Trial · NCT05359276
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| 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 | |
| Secondary | Change in interstitial pulmonary infiltration based on lung imaging (thoracic CT-scan) | From baseline to 12 months and 24 months | |
| Secondary | Change in platelet count | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years | |
| Secondary | Change in biomarkers (chitotriosidase and lysosphingomyelin) plasma levels | From baseline at 3, 6, 9, 12 months and every year up to 3 years | |
| Secondary | Change in liver function | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years | Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), total and direct bilirubin |
| Secondary | Change in lipid profile | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years | total cholesterol, high density lipoprotein (HDL) and low density lipoprotein (LDL) cholesterol |
| Secondary | Change in growth curve for pediatric patient | From baseline at 6, 12, 24 months and every year up to 3 years | |
| Secondary | Change in weight | From baseline to 12 months and 36 months | |
| Secondary | Number of Participants with Evolution of Comorbidities | From baseline to 12 months, 24 months and 36 months | Number of participants with evolution of comorbidities will be assessed by grade, attenuation or disappearance/absence |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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
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GET https://conductscience.org/api/trials/v1/NCT05359276/registered