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

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.
 
ClinicalTrials.gov Identifier: NCT02004704
Recruitment Status : Active, not recruiting
First Posted : December 9, 2013
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:

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 or disease Intervention/treatment Phase
Sphingomyelin Lipidosis Drug: GZ402665 Phase 2

Expanded Access : An investigational treatment associated with this study has been approved for sale to the public.   More info ...

Detailed Description:

The maximum study duration per patient is 9 years or until olipudase alfa becomes commercially accessible (see maximum duration below), whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible. The term "commercially accessible" is defined as when olipudase alfa is commercially accessible to each patient on an individual basis (eg, reimbursement being in place). The duration of study treatment with olipudase alfa between the local Regulatory approval and commercial accessibility should not exceed 90 days. Therefore, as described below, after local Regulatory approval, the patient can continue in the LTS13632 study for a maximum of 127 days. This will ensure 90 days of study treatment with olipudase alfa for patients after local Regulatory approval and a safety follow up phone call 30 to 37 days after the last dose of study treatment.

Notwithstanding the above, every pediatric patient will be treated in the LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.

The patient can switch immediately after the end of study treatment to commercial treatment without any gap in order to ensure continuity of treatment with olipudase alfa.

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).

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
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
Actual Study Start Date : December 4, 2013
Estimated Primary Completion Date : February 29, 2024
Estimated Study Completion Date : February 29, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: GZ402665
GZ402665 administered intravenously once every 2 weeks at the dose each patient 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 patient decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible.
 Drug: GZ402665

Pharmaceutical form: Powder for concentrate for solution for infusion

Route of administration: intravenous infusion

Other Name: Olipudase alfa


Outcome Measures
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Primary Outcome Measures :
  1. Adverse events (AEs)/treatment-emergent adverse events (TEAEs), including infusion-associated reactions and adverse events of special interest (AESIs) [ Time Frame: Baseline to up to 9 years ]
    Number of patients experiencing AEs, TEAEs, or AESIs

  2. Complete physical examinations including extended neurologic and abbreviated physical exams [ Time Frame: Baseline to up to 9 years ]
  3. Vital signs, electrocardiograms and echocardiograms with Doppler [ Time Frame: Baseline to up to 9 years ]
  4. Clinical laboratory tests [ Time Frame: Baseline to up to 9 years ]
  5. Safety biomarkers [ Time Frame: Baseline to up to 9 years ]
  6. Liver biopsy (patients previously enrolled in DFI13412) [ Time Frame: Baseline to after at least 3 years in the study ]
  7. Liver ultrasound/Doppler (patients previously enrolled in DFI13803) [ Time Frame: Baseline to 5 years ]
  8. Immune response assessments [ Time Frame: Baseline to up to 9 years ]

Secondary Outcome Measures :
  1. Spleen and liver volume [ Time Frame: Baseline to up to 9 years ]
    Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume

  2. Pulmonary imaging [ Time Frame: Baseline to up to 9 years ]
  3. Pulmonary function test [ Time Frame: Baseline to up to 9 years ]
  4. Hematology [ Time Frame: Baseline to up to 9 years ]
    (hemoglobin and platelet count)

  5. Lipid profile [ Time Frame: Baseline to up to 9 years ]
  6. Health outcome questionnaires (adults and pediatric) [ Time Frame: Baseline to up to 9 years ]
  7. Hand X ray for bone age and bone maturation (pediatric patients) [ Time Frame: Baseline to up to 9 years ]
  8. Linear patient growth by height Z -score (pediatric patients) [ Time Frame: Baseline to up to 9 years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
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) (only patients who previously participated in the DFI13412 study).
  • The patient requires medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).

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

Contacts and Locations
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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
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United States, New York
Investigational Site Number 840001
New York, New York, United States, 10029-6574
Belgium
Investigational Site Number 056001
Leuven, Belgium, 3000
Brazil
Investigational Site Number 076001
Porto Alegre, Brazil, 90035 003
France
Investigational Site Number 250002
Bron Cedex, France, 69677
Germany
Investigational Site Number 276002
Hochheim Am Main, Germany, 65239
Italy
Investigational Site Number 380002
Sassari, Italy, 07100
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
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Study Director: Clinical Sciences & Operations Sanofi
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02004704    
Other Study ID Numbers: LTS13632
2013-000051-40 ( EudraCT Number )
U1111-1141-65868 ( Other Identifier: (UTN) )
First Posted: December 9, 2013    Key Record Dates
Last Update Posted: June 6, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Additional relevant MeSH terms:
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Niemann-Pick Disease, Type A
Niemann-Pick Diseases
Niemann-Pick Disease, Type C
Lipidoses
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders