Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-03-31

Study Completion Date

2014-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

NCT00410566

Acid Sphingomyelinase Deficiency Niemann-Pick Disease

TERMINATED PHASE1

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

NCT02004704

Sphingomyelin Lipidosis

COMPLETED PHASE2

A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

NCT06949358

Niemann-Pick Disease

COMPLETED PHASE2

Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)

NCT07274826

Acid Sphingomyelinase Deficiency (ASMD)

ACTIVE_NOT_RECRUITING

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

NCT00025896

Pompe Disease Glycogen Storage Disease Type II Acid Maltase Deficiency Disease +1 more

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Human Acid Sphingomyelinase Deficiency

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Recombinant human acid sphingomyelinase

Participants will receive rhASM of an initial dose of 0.1 mg/kg, followed by several dose escalations, as tolerated, up to 3.0 mg/kg. All doses are given 2 weeks apart.

Group Type EXPERIMENTAL

Recombinant human acid sphingomyelinase

Intervention Type DRUG

Administered intravenously every 2 weeks for 26 weeks

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Recombinant human acid sphingomyelinase

Administered intravenously every 2 weeks for 26 weeks

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

GZ402665

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Patients with documented non-neuronopathic acid sphingomyelinase deficiency
* The patient has a diffusing capacity of carbon monoxide (DLco) \>20% and ≤80% of the predicted normal value.
* The patient has a spleen volume ≥6 multiples of normal(MN). A partial splenectomy will be permitted if performed ≥1 year prior to Screening/Baseline and residual spleen volume is ≥6 MN.
* The patient who is receiving lipid lowering therapy should be on a stable dose and regimen of lipid-lowering therapy(ies) for at least 12 weeks prior to Screening/Baseline, with the patient expected to remain on the same dose and regimen throughout the 26-week treatment period.
* The patient who is female and of childbearing potential must have a negative serum pregnancy test for β-HCG.

Exclusion Criteria

* The patient is female and pregnant or lactating.
* The patient has a Body Mass Index(BMI)\>30.
* The patient has received an investigational drug within 30 days prior to study enrollment
* The patient has a medical condition or any extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
* The patient has had a major organ transplant
* ALT or AST \>250 IU/L or total bilirubin \>1.5 mg/dL.
* The patient is unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each rhASM infusion for the duration of the study.
* The patient requires medications that may decrease rhASM
* The patient is unwilling or unable to avoid the use of medications or herbal supplements that may cause or prolong bleeding, or have potential hepatotoxicity within 10 days prior to and 3 days after liver biopsy

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No