Trial record 5 of 10 for:    Open Studies | "Niemann-Pick Diseases"

Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency

This study is not yet open for participant recruitment.
Verified December 2013 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT02004691
First received: November 26, 2013
Last updated: December 4, 2013
Last verified: December 2013
  Purpose

The primary objective of this study is to evaluate the safety, efficacy, and pharmacokinetics of different doses of recombinant human acid sphingomyelinase (rhASM) administered intravenously every 2 weeks for 52 weeks.


Condition Intervention Phase
Sphingomyelin Lipidosis
Drug: GZ402665
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Percentage change in spleen volume [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in Liver Volume [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]
  • Participants with Pulmonary imaging and function tests [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]
  • Exercise capacity by cycle ergometry [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]
  • Hematology [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]
  • Physician Global Assessment [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]
  • Efficacy biomarkers [ Time Frame: Baseline to Week 52 ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: January 2014
Estimated Study Completion Date: October 2015
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dose 1
GZ402665 dose (0,3 mg/kg body weight) once every 2 weeks for 52 weeks
Drug: GZ402665

Pharmaceutical form:Powder for concentrate for solution for infusion

Route of administration: intravenous infusion

Experimental: Dose 2
GZ402665 dose (1,0 mg/kg body weight) once every 2 weeks for 52 weeks
Drug: GZ402665

Pharmaceutical form:Powder for concentrate for solution for infusion

Route of administration: intravenous infusion

Experimental: Dose 3
GZ402665 dose (3,0 mg/kg body weight) once every 2 weeks for 52 weeks
Drug: GZ402665

Pharmaceutical form:Powder for concentrate for solution for infusion

Route of administration: intravenous infusion


Detailed Description:

One month screening to 60 days screening from the first dose + 30 to 37 days post study safety follow up

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • Patients with documented non-neuronopathic acid sphingomyelinase deficiency
  • The patient has a diffusing capacity of carbon monoxide (DLco) >20% and ≤80% of the predicted normal value.
  • The patient has a spleen volume ≥6 multiples of normal(MN). A partial splenectomy will be permitted if performed ≥1 year prior to Screening/Baseline and residual spleen volume is ≥6 MN.
  • The patient who is female and of childbearing potential must have a negative serum pregnancy test for β-HCG.
  • Patients must practice true abstinence or use 2 effective forms of contraception

Exclusion criteria:

  • The patient is female and pregnant or breast-feeding.
  • The patient has received an investigational drug within 30 days prior to study enrollment
  • The patient has a medical condition or any extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
  • The patient has had a major organ transplant
  • ALT or AST >250 IU/L or total bilirubin >1.5 mg/dL.
  • The patient is unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each rhASM infusion for the duration of the study.
  • The patient requires medications that may decrease rhASM
  • The patient is unwilling or unable to avoid the use of medications or herbal supplements that may cause or prolong bleeding, or have potential hepatotoxicity within 10 days prior to and 3 days after liver biopsy

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT02004691

Contacts
Contact: Trial Transparency Team Contact-US@sanofi.com

Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT02004691     History of Changes
Other Study ID Numbers: DFI12712, 2010-023953-12, U1111-1142-5963
Study First Received: November 26, 2013
Last Updated: December 4, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
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
Lysosomal Storage Diseases

ClinicalTrials.gov processed this record on April 17, 2014