Ignite Creation Date:
2024-10-26 @ 3:39 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06575829
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-22
Brief Title:
Treatment Frequency Reduction in Pompe Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Open Label Single-center Pilot Study to Investigate Alglucosidase Alfa 20 mgkg Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease TRIPOD-Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
TRIPOD
Brief Summary:
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mgkg 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
Detailed Description:
All eligible patients with late-onset Pompe disease will be treated with alglucosidase alfa 20 mgkg 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 mgkg 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 eg 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 mgkg eow
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 mgkg 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 eg 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 mgkg eow
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None