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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-05-01

Study Completion Date

2019-12-09

Brief Summary

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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.

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Detailed Description

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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 participants enrolled in a long-term extension treatment trial).

Conditions

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Sphingomyelin Lipidosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Olipudase alfa

Participants received intravenous (IV) infusion of olipudase alfa once every 2 weeks (Q2W) for 64 weeks. Each participant underwent a dose escalation according to the following paradigm: 0.03, 0.1, 0.3, 0.3, 0.6, 0.6, 1.0, 2.0, 3.0 milligram per kilogram (mg/kg). Three (3) mg/kg was the target maintenance dose, which was maintained for the remaining duration of 64 treatment weeks.

Group Type EXPERIMENTAL

Olipudase alfa

Intervention Type DRUG

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion

Interventions

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Olipudase alfa

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion

Intervention Type DRUG

Other Intervention Names

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GZ402665

Eligibility Criteria

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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.

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No