Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06614894
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-28
Brief Title:
An Open Label Dose Escalation Study to Assess the Safety Tolerability and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult 18 Years of Age Subjects With MPS III
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open Label Dose Escalation Study to Assess the Safety Tolerability and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult 18 Years of Age Subjects With MPS III
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
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:
None
Brief Summary:
A dose escalation study to evaluate the safety tolerability and pharmacologic properties of Ambroxol in adult participants with Sanfilippo diseases MPS3
Detailed Description:
This is a dose escalation study in which open label Ambroxol 30mg study drug will be administered to adult patients with Sanfilippo Disease MPS3 Administration route of study drug will be either crushed and mixed with soft foods or as an Ambroxol suspension through a feeding tube if applicable
Study Timeline - Screening 4 weeks 28 days Treatment Period 52 weeks Post-Treatment after Week 52 4 weeks withdrawalsafety follow-up period
Patients will be screened at which point a thorough review of the Informed Consent form will be completed sNFL levels urinary GAGs and serum HS resultsdata from the previous 12 months will be reviewed urine and blood will be collected complete questionnaires Motor skills assessments and evaluation by the Principal Investigator PI
Eligible patients will proceed to receive the initial Ambroxol dose of 9mgkgday maximum dose of 150 mg TID divided into three equal doses per day on-site Other assessments including blood and urine collection ECG motor skills assessments hearing test questionnaires and evaluation by the PI will be completed
Following the first day of dosing a virtual visit will be performed via Telemedicine within 1 week of dose start to assess safety
At Weeks 12 and 24 enrolled patients will return to site for Ambroxol dose escalation to 18mgkgday max dose of 300 mg TID and 27mgkgday max dose of 1350 mgday respectively Assessments including blood and urine collection ECG motor skills assessments hearing test questionnaires and evaluation by the PI will be completed at these visits
Telemedicine visits will take place at Weeks 13 and 25 to assess safety
At Week 36 a safety visit will be performed in which blood and urine will be collected
At Week 52 end of study assessments will be completed which includes blood and urine collection motor skills assessments hearing test questionnaires and evaluation by the PI will be conducted and treatment will be stopped
A safety follow-up visit will be done 4 weeks after Week 52 visit is completed in which the patient will be evaluated by the PI
Study Timeline - Screening 4 weeks 28 days Treatment Period 52 weeks Post-Treatment after Week 52 4 weeks withdrawalsafety follow-up period
Patients will be screened at which point a thorough review of the Informed Consent form will be completed sNFL levels urinary GAGs and serum HS resultsdata from the previous 12 months will be reviewed urine and blood will be collected complete questionnaires Motor skills assessments and evaluation by the Principal Investigator PI
Eligible patients will proceed to receive the initial Ambroxol dose of 9mgkgday maximum dose of 150 mg TID divided into three equal doses per day on-site Other assessments including blood and urine collection ECG motor skills assessments hearing test questionnaires and evaluation by the PI will be completed
Following the first day of dosing a virtual visit will be performed via Telemedicine within 1 week of dose start to assess safety
At Weeks 12 and 24 enrolled patients will return to site for Ambroxol dose escalation to 18mgkgday max dose of 300 mg TID and 27mgkgday max dose of 1350 mgday respectively Assessments including blood and urine collection ECG motor skills assessments hearing test questionnaires and evaluation by the PI will be completed at these visits
Telemedicine visits will take place at Weeks 13 and 25 to assess safety
At Week 36 a safety visit will be performed in which blood and urine will be collected
At Week 52 end of study assessments will be completed which includes blood and urine collection motor skills assessments hearing test questionnaires and evaluation by the PI will be conducted and treatment will be stopped
A safety follow-up visit will be done 4 weeks after Week 52 visit is completed in which the patient will be evaluated by the PI
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