A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 3 October 2023
• Main ID: NCT02004704
• Date of registration: 26/11/2013
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Long-Term Study of Olipudase Alfa 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: December 4, 2013
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02004704
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

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

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

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.

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]).

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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Sphingomyelin Lipidosis

Intervention(s)

Drug: GZ402665

Primary Outcome(s)

Safety biomarkers [Time Frame: Baseline to up to 9 years]

Adverse events (AEs)/treatment-emergent adverse events (TEAEs), including infusion-associated reactions and adverse events of special interest (AESIs) [Time Frame: Baseline to up to 9 years]

Liver biopsy (patients previously enrolled in DFI13412) [Time Frame: Baseline to after at least 3 years in the study]

Clinical laboratory tests [Time Frame: Baseline to up to 9 years]

Vital signs, electrocardiograms and echocardiograms with Doppler [Time Frame: Baseline to up to 9 years]

Immune response assessments [Time Frame: Baseline to up to 9 years]

Complete physical examinations including extended neurologic and abbreviated physical exams [Time Frame: Baseline to up to 9 years]

Liver ultrasound/Doppler (patients previously enrolled in DFI13803) [Time Frame: Baseline to 5 years]

Secondary Outcome(s)

Hematology [Time Frame: Baseline to up to 9 years]

Pulmonary imaging [Time Frame: Baseline to up to 9 years]

Spleen and liver volume [Time Frame: Baseline to up to 9 years]

Health outcome questionnaires (adults and pediatric) [Time Frame: Baseline to up to 9 years]

Linear patient growth by height Z -score (pediatric patients) [Time Frame: Baseline to up to 9 years]

Hand X ray for bone age and bone maturation (pediatric patients) [Time Frame: Baseline to up to 9 years]

Lipid profile [Time Frame: Baseline to up to 9 years]

Pulmonary function test [Time Frame: Baseline to up to 9 years]

Secondary ID(s)

LTS13632

2013-000051-40

U1111-1141-65868

Source(s) of Monetary Support

Please refer to primary and secondary sponsors