Mucopolysaccharidosis IPhase 3NCT00146770
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
A Phase 3 study testing Aldurazyme in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 25 sites in 7 countries.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome
- What is being tested?
- Aldurazyme
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 25 sitesin 7 countries
- Study dates
- May 2001to Mar 2005
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
25 sites in 7 countries.
25 sites in 7 countries
United States8 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Merle West Medical Center
Klamath Falls, Oregon, 97601
University of South Alabama
Mobile, Alabama, 36604
The Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Rhode Island Hospital
Providence, Rhode Island, 02903
University of Rochester
Rochester, New York, 14542
Toledo Children's Hospital
Toledo, Ohio, 43606
United Kingdom7 sites
Belfast City Hospital
Belfast, BT9 78A
Birmingham Children's Hospital
Birmingham, B4 6NH
Blackpool Victoria Hospital
Blackpool, Lancashire, FY3 8NR
Bristol Royal Hospital for Children and Frenchay Hospital
Bristol, BS16 1LE
Gartnavel Hospital
Glasgow, G11 6NT
Great Ormond Street Hospital for Sick Children and NHS Trust
London, WC1N 3GH
Royal Victoria Hospital
Newcastle upon Tyne, NE1 4LB
Germany4 sites
Children's Hospital Klinikum Nord Heidberg
Hamburg, 22143
Medizinishe Hochshule Hannover
Hanover, 30625
Children's Hospital at the University Hospital of Heidelberg
Heidelberg, 69120
Children's Hospital Klinikum der F.S. Universitat
Jena, 07740
Brazil2 sites
Hospital Universatario de Universidade Federal de Santa Catarina
Florianópolis, 88040-500
Hospital Infantil Joana de Gusmao
Florianópolis, CEP 88025-301
Canada2 sites
Alberta Children's Hospital
Calgary, T2T 5C7
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
About this study
Official title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
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.
- Being tested
- Aldurazyme, placebo
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AldurazymeBiological
Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks
AldurazymeBiological
0.58 mg/kg Aldurazyme every week for 208 weeks
placeboBiological
Placebo for 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| Placebo/Aldurazyme | Active comparator | 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. Receives: Biological: Aldurazyme; Biological: placebo |
| Aldurazyme/Aldurazyme | Active comparator | 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. Receives: Biological: Aldurazyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Week 182
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.
Measured: Baseline to Week 182
Secondary outcomes (4)
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.
Measured: Baseline to Week 182
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.
Measured: Baseline to Week 182
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.
Measured: Baseline to Week 182
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.
Measured: Baseline to Week182
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2005 (estimated)
- Study startMay 2001
- Primary completionMar 2005
- Study completionMar 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- BioMarin/Genzyme LLC
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00146770
- Sponsor's protocol ID
- ALID-006-01
- Status last verified
- Mar 2015
- First submitted
- 2 Sep 2005
- Last updated
- 3 Apr 2015
- Results posted
- Yes, on ClinicalTrials.gov
Publications
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.
PubMed 33874971Clarke 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.
PubMed 19117887
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
- ClinicalTrials.gov ID
- NCT00146770
- Acronym / sponsor ID
- ALID-006-01
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- BioMarin/Genzyme LLC
- Study start
- May 2001
- Primary completion
- Mar 2005 (actual)
- Study completion
- Mar 2005 (actual)
- First posted
- 7 Sep 2005 (estimated)
- Results
- Posted 16 Jun 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo/Aldurazyme | Active comparator | Biological: Aldurazyme, Biological: placeboPatients 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. |
| Aldurazyme/Aldurazyme | Active comparator | Biological: AldurazymePatients 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AldurazymePlacebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks | Biological | Placebo/Aldurazyme |
| Aldurazyme0.58 mg/kg Aldurazyme every week for 208 weeks | Biological | Aldurazyme/Aldurazyme |
| placeboPlacebo for 26 weeks | Biological | Placebo/Aldurazyme |
Primary outcome
Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)
Time frame: Baseline to Week 182
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.
Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)
Time frame: Baseline to Week 182
Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
4 secondary outcomes
Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)
Time frame: Baseline to Week 182
Change From Baseline to Week 182 in Liver Volume
Time frame: Baseline to Week 182
Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score
Time frame: Baseline to Week 182
Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)
Time frame: Baseline to Week182
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33874971Tandon 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.
- Derived from this study: PubMed 19117887Clarke 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
25 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Aldurazyme in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 25 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, United Kingdom, Germany, Brazil, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Jun 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) (time frame: Baseline to Week 182). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00146770, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2015.