CompletedPhase 2

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

United States, Belgium, Brazil25 participantsStarted 2013-12-04

Plain-language summary

The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

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 completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor. * The participant and/or the participant's parent(s)/legal guardian(s) was willing and able to provide signed written informed consent. * The participant who is female and of childbearing potential must have had a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG). * Female participants of childbearing potential and sexually mature male participants must have been willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug. Exclusion criteria: * The participant had any new condition or worsening of an existing condition which in the opinion of the investigator would make the participant unsuitable for enrollment, or could interfere with the participation or completion the study. * The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study. * The participant was unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period. * The participant was unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatoto…

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

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs)

Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first

Number of Participants With IARs

Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first

For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam

Timeframe: Baseline (Day 1 of original study), Month 78

For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam

Timeframe: Baseline (Day 1 of original study), Month 84

For Adults: Number of Participants With Abnormality in Extended Neurological Examinations

Timeframe: Baseline (Day 1 of original study), Month 78

For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations

Trial details

NCT IDNCT02004704

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-09-06

Results submitted2024-04-23

View on ClinicalTrials.gov More Sphingomyelin Lipidosis trials

Plain-language summary

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.

What they're measuring

Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first

Number of Participants With IARs

Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first

For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam

Timeframe: Baseline (Day 1 of original study), Month 78

For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam

Timeframe: Baseline (Day 1 of original study), Month 84

For Adults: Number of Participants With Abnormality in Extended Neurological Examinations

Timeframe: Baseline (Day 1 of original study), Month 78

For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations