Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Completed · Phase 1/Phase 2

Conditions studied: Sphingomyelin Lipidosis

In brief

Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.

Key facts

Study ID

NCT02292654

Run by

Genzyme, a Sanofi Company

People needed

20

Starts

2015-05-01

Expected to finish

2019-12-09

Last updated by the study team

2022-03-23

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed.
• The participant was <18 years of age on the date of informed assent/consent.
• The participant had documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes.
• The participant had a spleen volume greater than or equal to (>=) 5 multiples of normal (MN) measured by magnetic resonance imaging (MRI); participants who had partial splenectomy were allowed if the procedure was performed >=1 year before screening and the residual spleen volume was >=5 MN.
• The participant's height was -1 Z-score or lower.
• A negative serum pregnancy test in female participants of childbearing potential.
• Female participants of childbearing potential and male participants must be willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception.

You may not qualify if…

• The participant had received an investigational drug within 30 days before study enrollment.
• The participant had any of the following medical conditions:
• An active, serious, intercurrent illness.
• Active hepatitis B or hepatitis C infection.
• Infection with human immunodeficiency virus (HIV).
• Cirrhosis (determined by clinical evaluation).
• Significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40 percent (%) left ventricular ejection fraction by echocardiogram).
• Malignancy diagnosed within the previous 5 years (except basal cell carcinoma).
• Any other extenuating circumstance that can significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
• The participant had acute or rapidly progressive neurological abnormalities.
• The participant was homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations.
• The participant had a delay of gross motor skills.
• The participant had a major organ transplant (eg, bone marrow, liver).
• The participant required use of invasive ventilatory support.
• The participant required use of noninvasive ventilatory support while awake and for greater than (>)12 hours a day.
• The participant in the investigator's opinion, was unable to adhere to the requirements of the study.
• The participant had a platelet count <60 × 10\^3/µL (based on the average of 2 screening samples obtained up to 24 hours apart).
• The participant had alanine aminotransferase or aspartate aminotransferase >250 IU/L or total bilirubin >1.5 mg/dL.
• The participant had an international normalized ratio (INR) >1.5.
• The participant was unwilling or unable to abstain from ingesting alcohol the day before through 3 days after each infusion of olipudase alfa during the treatment period. Measuring alcohol concentration in blood was not required.
• The participant was scheduled during the study for in-patient hospitalization including elective surgery.
• The participant required medication(s) that may can decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, or desipramine]).
• The participant was breast-feeding.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Investigational Site Number 840001 — New York, New York, United States
• Investigational Site Number 076001 — Porto Alegre, Brazil
• Investigational Site Number 250002 — Bron, France
• Investigational Site Number 276001 — Mainz, Germany
• Investigational Site Number 380001 — Udine, Italy
• Investigational Site Number 826001 — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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