Phase 3 N=45 Randomized Quadruple-blind Treatment

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Mucopolysaccharidosis I · Hurlers Syndrome · Hurler-Scheie Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT00912925 ↗

Enrolled (actual)

Serious AEs

6.7%

Results posted

Aug 2009

Primary outcome: Primary: Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) — 54.2; 48.4; 51.5; 50.2 Percent predicted FVC

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: rhIDU (recombinant human-Alpha-L-Iduronidase) (Biological); Placebo (Biological)
Age: Pediatric, Adult, Older Adult · 5+ yrs
Sex: All
Sponsor: Genzyme, a Sanofi Company
Primary completion: Sep 2001

Outcome Measures

Outcome	Result	p-value
PRIMARY Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)	54.2; 48.4; 51.5; 50.2; -2.7; 1.8	—
PRIMARY Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)	366.7; 319.1; 348.3; 338.8; -18.4; 19.7	—
SECONDARY Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)	14.7; 20.5; 15.5; 17.6; 0.4; -2.9	—
SECONDARY Overall Percent Change From Baseline to Week 26 in Liver Volume	1368.1; 12212.2; 1366.5; 979.8; 1.3; -18.9	—
SECONDARY Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score	1.9; 2.0; 1.8; 1.9; -0.1; -0.2	—
SECONDARY Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)	89.8; 96.1; 85.2; 88.9; -4.6; -1.2	—
SECONDARY Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels	183.3; 188.9; 250.2; 81.3; 47.3; -54.1	—

Summary

This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients

Eligibility Criteria

Inclusion Criteria

The patient had a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
Female patients of childbearing potential had a negative pregnancy test (urine-beta-human chorionic gonadotropin (hCG)) at baseline (all female patients of childbearing potential and sexually mature male patients were advised to use a medically accepted method of contraception throughout the study).
The patient was capable of standing independently for 6 minutes and walking a minimum of 5 meters within 6 minutes.
The patient was capable of performing a reproducible FVC maneuver.
The patient had a baseline FVC value that was less than or equal to 80% of the patient's predicted normal FVC value based on polgar predicted values for standing height for children 5 through 7 years of age and the Hankinson predicted values for ages 8 and above.

Exclusion Criteria

The patient had undergone a tracheostomy.
The patient had previously undergone a bone marrow transplantation.
The patient was pregnant or lactating.
The patient has received an investigational drug within 30 days prior to study enrollment.
The patient had a medical condition, serious intercurrent illness, or other extenuating circumstance that could have significantly interfered with study compliance including all prescribing evaluations and follow-up activities.
The patient had a known hypersensitivity to rhIDU or to components of the active or placebo test solutions.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00912925). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.