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

Mucopolysaccharidosis I Clinical Trial

Official title:

A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00146770
• Other study ID #: ALID-006-01
• Status: Completed
• Phase: Phase 3
• First received: September 2, 2005
• Last updated: March 17, 2015
• Start date: May 2001
• Est. completion date: March 2005

Study information

• Verified date: March 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: March 2005
• Est. primary completion date: March 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

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

Intervention

Biological:
• Aldurazyme
Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks
• Aldurazyme
0.58 mg/kg Aldurazyme every week for 208 weeks
• placebo
Placebo for 26 weeks

Locations

Country	Name	City	State
Brazil	Hospital Infantil Joana de Gusmao	Florianopolis
Brazil	Hospital Universatario de Universidade Federal de Santa Catarina	Florianopolis
Canada	Alberta Children's Hospital	Calgary
Canada	The Hospital for Sick Children	Toronto	Ontario
Germany	Children's Hospital Klinikum Nord Heidberg	Hamburg
Germany	Medizinishe Hochshule Hannover	Hannover
Germany	Children's Hospital at the University Hospital of Heidelberg	Heidelberg
Germany	Children's Hospital Klinikum der F.S. Universitat	Jena
Italy	Catholic University Sacro Cuore	Rome
Netherlands	Academisch Ziekenhuis Rotterdam	Rotterdam
United Kingdom	Belfast City Hospital	Belfast
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Blackpool Victoria Hospital	Blackpool	Lancashire
United Kingdom	Bristol Royal Hospital for Children and Frenchay Hospital	Bristol
United Kingdom	Gartnavel Hospital	Glasgow
United Kingdom	Great Ormond Street Hospital for Sick Children and NHS Trust	London
United Kingdom	Royal Victoria Hospital	Newcastle upon Tyne
United States	Emory University School of Medicine	Atlanta	Georgia
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	Merle West Medical Center	Klamath Falls	Oregon
United States	University of South Alabama	Mobile	Alabama
United States	The Childrens Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Rhode Island Hospital	Providence	Rhode Island
United States	University of Rochester	Rochester	New York
United States	Toledo Children's Hospital	Toledo	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company	BioMarin/Genzyme LLC

Countries where clinical trial is conducted

United States,  Brazil,  Canada,  Germany,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change From Baseline to Week 182 in Urinary GAG Level	Urinary Glycosaminoglycan (GAG) Levels: >> Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.	Baseline to Week 182	No
Primary	Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)	Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.	Baseline to Week 182	No
Primary	Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)	Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.	Baseline to Week 182	No
Secondary	Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)	Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events indicates a greater response.	Baseline to Week 182	No
Secondary	Change From Baseline to Week 182 in Liver Volume	Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.	Baseline to Week 182	No
Secondary	Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score	CHAQ/HAQ = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.	Baseline to Week 182	No
Secondary	Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)	Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.	Baseline to Week182	No