A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants… (NCT06949358) | Clinical Trial Compass
CompletedPhase 2
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
France3 participantsStarted 2021-11-18
Plain-language summary
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement was granted in France.
Study and treatment duration:
The period between the participant's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement was available in France.
In case reimbursement was not obtained, this study ended 5 years after starting.
Visit frequency: every 2 weeks.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* The participant has completed Study DFI12712 (ASCEND) or LTS13632 in France
* The participant must provide signed, informed consent prior to performing any study-related procedures.
* The participant was willing to comply with the clinical protocol.
* The participant, if female and of childbearing potential, must have had a negative pregnancy test result \[urine beta-human chorionic gonadotropin (β-HCG)\] at enrollment.
* Sexually active female participants of childbearing potential and male participants were required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the treatment period and for at least 28 days after receiving the last study drug dose.
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
* Any participant who has not participated in the Study DFI12712 or the Study LTS13632
* A participant who experienced any systemic hypersensitivity reactions to olipudase alfa in Study DFI12712 or Study LTS13632 which, in the opinion of the Investigator, could indicate that treatment continuation may present an unreasonable risk.
* The participant, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
* The participant was unwilling or unable to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the tr…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timeframe: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months