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

• Register: EUCTR
• Last refreshed on: 22 December 2015
• Main ID: EUCTR2010-023953-12-IT
• Date of registration: 24/09/2013
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation
• Public title: Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency
• Scientific 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 - ASCEND
• Date of first enrolment: 28/01/2014
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-023953-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 3

Countries of recruitment

Brazil, Chile, France, Germany, Italy, Tunisia, United Kingdom, United States

Contacts

• Name: Medical Information
• Affiliation: Genzyme Europe B.V.

• Name: Medical Information
• Affiliation: Genzyme Europe B.V.

Key inclusion & exclusion 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 ß-HCG.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
• The patient is female and pregnant or lactating.
• 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 16.0 Level: LLT Classification code 10041515 Term: Sphingomyelin lipidosis System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: rhASM
Product Code: GZ402665
Pharmaceutical Form: Powder for concentrate for solution for infusion
CAS Number: 927883-84-9
Current Sponsor code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Primary end point(s): Percentage change in spleen volume

Secondary Objective:

Main Objective: The primary objective is to evaluate the safety, efficacy, and pharmacokinetics of different doses of rhASM administered intravenously (IV) every 2 weeks for 52 weeks.

Timepoint(s) of evaluation of this end point: Baseline to end of study

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Baseline to end of study

Secondary end point(s): Liver volume
Pulmonary imaging and function testing
Exercise capacity by cycle ergometry
Hematology
Physician Global Assessment
Efficacy biomarkers

Secondary ID(s)

DFI12712

2010-023953-12-GB

Source(s) of Monetary Support

Genzyme Corporation