Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
3
Countries
1
Sites
2
Niemann-Pick disease
The objective of this study is to assess safety and tolerability of olipudase alfa in patients with ASMD who have completed the DFI12712 or LTS13632 study in France until olipudase alfa reimbursement is granted in France or until 5 years whichever comes first.
Key facts
- Sponsor
- Sanofi Winthrop Industrie
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 18 Nov 2021 → 8 Apr 2025
- Decision date (initial)
- 2024-08-02
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Sanofi Winthrop Industrie
External identifiers
- EU CT number
- 2024-515304-39-00
- EudraCT number
- 2021-004109-39
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
The objective of this study is to assess safety and tolerability of olipudase alfa in patients with ASMD who have completed the DFI12712 or LTS13632 study in France until olipudase alfa reimbursement is granted in France or until 5 years whichever comes first.
Conditions and MedDRA coding
Niemann-Pick disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10029403 | Niemann-Pick disease | 100000004850 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- The patient has completed Study the DFI12712 (ASCEND) or LTS13632 in France
- The patient must provide signed, informed consent prior to performing any study related procedures.
- The patient is willing to comply with the clinical protocol.
- The patient, if female and of childbearing potential, must have a negative pregnancy test result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
- Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the treatment period and for at least 28 days after receiving the last study drug dose.
Exclusion criteria 7
- Any patient who has not participated in the Study DFI12712 or the Study LTS13632
- A patient who experienced any systemic hypersensitivity reactions to olipudase alfa in Study DFI12712 or Study LTS13632 which, in the opinion of the Investigator, could indicate that treatment continuation may present an unreasonable risk.
- The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
- The patient is unwilling or unable to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
- Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized
- The patient is concurrently participating in another clinical study of investigational treatment
- Any of the following medical conditions: - The patient has any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the patient unsuitable for enrollment or could interfere with the patient’s participating in or completing the study. - Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months. - Pregnancy or breastfeeding.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Number of adverse events (AEs) / serious adverse events (SAEs)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Xenpozyme (olipudase alfa,GZ402665)
PRD11449283 · Product
- Active substance
- Olipudase Alfa
- Substance synonyms
- GZ-402665, RECOMBINANT HUMAN ACID SPHINGOMYELINASE
- Pharmaceutical form
- POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 3 mg/kg milligram(s)/kilogram
- Max total dose
- 360 mg/kg milligram(s)/kilogram
- Max treatment duration
- 60 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- GENZYME CORPORATION
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/01/056
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Sanofi Winthrop Industrie
- Sponsor organisation
- Sanofi Winthrop Industrie
- Address
- 82 Avenue Raspail
- City
- Gentilly
- Postcode
- 94250
- Country
- France
Scientific contact point
- Organisation
- Sanofi Winthrop Industrie
- Contact name
- Clinical Sciences and Operations
Public contact point
- Organisation
- Sanofi Winthrop Industrie
- Contact name
- Clinical Sciences and Operations
Third parties 3
| Organisation | City, country | Duties |
|---|---|---|
| Unite Paramedicale Ambulatoire De Recherche Clinique ORG-100050242
|
Paris, France | Other |
| ICTA Project Management En Abrege ICTA P.M. ORG-100008364
|
Fontaine Les Dijon, France | Data management |
| ESMS Global Limited ORG-100023149
|
London, United Kingdom | Other |
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 3 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2021-11-18 | 2025-04-08 | 2021-11-18 | 2022-09-20 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| pta17397-summary-results SUM-100199
|
2025-10-06T10:54:41 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| pta17397-lay-summary-en | 2025-10-06T10:55:10 | Submitted | Laypersons Summary of Results |
| pta17397-lay-summary-fr | 2025-10-06T10:54:58 | Submitted | Laypersons Summary of Results |
Documents 11 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | pta17397-lay-summary-en | 1 |
| Laypersons summary of results (for publication) | pta17397-lay-summary-fr | 1 |
| Protocol (for publication) | d1-protocol-en-2024-515304-39 | 1 |
| Recruitment arrangements (for publication) | K1-recruitment-arrangements-en-waiver | 1 |
| Subject information and informed consent form (for publication) | L1-sis-icf-main-addendum1-fr | 1.1 |
| Subject information and informed consent form (for publication) | L1-sis-icf-main-fr | 2 |
| Subject information and informed consent form (for publication) | L1-sis-icf-parents-addendum1-fr | 1.1 |
| Subject information and informed consent form (for publication) | L1-sis-icf-parents-fr | 1 |
| Subject information and informed consent form (for publication) | L2-other-subject-information-material-child-leaflet-fr | 1 |
| Summary of results (for publication) | pta17397-summary-results | 1 |
| Synopsis of the protocol (for publication) | d1-lay-protocol-synopsis-en-2024-515304-39 | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-19 | France | Acceptable 2024-07-30
|
2024-08-02 |