Clinical trial NCT00912925: Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Mucopolysaccharidosis I
Hurlers Syndrome
Hurler-Scheie Syndrome
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: BioMarin/Genzyme LLC
First posted: 2009-06-03
Enrollment: 45
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Primary outcomes:
measure: Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)
timeFrame: Baseline to Week 26
measure: Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
timeFrame: Baseline to Week 26
Secondary outcomes:
measure: Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)
timeFrame: Baseline to Week 26
measure: Overall Percent Change From Baseline to Week 26 in Liver Volume
timeFrame: Baseline to Week 26
measure: Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score
timeFrame: Baseline to week 26
measure: Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)
timeFrame: Baseline to Week 26
measure: Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels
timeFrame: Baseline to Week 26
Other outcomes:
Locations:
city: New York
country: United States
state: New York
city: Chapel Hill
country: United States
state: North Carolina
city: Vancouver
country: Canada
state: British Columbia
city: Mainz
country: Germany
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.