Acid Sphingomyelinase Deficiency (ASMD)OPERANCT05359276
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
An observational study of Acid Sphingomyelinase Deficiency (ASMD), run by Sanofi at 1 site in France.
Trial summary
Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: To describe the patient's characteristics To describe conditions of olipudase alfa use To describe safety data related to the use of olipudase alfa To describe complementary effectiveness outcomes parameters Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Acid Sphingomyelinase Deficiency (ASMD)
- What is being tested?
- Olipudase alfa
- How many people
- 40 participants
- Where
- 1 siteFrance
- Study dates
- 10 Jun 2022to 31 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (with a written informed consent)
Who may be able to join
- The patient, or the patient's parent(s)/guardian(s), has signed written informed consent.
- Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (ie, nominative compassionate use, pre marketing authorization early access, post marketing authorization early access).
- The patient has documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
- Male and female patients of all ages.
Who cannot join
- The patient or legal guardian(s) who has not received information notice or who opposes to data collection.
- Patient who died before study initiation and who was opposed to data collection for research purpose when he/she was alive.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France
Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
- To describe the patient's characteristics
- To describe conditions of olipudase alfa use
- To describe safety data related to the use of olipudase alfa
- To describe complementary effectiveness outcomes parameters
Read the detailed description
Approximate duration of enrollment: 30 months
Total study duration: approximately 30 months
This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.
- Conditions
- Acid Sphingomyelinase Deficiency (ASMD)
- Being tested
- Olipudase alfa
- Medical subject headings
- Niemann-Pick Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Olipudase alfaDrug
GZ402665
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France Receives: Drug: Olipudase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)
Measured: From baseline to 24 months
Change in spleen size
Measured: From baseline to 24 months
Change in liver size
Measured: From baseline to 24 months
Secondary outcomes (15)
Baseline patient characteristics
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)]
Measured: At baseline
Condition of olipudase alfa use
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)
Measured: From baseline up to 3 years
Safety: AE
Number of Participants with Adverse events (AE) including infusion-associated reactions
Measured: From baseline up to 3 years
Safety: immunogenicity
Immune response assessments (antibodies anti-olipudase alfa IgG)
Measured: From baseline up to 3 years
Complementary effectiveness: change in pulmonary function DLco
Measured: From baseline to 12 months and 36 months
Complementary effectiveness: change in spleen size
Measured: From baseline to 12 months and 36 months
Complementary effectiveness: change in liver size
Measured: FFrom baseline to 12 months and 36 months
Change in interstitial pulmonary infiltration based on lung imaging (thoracic CT-scan)
Measured: From baseline to 12 months and 24 months
Change in platelet count
Measured: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in biomarkers (chitotriosidase and lysosphingomyelin) plasma levels
Measured: From baseline at 3, 6, 9, 12 months and every year up to 3 years
Change in liver function
Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), total and direct bilirubin
Measured: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in lipid profile
total cholesterol, high density lipoprotein (HDL) and low density lipoprotein (LDL) cholesterol
Measured: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in growth curve for pediatric patient
Measured: From baseline at 6, 12, 24 months and every year up to 3 years
Change in weight
Measured: From baseline to 12 months and 36 months
Number of Participants with Evolution of Comorbidities
Number of participants with evolution of comorbidities will be assessed by grade, attenuation or disappearance/absence
Measured: From baseline to 12 months, 24 months and 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2022
- Study start10 Jun 2022
- Primary completion31 Dec 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acid Sphingomyelinase Deficiency (ASMD)Studies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05359276
- Sponsor's protocol ID
- OBS17422
- Acronym
- OPERA
- Status last verified
- Feb 2025
- First submitted
- 28 Apr 2022
- Last updated
- 6 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France
- ClinicalTrials.gov ID
- NCT05359276
- Acronym / sponsor ID
- OPERA / OBS17422
- Conditions
- Acid Sphingomyelinase Deficiency (ASMD)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 10 Jun 2022 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 3 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
- To describe the patient's characteristics
- To describe conditions of olipudase alfa use
- To describe safety data related to the use of olipudase alfa
- To describe complementary effectiveness outcomes parameters
Detailed description
Approximate duration of enrollment: 30 months
Total study duration: approximately 30 months
This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: Olipudase alfaPatients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (with a written informed consent)
Key inclusion criteria (4 listed)
- The patient, or the patient's parent(s)/guardian(s), has signed written informed consent.
- Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (ie, nominative compassionate use, pre marketing authorization early access, post marketing authorization early access).
- The patient has documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
- Male and female patients of all ages.
Key exclusion criteria (3 listed)
- The patient or legal guardian(s) who has not received information notice or who opposes to data collection.
- Patient who died before study initiation and who was opposed to data collection for research purpose when he/she was alive.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Olipudase alfaGZ402665 | Drug | Cohort 1 |
Primary outcome
Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)
Time frame: From baseline to 24 months
Change in spleen size
Time frame: From baseline to 24 months
Change in liver size
Time frame: From baseline to 24 months
15 secondary outcomes
Baseline patient characteristics
Time frame: At baseline
Condition of olipudase alfa use
Time frame: From baseline up to 3 years
Safety: AE
Time frame: From baseline up to 3 years
Safety: immunogenicity
Time frame: From baseline up to 3 years
Complementary effectiveness: change in pulmonary function DLco
Time frame: From baseline to 12 months and 36 months
Complementary effectiveness: change in spleen size
Time frame: From baseline to 12 months and 36 months
Complementary effectiveness: change in liver size
Time frame: FFrom baseline to 12 months and 36 months
Change in interstitial pulmonary infiltration based on lung imaging (thoracic CT-scan)
Time frame: From baseline to 12 months and 24 months
Change in platelet count
Time frame: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in biomarkers (chitotriosidase and lysosphingomyelin) plasma levels
Time frame: From baseline at 3, 6, 9, 12 months and every year up to 3 years
Change in liver function
Time frame: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in lipid profile
Time frame: From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years
Change in growth curve for pediatric patient
Time frame: From baseline at 6, 12, 24 months and every year up to 3 years
Change in weight
Time frame: From baseline to 12 months and 36 months
Number of Participants with Evolution of Comorbidities
Time frame: From baseline to 12 months, 24 months and 36 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2025.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Related trials for Acid Sphingomyelinase Deficiency (ASMD)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acid Sphingomyelinase Deficiency (ASMD), run by Sanofi at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco) (time frame: From baseline to 24 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2025.