Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency
- Conditions
- Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)MedDRA version: 20.1Level: LLTClassification code 10041515Term: Sphingomyelin lipidosisSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2015-000371-26-DE
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 36
-The patient is willing and able to provide signed written informed consent.
-The patient is male or female aged 18 years or older.
-The patient has documented deficiency of acid sphingomyelinase as
measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes;
and a clinical diagnosis consistent with Niemann-Pick disease type B
(NPD B).
-The patient has diffuse capacity of the lung for carbon monoxide =70%
of the predicted normal value.
-The patient has a spleen volume =6 multiples of normal (MN) measured
by MRI; patients who have had partial splenectomy will be allowed if the
procedure was performed =1 year before screening/baseline and the
residual spleen volume is =6 MN.
-The patient has an SRS =5..
-Female patients of childbearing potential must have a negative serum
pregnancy test for beta-human chorionic gonadotropin (ß-HCG).
-Female patients of childbearing potential and 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 for up
to 15 days following their last dose of study drug.
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 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
-The patient has received an investigational drug within 30 days before study enrollment.
-The patient has a medical condition, including significant intercurrent illness; significant cardiac disease (e.g., clinically significant arrhythmia,
moderate or severe pulmonary hypertension or clinically significant valve dysfunction, or <40% left ventricular ejection fraction by echocardiogram); active hepatitis B or hepatitis C, or infection with human immunodeficiency virus (HIV); malignancy diagnosed within the
past 5 years (other than non-melanoma skin cancer), or any other serious medical condition that may preclude participation in the study.
-The patient has a platelet count <60,000/µL based on the average of 2 samples.
-The patient has an international normalized ratio (INR) >1.5.
-The patient has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL (except for patients with Gilbert's syndrome).
-The patient has had a major organ transplant (eg, bone marrow or liver).
-The patient is scheduled during the study for in-patient hospitalization including elective surgery and excluding the liver biopsies required per
protocol.
-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 before and 3 days after each study drug infusion. Testing for blood alcohol levels will not be required.
-The patient is unwilling or unable to avoid 10 days before and 3 days after the protocol scheduled liver biopsies the use of medications or
herbal supplements that are potentially hepatotoxic (eg, 3-hydroxy-3-methyl glutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, anti-depressants, kava, echinacea) and/or may cause or prolong bleeding (eg, anti-coagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).
-The patient requires medications that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine, tricyclic antidepressants [eg,
imipramine, or desipramine]).
-The patient requires use of invasive ventilatory support.
-The patient requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
-The patient is breast-feeding.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method