Status:
COMPLETED
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Sphingomyelin Lipidosis
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE1
PHASE2
Brief Summary
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 th...
Detailed Description
The maximum study duration per participant was approximately 18 months (screening period: up to 60 days; treatment period: 64 weeks; post-treatment period: up to 37 days, not applicable if participant...
Eligibility Criteria
Inclusion
- Inclusion criteria :
- 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.
- Exclusion criteria:
- 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.
Exclusion
Key Trial Info
Start Date :
May 1 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 9 2019
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT02292654
Start Date
May 1 2015
End Date
December 9 2019
Last Update
March 23 2022
Active Locations (6)
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1
Investigational Site Number 840001
New York, New York, United States, 10029
2
Investigational Site Number 076001
Porto Alegre, Brazil, 90035 003
3
Investigational Site Number 250002
Bron, France, 69677
4
Investigational Site Number 276001
Mainz, Germany, 55131