A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
NCT ID: NCT02004704
Last Updated: 2024-07-03
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
25 participants
INTERVENTIONAL
2013-12-04
2023-09-06
Brief Summary
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The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.
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Detailed Description
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The maximum study duration per participant was 9 years or until olipudase alfa becomes commercially accessible. Notwithstanding the above, every pediatric participant were treated in LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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GZ402665
GZ402665 administered intravenously once every 2 weeks at the dose each participant was receiving at the end of their previous olipudase alfa study, for 9 years or until olipudase alfa becomes commercially accessible, whichever comes first, unless the participant decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible.
GZ402665
Pharmaceutical form: Powder for concentrate for solution for infusion
Route of administration: intravenous infusion
Interventions
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GZ402665
Pharmaceutical form: Powder for concentrate for solution for infusion
Route of administration: intravenous infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* The participant and/or the participant's parent(s)/legal guardian(s) was willing and able to provide signed written informed consent.
* The participant who is female and of childbearing potential must have had a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
* Female participants of childbearing potential and sexually mature male participants must have been willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug.
Exclusion Criteria
* The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study.
* The participant was unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
* The participant was unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (only participants who previously participated in the DFI13412 study).
* The participant required medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants \[eg, imipramine, desipramine\]).
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
ALL
No
Sponsors
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Genzyme, a Sanofi Company
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Sciences & Operations
Role: STUDY_DIRECTOR
Sanofi
Locations
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Investigational Site Number 840001
New York, New York, United States
Investigational Site Number 056001
Leuven, , Belgium
Investigational Site Number 076001
Porto Alegre, , Brazil
Investigational Site Number 250002
Bron, , France
Investigational Site Number 276002
Hochheim am Main, , Germany
Investigational Site Number 380002
Sassari, , Italy
Investigational Site Number 380001
Udine, , Italy
Investigational Site Number 826001
London, , United Kingdom
Investigational Site Number 826002
Manchester, , United Kingdom
Countries
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References
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Scarpa M, Diaz GA, Giugliani R, Jones SA, Mengel E, Guffon N, Witters P, Ganesh J, Armstrong NM, Srivastava S, Kim Y. Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency. J Inherit Metab Dis. 2025 Sep;48(5):e70086. doi: 10.1002/jimd.70086.
Thurberg BL, Diaz GA, Lachmann RH, Schiano T, Wasserstein MP, Ji AJ, Zaher A, Peterschmitt MJ. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020 Sep-Oct;131(1-2):245-252. doi: 10.1016/j.ymgme.2020.06.010. Epub 2020 Jun 24.
Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018 Sep;41(5):829-838. doi: 10.1007/s10545-017-0123-6. Epub 2018 Jan 5.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2013-000051-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LTS13632
Identifier Type: -
Identifier Source: org_study_id
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