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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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    Page 1 of 2 (6 locations)

    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