Sphingomyelin LipidosisNCT04877132
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
A study testing olipudase alfa (GZ402665) in people with Sphingomyelin Lipidosis, run by Sanofi.
Trial summary
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical trials. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Sphingomyelin Lipidosis
- What is being tested?
- olipudase alfa (GZ402665)
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 3 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- All patients (adult and pediatric)
- Unsolicited request for the patient from a site with at least 3 years of experience in administration and safety management of ERT.
- Written informed consent signed by the patient or the patient's parent(s)/guardian(s), where applicable.
- Documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
- Adult patients
- Age ≥ 18 years.
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- Clinically documented advanced disease evidenced by defined thresholds for lung, spleen, liver, and hematologic parameters.
- Pediatric patients
- Age >3 years and <18 years or clinical diagnosis consistent with ASMD Type A/B or Type B.
Who cannot join
- All patients (adult and pediatric)
- Active serious intercurrent illness which will preclude enrollment, significant liver disease with etiology other than ASMD, Malignancy with poor prognosis, serious medical or psychiatric condition that may preclude participation, or circumstances that may interfere with compliance in this compassionate use program, requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
- Pregnancy or breastfeeding.
- For female patients of childbearing potential, a positive serum pregnancy (β human chorionic gonadotropin [HCG]) test result.
- For female patients of childbearing potential and sexually active male patients, unwillingness to abstain from heterosexual intercourse in accordance with their preferred and usual lifestyle, or to use 2 acceptable, effective contraceptive methods, while participating in this program and for 15 days after the last infusion of olipudase alfa.
- For pediatric patients, clinical diagnosis, or suspicion of infantile onset ASMD. Genotype compatible with ASMD type A.
About this study
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical trials. The program will provide access to olipudase alfa prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.
- Conditions
- Sphingomyelin Lipidosis
- Being tested
- olipudase alfa (GZ402665)
- Medical subject headings
- Niemann-Pick Disease, Type A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
olipudase alfa (GZ402665)Drug
Patients will receive intravenous (IV) infusion of olipudase alfa
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sphingomyelin LipidosisStudies 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
- Expanded access
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
- NCT04877132
- Sponsor's protocol ID
- RHASHC09706
- Status last verified
- Sep 2022
- First submitted
- 3 May 2021
- Last updated
- 19 Sep 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
- ClinicalTrials.gov ID
- NCT04877132
- Acronym / sponsor ID
- RHASHC09706
- Conditions
- Sphingomyelin Lipidosis
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- First posted
- 7 May 2021 (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.
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical trials. The program will provide access to olipudase alfa prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.
Study design
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- All patients (adult and pediatric)
- Unsolicited request for the patient from a site with at least 3 years of experience in administration and safety management of ERT.
- Written informed consent signed by the patient or the patient's parent(s)/guardian(s), where applicable.
- Documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
- Adult patients
Key exclusion criteria (6 listed)
- All patients (adult and pediatric)
- Active serious intercurrent illness which will preclude enrollment, significant liver disease with etiology other than ASMD, Malignancy with poor prognosis, serious medical or psychiatric condition that may preclude participation, or circumstances that may interfere with compliance in this compassionate use program, requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
- Pregnancy or breastfeeding.
- For female patients of childbearing potential, a positive serum pregnancy (β human chorionic gonadotropin [HCG]) test result.
- For female patients of childbearing potential and sexually active male patients, unwillingness to abstain from heterosexual intercourse in accordance with their preferred and usual lifestyle, or to use 2 acceptable, effective contraceptive methods, while participating in this program and for 15 days after the last infusion of olipudase alfa.
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 alfa (GZ402665)Patients will receive intravenous (IV) infusion of olipudase alfa | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2022.
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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
Questions about this trial
What is this clinical trial studying?
A study testing olipudase alfa (GZ402665) in people with Sphingomyelin Lipidosis, run by Sanofi.
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 9 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 19 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
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.
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 19 Sep 2022.