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

NCT00410566 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2015-03-19

No results posted yet for this study

Summary

The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.

Conditions

  • Acid Sphingomyelinase Deficiency
  • Niemann-Pick Disease

Interventions

DRUG

rhASM

Single dose of 0.03mg/kg body weight IV

DRUG

rhASM

Single dose of 0.1mg/kg body weight IV

DRUG

rhASM

Single dose of 0.3mg/kg body weight IV

DRUG

rhASM

Single dose of 0.6mg/kg body weight IV

DRUG

rhASM

Single dose of 1.0mg/kg body weight IV

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2006-12-31
Primary Completion
2009-03-31
Completion
2009-04-30

Countries

  • United States

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 NCT00410566 on ClinicalTrials.gov