Trial · NCT00741338
Immune Tolerance Study With Aldurazyme® (Laronidase)
NCT00741338, Phase 1, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2014-07-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Immune Tolerance Study With Aldurazyme® (Laronidase). Trials, record NCT00741338. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00741338
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Immune Tolerance Study With Aldurazyme® (Laronidase)
- NCT number
- NCT00741338
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 7 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Mucopolysaccharidosis I
- Interventions
- Laronidase, Cyclosporine A (CsA), Azathioprine (Aza)
- First posted
- 2008-08-26
- Last updated
- 2014-07-02
- Other registries' IDs
- eudract:2007-001163-30
- Results first posted
- 2014-07-02
- Limitations and caveats
Study was discontinued on September 10,2013 due to changing standards of care for this population, practical infeasibility of routinely monitoring plasma CsA in clinical setting, inconclusive results of interim analysis and not due to safety concern.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Who Achieved Immune Tolerance Induction | 24 weeks after start of full-dose laronidase therapy | Immune tolerance induction success was defined as development of an anti-laronidase immunoglobulin G (IgG) antibody titer less than or equal to (\<=) 1:3200 after 24 weeks of receiving full-dose (0.58 mg/kg) laronidase therapy. |
| Secondary | Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal | Baseline, end of treatment/early withdrawal (up to 24 weeks after start of full-dose laronidase therapy) | Urinary Glycosaminoglycan (uGAG) Levels: concentration of glycosaminoglycan (GAG) relative to creatinine in urine. A greater decrease in uGAG level indicates a greater response. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Who Achieved Immune Tolerance Induction | 24 weeks after start of full-dose laronidase therapy | Safety population included all participants who received any study drug treatment. | participants | NUMBER |
| SECONDARY | Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal | Baseline, end of treatment/early withdrawal (up to 24 weeks after start of full-dose laronidase therapy) | Safety population included all participants who received any study drug treatment. | percent change in uGAG level | MEDIAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Who Achieved Immune Tolerance Induction | Cohort 1 | 0 | participants | ||
| Number of Participants Who Achieved Immune Tolerance Induction | Cohort 2 | 1 | participants | ||
| Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal | Cohort 1 | -43.80 | Full Range -61.70 to -6.70 | percent change in uGAG level | |
| Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal | Cohort 2 | -72.50 | Full Range -84.20 to -62.50 | percent change in uGAG level |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 3 | |
| Overall Study | STARTED | Cohort 2 | 3 | |
| Overall Study | COMPLETED | Cohort 1 | 3 | |
| Overall Study | COMPLETED | Cohort 2 | 2 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2 | 1 |
Source ClinicalTrials.gov
Adverse events136
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Cohort 1 | 0 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Cohort 2 | 1 | 3 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 1 | 1 | 3 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 2 | 0 | 3 | |
| other | Lymphocytosis | Blood and lymphatic system disorders | Cohort 1 | 0 | 3 | |
| other | Lymphocytosis | Blood and lymphatic system disorders | Cohort 2 | 1 | 3 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 1 | 1 | 3 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 2 | 1 | 3 | |
| other | Thrombocytosis | Blood and lymphatic system disorders | Cohort 1 | 0 | 3 | |
| other | Thrombocytosis | Blood and lymphatic system disorders | Cohort 2 | 1 | 3 |
1 to 10 of 136Source ClinicalTrials.gov
See all 136 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | ||
| Total, all-cause mortality | Cohort 2 | ||
| Total, other adverse events | Cohort 1 | 3 | 3 |
| Total, other adverse events | Cohort 2 | 3 | 3 |
| Total, serious adverse events | Cohort 1 | 2 | 3 |
| Total, serious adverse events | Cohort 2 | 1 | 3 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28119821 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort 1 vs Cohort 2: Number of Participants Who Achieved Immune Tolerance Induction | Trial comparisons |
| Cohort 1 vs Cohort 2: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00741338
GET https://conductscience.org/api/trials/v1/NCT00741338/events?page=2