Olipudase Alfa for Niemann-Pick Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the long-term safety of olipudase alfa, an enzyme replacement therapy, for young children with acid sphingomyelinase deficiency (ASMD), a rare genetic disorder that can cause organ damage. The trial observes how well olipudase alfa works and whether it causes any side effects over several years. Children under 2 years old with ASMD type A/B or B, or of any age with type A, who weigh at least 2 kg and have started or are about to start this treatment may be eligible. The study gathers real-world data without altering standard treatments.
As a Phase 4 trial, this study seeks to understand how the already FDA-approved treatment benefits more patients, offering a chance to contribute to valuable research.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, you cannot have taken an investigational drug within 30 days before joining the study.
What is the safety track record for olipudase alfa?
Research has shown that olipudase alfa is generally safe for children. Common side effects include fever, cough, diarrhea, runny nose, stomach pain, and vomiting, occurring in about 20% or more of patients.
Long-term studies have indicated that olipudase alfa is usually safe for up to two years of treatment. Most side effects are mild to moderate, but serious allergic reactions, though less common, can occur.
The FDA has also approved this treatment for other conditions, suggesting it is reasonably safe. However, individual experiences with any treatment can vary.12345Why are researchers excited about this trial?
Olipudase alfa is unique because it directly targets the root cause of Acid Sphingomyelinase Deficiency (ASMD) by replacing the deficient enzyme in patients. Unlike existing treatments that might only ease symptoms, olipudase alfa provides a more direct approach by addressing the enzyme deficiency itself. This treatment is especially exciting for researchers as it has the potential to improve the underlying condition rather than just managing its symptoms, which could lead to significant improvements in patient health and quality of life.
What evidence suggests that this trial's treatment could be effective for ASMD?
Research has shown that olipudase alfa, which participants in this trial may receive, can help manage symptoms of acid sphingomyelinase deficiency (ASMD). In one study, 64% of adults reported significant satisfaction with symptom improvement. Another study found that it significantly improved lung function and reduced spleen size. Specifically, spleen size decreased by nearly 40% in patients who took olipudase alfa compared to those who took a placebo. These results suggest that olipudase alfa is a promising treatment for managing ASMD.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Enrollment
Participants are enrolled in the study over a period of up to 4 years
Follow-up
Participants are monitored for safety and immunogenicity after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Olipudase alfa
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University
Pulse Infoframe Ltd.
Collaborator
Pulse Infoframe Ltd.
Industry Sponsor
Citations
Real-life impacts of olipudase alfa: experiences of adults ...
64% were extremely satisfied with its management of ASMD symptoms, whilst 27% were somewhat satisfied and 9% were somewhat dissatisfied.
Long-term safety and clinical outcomes of olipudase alfa ...
Olipudase alfa was associated with clinically significant improvements in disease pathology and multiple endpoints compared with placebo in ...
Efficacy in Adults | XENPOZYME® (olipudase alfa-rpcp)
†First post-dose assessment. XENPOZYME arm: Baseline mean: 49.1% predicted DLco; Week 52 mean: 59.4% predicted DLco. Placebo arm:
4.
news.sanofi.us
news.sanofi.us/2020-10-27-Olipudase-alfa-demonstrated-significant-improvement-in-lung-function-and-spleen-volume-in-patients-with-ASMDOlipudase alfa demonstrated significant improvement in ...
Olipudase alfa significantly reduced spleen volume by 39.5% compared with a 0.5% increase in the placebo arm (p<0.0001), as assessed by percent ...
Study Details | NCT02004691 | Efficacy, Safety, ...
To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures assessed sequentially: The effect of ...
Safety Results in Pediatric Patients
Most frequently reported adverse drug reactions in pediatric patients (incidence ≥20%) were pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, ...
Olipudase alfa-rpcp (Xenpozyme): A Clinical Dose ...
Patient data from early phase tolerability and pharmacokinetic/ pharmacodynamic (PK/PD) studies can be used to inform safety and subsequent dosing strategies. • ...
Long-term safety and clinical outcomes of olipudase alfa ...
Olipudase alfa was generally well-tolerated during 2 years of treatment. Improvements in clinically relevant disease endpoints observed during ...
Xenpozyme | European Medicines Agency (EMA)
In terms of safety, Xenpozyme's side effects are generally mild to moderate. More serious side effects, in particular severe allergic reactions, can occur but ...
Safety Results in Adults
Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis.
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