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

NCT01722526 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2015-07-30

No results posted yet for this study

Summary

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

Conditions

  • Human Acid Sphingomyelinase Deficiency

Interventions

DRUG

Recombinant human acid sphingomyelinase

Administered intravenously every 2 weeks for 26 weeks

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-03-31
Primary Completion
2014-01-31
Completion
2014-01-31

Countries

  • United States
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01722526 on ClinicalTrials.gov