Trial record 7 of 26 for:    Niemann-Pick Disease

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01722526
First received: November 5, 2012
Last updated: January 16, 2014
Last verified: January 2014
  Purpose

To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.


Condition Intervention Phase
Human Acid Sphingomyelinase Deficiency
Drug: Recombinant human acid sphingomyelinase
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Summary of Adverse Events (AEs) [ Time Frame: at least 26 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics as measured by peak plasma concentration (Cmax), time to peak concentration (tmax), area under curve (AUC), half life (t1/2), drug clearance (CL), and volume of distribution (Vss) [ Time Frame: up to 26 weeks ] [ Designated as safety issue: No ]
  • Pharmacodynamics as measured by liver and skin biopsies, plasma, and dried blood spot [ Time Frame: up to 26 weeks ] [ Designated as safety issue: No ]

Enrollment: 5
Study Start Date: March 2013
Study Completion Date: January 2014
Primary Completion Date: January 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Recombinant human acid sphingomyelinase
Participants will receive rhASM of an initial dose of 0.1 mg/kg, followed by several dose escalations, as tolerated, up to 3.0 mg/kg. All doses are given 2 weeks apart.
Drug: Recombinant human acid sphingomyelinase
Administered intravenously every 2 weeks for 26 weeks
Other Name: GZ402665

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
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 receiving lipid lowering therapy should be on a stable dose and regimen of lipid-lowering therapy(ies) for at least 12 weeks prior to Screening/Baseline, with the patient expected to remain on the same dose and regimen throughout the 26-week treatment period.
  • The patient who is female and of childbearing potential must have a negative serum pregnancy test for β-HCG.

Exclusion Criteria:

  • The patient is female and pregnant or lactating.
  • The patient has a Body Mass Index(BMI)>30.
  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01722526

Locations
United States, New York
Mount Sinai School of Medicine
New York, New York, United States
United Kingdom
St. Mary's Hospital
Manchester, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01722526     History of Changes
Other Study ID Numbers: DFI13412, 2012-003542-32
Study First Received: November 5, 2012
Last Updated: January 16, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Sanofi:
Human acid sphingomyelinase deficiency

Additional relevant MeSH terms:
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
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
Sphingolipidoses
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on April 16, 2014