Trial · NCT00176891
Stem Cell Transplant w/Laronidase for Hurler
NCT00176891, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2019-07-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Stem Cell Transplant w/Laronidase for Hurler. Trials, record NCT00176891. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00176891
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Stem Cell Transplant w/Laronidase for Hurler
- NCT number
- NCT00176891
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome
- Interventions
- Stem Cell Transplant, Laronidase ERT
- First posted
- 2005-09-15
- Last updated
- 2019-07-11
- Results first posted
- 2019-07-11
- Limitations and caveats
All of the patients on this trial also underwent allogeneic blood/marrow transplantation. All the serious and non-serious adverse events listed were related to the process of transplantation and not to the study medication, Laronidase.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Alive at One Year Post Transplant | one year | |
| Primary | Number of Patients Requiring Ventilator Support at One Year Post Transplant | one year | |
| Secondary | Donor Engraftment | Day 100 post transplant | |
| Secondary | Patients With Grade III-IV Acute GVHD | Day 100 post transplant | |
| Secondary | Reduction in Glycosaminoglycans (GAG) | Prior to, During and After ERT | Data was not collected on this outcome measure and is not available for reporting. |
| Secondary | Toxicity (Adverse Events) Associated With Infusions of Laronidase | 1 year post transplant | Data was not collected on this outcome measure and is not available for reporting. |
| Secondary | Development of Anti-iduronidase Antibodies in Serum | 1 Year | |
| Secondary | Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography | Baseline, 12 weeks after laronidase, after transplant |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients Alive at One Year Post Transplant | one year | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number of Patients Requiring Ventilator Support at One Year Post Transplant | one year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Donor Engraftment | Day 100 post transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Patients With Grade III-IV Acute GVHD | Day 100 post transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Reduction in Glycosaminoglycans (GAG) | Prior to, During and After ERT | Data was not collected on this outcome measure and is not available for reporting. | ||
| SECONDARY | Toxicity (Adverse Events) Associated With Infusions of Laronidase | 1 year post transplant | Data was not collected on this outcome measure and is not available for reporting. | ||
| SECONDARY | Development of Anti-iduronidase Antibodies in Serum | 1 Year | Data was not collected on this outcome measure and is not available for reporting. | ||
| SECONDARY | Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography | Baseline, 12 weeks after laronidase, after transplant | Data was not collected on this outcome measure and is not available for reporting. |
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 Patients Alive at One Year Post Transplant | Laronidase ERT Treatment | 18 | Participants | ||
| Number of Patients Requiring Ventilator Support at One Year Post Transplant | Laronidase ERT Treatment | 6 | Participants | ||
| Donor Engraftment | Laronidase ERT Treatment | 21 | Participants | ||
| Patients With Grade III-IV Acute GVHD | Laronidase ERT Treatment | 1 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Laronidase ERT Treatment | 25 | |
| Overall Study | COMPLETED | Laronidase ERT Treatment | 22 | |
| Overall Study | NOT COMPLETED | Laronidase ERT Treatment | 3 |
Source ClinicalTrials.gov
Adverse events41
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 | Increased Liver Function Test | Hepatobiliary disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Acute Respiratory Distress | Respiratory, thoracic and mediastinal disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Hyperbilirubinemia | Hepatobiliary disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Blood/Bone Marrow, NOS | Blood and lymphatic system disorders | Laronidase ERT Treatment | 2 | 22 | |
| other | Cerebrovascular Ischemia | Nervous system disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Cardiac, NOS | Cardiac disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Cholecystitis | Hepatobiliary disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Dehydration | Gastrointestinal disorders | Laronidase ERT Treatment | 1 | 22 | |
| other | Diarrhea | Gastrointestinal disorders | Laronidase ERT Treatment | 4 | 22 | |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Laronidase ERT Treatment | 1 | 22 |
1 to 10 of 41Source ClinicalTrials.gov
See all 41 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Laronidase ERT Treatment | ||
| Total, other adverse events | Laronidase ERT Treatment | 21 | 22 |
| Total, serious adverse events | Laronidase ERT Treatment | 6 | 22 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00176891
GET https://conductscience.org/api/trials/v1/NCT00176891/events?page=2