Clinical trial NCT00146770: Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Official title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: BioMarin/Genzyme LLC
First posted: 2005-09-07
Enrollment: 45
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)
timeFrame: Baseline to Week 182
measure: Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)
timeFrame: Baseline to Week 182
Secondary outcomes:
measure: Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)
timeFrame: Baseline to Week 182
measure: Change From Baseline to Week 182 in Liver Volume
timeFrame: Baseline to Week 182
measure: Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score
timeFrame: Baseline to Week 182
measure: Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)
timeFrame: Baseline to Week182
Other outcomes:
measure: Change From Baseline to Week 182 in Urinary GAG Level
timeFrame: Baseline to Week 182
Locations:
city: Mobile
country: United States
facility: University of South Alabama
state: Alabama
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Toledo
country: United States
facility: Toledo Children's Hospital
state: Ohio
city: Klamath Falls
country: United States
facility: Merle West Medical Center
state: Oregon
city: Philadelphia
country: United States
facility: The Childrens Hospital of Philadelphia
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Florianopolis
country: Brazil
facility: Hospital Universatario de Universidade Federal de Santa Catarina
city: Florianopolis
country: Brazil
facility: Hospital Infantil Joana de Gusmao
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Calgary
country: Canada
facility: Alberta Children's Hospital
city: Hamburg
country: Germany
facility: Children's Hospital Klinikum Nord Heidberg
city: Hannover
country: Germany
facility: Medizinishe Hochshule Hannover
city: Heidelberg
country: Germany
facility: Children's Hospital at the University Hospital of Heidelberg
city: Jena
country: Germany
facility: Children's Hospital Klinikum der F.S. Universitat
city: Rome
country: Italy
facility: Catholic University Sacro Cuore
city: Rotterdam
country: Netherlands
facility: Academisch Ziekenhuis Rotterdam
city: Blackpool
country: United Kingdom
facility: Blackpool Victoria Hospital
state: Lancashire
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Bristol
country: United Kingdom
facility: Bristol Royal Hospital for Children and Frenchay Hospital
city: Glasgow
country: United Kingdom
facility: Gartnavel Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Sick Children and NHS Trust
city: Newcastle upon Tyne
country: United Kingdom
facility: Royal Victoria Hospital
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.
citation: Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Sidman M, Kakkis ED, Cox GF. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847.