A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency

• Register: EUCTR
• Last refreshed on: 12 March 2024
• Main ID: EUCTR2013-000051-40-DE
• Date of registration: 01/10/2018
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation
• Public title: A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency
• Scientific title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
• Date of first enrolment: 14/11/2018
• Target sample size: 25
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-000051-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Brazil, France, Germany, Italy, United Kingdom, United States

Contacts

• Affiliation: Sanofi-Aventis Deutschand GmbH

• Affiliation: Sanofi-Aventis Deutschand GmbH

Key inclusion & exclusion 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 (ß 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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]).



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: 20.1 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: Olipudase alfa (rhASM)
Product Code: GZ402665
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: Olipudase alfa
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)

Timepoint(s) of evaluation of this end point: From 1/ to 5/ and 8/: Time Frame: Baseline to up to 9 years:

6/: Time Frame: Baseline to after at least 3 years in the study

7/ Baseline to 5 years for liver ultrasound/doppler

Main Objective: 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

Secondary Objective: 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

Primary end point(s): 1/ AE/TEAEs, including infusion-associated reactions and AESIs.
2/ Complete physical examinations including extended neurologic,
weight, height (pediatric patients only) and abbreviated physical exams.
3/ Vital signs, echocardiograms and electrocardiograms with Doppler.
4/ Clinical laboratory tests.
5/ Safety biomarkers.
6/ Liver biopsy (patients previously enrolled in DFI13412).
7/ Liver ultrasound/Doppler (patients previously enrolled in DFI13803)
8/ Immune response assessments

Secondary Outcome(s)

Secondary end point(s): - Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume
- Pulmonary Imaging
- Pulmonary function test
- Hematology (hemoglobin and platelet count)
- Lipid profile
- Health outcome questionnaires
- Hand X ray for bone age and bone maturation (pediatric patients)
- Linear patient growth by height Z -score (pediatric patients)

Timepoint(s) of evaluation of this end point: Baseline to up to 9 years

Secondary ID(s)

2013-000051-40-GB

LTS13632

Source(s) of Monetary Support

Genzyme Corporation

Ethics review

Status: Approved
Approval date: 14/11/2018
Contact: