associated with (accelerated) deterioration of muscle and pulmonary function will also be evaluated. Any signs and symptoms experienced by the patient from the time of signing the informed consent through the final study visit will be documented.
Measured: At baseline and every 3 months for a duration of 21 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Oct 202431 Dec 2027

First posted28 Aug 2024
Study start1 Oct 2024 (estimated)
Primary completion31 Dec 2026 (estimated)
Study completion31 Dec 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
More trials run by Erasmus Medical CenterThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: This study is a self-controlled cohort study.
Masking: None (open label)
Enrolment: 10 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06575829
Sponsor's protocol ID: NL99999.999.99
Other IDs: 2024-514255-15-00
Acronym: TRIPOD
Status last verified: Aug 2024
First submitted: 22 Aug 2024
Last updated: 28 Aug 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Yes: All data will be shared upon a reasonable request to the PI of the study.

Interested in taking part?

Ina Barzel, MSc

+31(0)107031182 |

i.barzel@erasmusmc.nl

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See sites and contacts

RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)
Active, not recruitingPhase 3
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Pompe Disease (Late-onset)
Active, not recruitingObservational study
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Pompe Disease, Pompe Disease (Late-onset)

All Pompe Disease (Late-onset) trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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)
ClinicalTrials.gov ID: NCT06575829
Acronym / sponsor ID: TRIPOD / NL99999.999.99
Conditions: Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II, Acid Maltase Deficiency
Phase: Phase 4
Study type: Interventional
Recruitment status: Not yet recruiting
Enrolment: 10 participants (estimated)
Lead sponsor: Erasmus Medical CenterUniversity, hospital or other
Study start: 1 Oct 2024 (estimated)
Primary completion: 31 Dec 2026 (estimated)
Study completion: 31 Dec 2027 (estimated)
First posted: 28 Aug 2024 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

All eligible patients with late-onset Pompe disease will be treated with alglucosidase alfa 20 mg/kg once every 4 weeks for 9 months. During the study, patients will be monitored once every 3 months.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: This study is a self-controlled cohort study.

Arm	Type	Receives
Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks	Experimental	Drug: Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeksAlglucosidase 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.

Who may qualify

Ages: 50 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confirmed disease-causing variants in the GAA gene)
Age ≥50 years
Current treatment with alglucosidase alfa at a standard dose of 20 mg/kg once every 2 weeks for ≥4 years
Relatively stable clinical condition over the past year
Able to walk ≥150 m within 6 minutes (6MWT)

Key exclusion criteria (4 listed)

Rapidly progressive muscle weakness
Severely limited muscle strength almost requiring/requiring daily wheelchair use
Requiring respiratory support (non-invasive/invasive ventilation) or being at high risk to require respiratory support (ventilation) due to further deterioration of current pulmonary function. Using continuous positive airway pressure (CPAP) support only for obstructive sleep apnea syndrome (OSAS) is permitted.
Comorbidities which are expected to influence the primary outcome measures within the next 2 years

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeksThe 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.	Drug	Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks

Primary outcome

Changes in muscle strength and function by manual muscle testing with Medical Research Council (MRC) grading scale
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in muscle strength and function by testing hand-held dynamometry (HHD)
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in muscle strength and function by quick motor function test (QMFT)
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in muscle strength and function by 6-minute walk test (6MWT)
Time frame: Every 3 months for a duration of 21 months.
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.
Changes in pulmonary function by testing forced vital capacity (FVC) in sitting and supine positions
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in pulmonary function by testing maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP)
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in patient reported outcome measures by filling in the Rasch-built Pompe-specific activity (R-PAct) scale
Time frame: At baseline and every 3 months for a duration of 21 months.
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.
Changes in patient reported outcome measures by filling in the SF-36 questionnaire
Time frame: At baseline, after 9 months and at the end of the study (21 months).
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.
Body weight
Time frame: At baseline and every 3 months for a duration of 21 months.
Body weight will be measured in kilograms (kg).
Heart rate
Time frame: 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.
Heart rate will be measured in beats per minute (bpm).
Systolic and diastolic blood pressure
Time frame: 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 will be measured in millimeter of mercury (mmHg).
Respiratory rate
Time frame: 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.
Respiratory rate will be measured in breaths per minute.

8 secondary outcomes

Changes in lean body mass assessed by DEXA-scans
Time frame: At baseline, after 9 months and at the end of the study (21 months).
Changes in lean body mass assessed by DEXA-scans
Time frame: At baseline, after 9 months and at the end of the study (21 months).
Bone density assessed by DEXA-scans
Time frame: At baseline, after 9 months and at the end of the study (21 months).
Need for walking devices and artificial ventilation (yes/no)
Time frame: At baseline and every 3 months for a duration of 21 months.
Changes in alglucosidase alfa activity in plasma
Time frame: At 3 months and 9 months.
Serological testing of serum creatine kinase (CK) and antibodies against alglucosidase alfa
Time frame: At baseline and every 3 months for a duration of 21 months.
Urine tetraglucoside
Time frame: At baseline and every 3 months for a duration of 21 months.
Assessment of treatment-emergent adverse events (TEAEs), including infusion associated reactions (IARs)
Time frame: At baseline and every 3 months for a duration of 21 months.

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2024.
Individual participant dataSharing planned: Yes. All data will be shared upon a reasonable request to the PI of the study.

Related trials for Pompe Disease (Late-onset)

RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

Ina Barzel, MSc contact

+31(0)107031182 |

i.barzel@erasmusmc.nl

Lianne H. Potters, MSc contact

+31(0)0633342010

l.potters@erasmusmc.nl

Pieter A. van Doorn, Prof. dr. principal investigator

Erasmus Medical Center

Nadine A.M.E. van der Beek, Dr. principal investigator

Erasmus Medical Center

Tim Preijers, Dr. principal investigator

Erasmus Medical Center

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks in people with Pompe Disease (Late-onset) and GAA Deficiency, run by Erasmus Medical Center.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 28 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Ina Barzel, MSc (+31(0)107031182 |, i.barzel@erasmusmc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Changes in muscle strength and function by manual muscle testing with Medical Research Council (MRC) grading scale (time frame: At baseline and every 3 months for a duration of 21 months.). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06575829.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2024.