Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Genzyme logo

Genzyme

Status and phase

Completed
Phase 3

Conditions

Hurler-Scheie Syndrome
Hurler's Syndrome
Mucopolysaccharidosis I
Scheie Syndrome

Treatments

Biological: Aldurazyme
Biological: placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00146770
ALID-006-01

Details and patient eligibility

About

This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.

Enrollment

45 patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

  • The patient or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
  • The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
  • The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
  • A female patient of childbearing potential must have a negative pregnancy test at entry

Exclusion criteria

  • The patient is pregnant or lactating.
  • The patient has received an investigational drug within 30 days prior to the study enrollment.
  • The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

45 participants in 2 patient groups

Placebo/Aldurazyme
Active Comparator group
Description:
Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme.
Treatment:
Biological: placebo
Biological: Aldurazyme
Aldurazyme/Aldurazyme
Active Comparator group
Description:
Patients received 26 weeks of Aldurazyme in the Double-Blind Study and then received 182 weeks of Aldurazyme in this Extension Study; patients received a total of 208 weeks of Aldurazyme.
Treatment:
Biological: Aldurazyme

Trial contacts and locations

25

Loading...

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems