Trial · NCT05359276
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
NCT05359276, Completed, Sanofi
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France. Trials, record NCT05359276. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05359276
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
- NCT number
- NCT05359276
- Status
- Completed
- Enrollment
- 40 participants, actual
- Lead sponsor
- Sanofi
- Conditions
- Acid Sphingomyelinase Deficiency (ASMD)
- Interventions
- Olipudase alfa
- First posted
- 2022-05-03
- Last updated
- 2025-02-06
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco) | From baseline to 24 months | |
| Primary | Change in spleen size | From baseline to 24 months | |
| Primary | Change in liver size | From baseline to 24 months | |
| Secondary | Baseline patient characteristics | At baseline | Demographic and baseline data \[age, gender, weight, phenotype and genotype of ASMD, acid sphingomyelinase activity in peripheral leukocytes, lymphocytes, or cultured fibroblasts, age at diagnosis, age at first symptom onset, history of splenectomy (month/year), habits (i.e., smoking, alcoholism), known metabolic conditions or diseases (obesity, diabetes, familial dyslipidemias), known respiratory diseases; known hepatic diseases; others)\] |
| Secondary | Condition of olipudase alfa use | From baseline up to 3 years | Conditions of olipudase alfa use (time to reach maximum dose \[3 mg/kg\] or the maximum tolerated dose for the patient, center profile, treater specialty, need of a premedication before the infusion \[if yes, precise\], treatment duration \[start and end dates\], treatment discontinuation \[Yes/No\] and reason of treatment discontinuation if any) |
| Secondary | Safety: AE | From baseline up to 3 years | Number of Participants with Adverse events (AE) including infusion-associated reactions |
| Secondary | Safety: immunogenicity | From baseline up to 3 years | Immune response assessments (antibodies anti-olipudase alfa IgG) |
| Secondary | Complementary effectiveness: change in pulmonary function DLco | From baseline to 12 months and 36 months | |
| Secondary | Complementary effectiveness: change in spleen size | From baseline to 12 months and 36 months | |
| Secondary | Complementary effectiveness: change in liver size | FFrom baseline to 12 months and 36 months |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05359276
GET https://conductscience.org/api/trials/v1/NCT05359276/registered