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

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02004704
First Posted: December 9, 2013
Last Update Posted: June 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose

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: Intervention Model: Single Group Assignment
Masking: None (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 ( Genzyme, a Sanofi Company ):

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

  • Physical examinations including neurologic examinations [ Time Frame: Baseline to 5 years ]
  • Clinical laboratory tests [ Time Frame: Baseline to 5 years ]
  • Safety biomarkers [ Time Frame: Baseline to 5 years ]
  • Immune response assessments [ Time Frame: Baseline to 5 years ]
  • Vital signs, echocardiogram and electrocardiogram [ Time Frame: Baseline to 5 years ]
  • Liver biopsy [ Time Frame: Baseline to 5 years ]
    Patients previously enrolled in DFI13412

  • Liver ultrasound/Doppler [ Time Frame: Baseline to 5 years ]
    Patients previously enrolled in DFI13803


Secondary Outcome Measures:
  • Spleen and Liver Volume [ Time Frame: Baseline to 5 years ]
    Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume

  • Pulmonary imaging tests [ Time Frame: Baseline to 5 years ]
  • Pulmonary function tests [ Time Frame: Baseline to 5 years ]
  • Hematology [ Time Frame: Baseline to 5 years ]
    Hemoglobin and platelet count

  • Lipid profile [ Time Frame: Baseline to 5 years ]
  • Health Outcomes Questionnaires [ Time Frame: Baseline to 5 years ]
  • Hand X-ray for bone age and bone maturation [ Time Frame: Baseline to 5 years ]
    Pediatric patients

  • Tanner staging [ Time Frame: Baseline to 5 years ]
    Pediatric patients

  • Linear patient growth by height Z-score [ Time Frame: Baseline to 5 years ]
    Pediatric patients


Estimated Enrollment: 17
Study Start Date: December 2013
Estimated Study Completion Date: October 2021
Estimated Primary Completion Date: October 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).
  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02004704


Locations
United States, New York
Investigational Site Number 840001
New York, New York, United States, 10029-6574
France
Investigational Site Number 250002
Bron Cedex, France, 69677
Italy
Investigational Site Number 380001
Udine, Italy, 33100
United Kingdom
Investigational Site Number 826001
London, United Kingdom, WC1N 3JZ
Investigational Site Number 826002
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02004704     History of Changes
Other Study ID Numbers: LTS13632
2013-000051-40 ( EudraCT Number )
U1111-1141-65868 ( Other Identifier: (UTN) )
First Submitted: November 26, 2013
First Posted: December 9, 2013
Last Update Posted: June 2, 2017
Last Verified: June 2017

Additional relevant MeSH terms:
Niemann-Pick Disease, Type A
Niemann-Pick Diseases
Niemann-Pick Disease, Type C
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lymphatic Diseases
Lysosomal Storage Diseases
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Sphingolipidoses
Histiocytosis, Non-Langerhans-Cell
Histiocytosis