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A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2016 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: November 26, 2013
Last updated: October 12, 2016
Last verified: October 2016

The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa.

The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

Condition Intervention Phase
Sphingomyelin Lipidosis
Drug: GZ402665
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Adverse events and treatment emergent adverse events including infusion-associated reactions [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
    Number of patients experiencing Adverse Events

  • Physical examinations including neurologic examinations [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
  • Clinical laboratory tests [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
  • Safety biomarkers [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
  • Immune response assessments [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
  • Vital signs, echocardiogram and electrocardiogram [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
  • Liver biopsy [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
    Patients previously enrolled in DFI13412

  • Liver ultrasound/Doppler [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: Yes ]
    Patients previously enrolled in DFI13803

Secondary Outcome Measures:
  • Spleen and Liver Volume [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
    Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume

  • Pulmonary imaging tests [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
  • Pulmonary function tests [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
  • Hematology [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
    Hemoglobin and platelet count

  • Lipid profile [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
  • Health Outcomes Questionnaires [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
  • Hand X-ray for bone age and bone maturation [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
    Pediatric patients

  • Tanner staging [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
    Pediatric patients

  • Linear patient growth by height Z-score [ Time Frame: Baseline to 5 years ] [ Designated as safety issue: No ]
    Pediatric patients

Estimated Enrollment: 17
Study Start Date: December 2013
Estimated Study Completion Date: August 2021
Estimated Primary Completion Date: August 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GZ402665
GZ402665 (olipudase alfa) administered intravenously once every 2 weeks for up to 5 years at the dose each patient was receiving at the end of their previous olipudase alfa study.
Drug: GZ402665

Pharmaceutical form: Powder for concentrate for solution for infusion

Route of administration: intravenous infusion

Other Name: Olipudase alfa

Detailed Description:
5 years: This study is an extension study for patients who have completed a previous study with olipudase alfa (DFI13803 for pediatric patients, and DFI13412 for adult patients).

Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion criteria :

  • The patient completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor.
  • The patient and/or the patient's parent(s)/legal guardian(s) is willing and able to provide signed written informed consent.
  • The patient who is female and of childbearing potential must have a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
  • Female patients of childbearing potential and sexually mature male patients must be 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 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 participating in or completing the study.
  • 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 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 patient is unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (eg, 3 hydroxy 3 methylglutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, antidepressants, kava, echinacea) or may cause or prolong bleeding (eg, anticoagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).
  • The patient requires medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
  • The patient has an alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02004704

Contact: For site information, send an email with site number to

United States, New York
Investigational Site Number 840001 Recruiting
New York, New York, United States, 10029-6574
Investigational Site Number 380001 Recruiting
Udine, Italy, 33100
United Kingdom
Investigational Site Number 826001 Recruiting
London, United Kingdom, WC1N 3JZ
Investigational Site Number 826002 Recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT02004704     History of Changes
Other Study ID Numbers: LTS13632  2013-000051-40  U1111-1141-65868 
Study First Received: November 26, 2013
Last Updated: October 12, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Niemann-Pick Disease, Type A
Niemann-Pick Diseases
Niemann-Pick Disease, Type C
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lymphatic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Histiocytosis, Non-Langerhans-Cell
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors processed this record on October 21, 2016