Trial · NCT01173016
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
NCT01173016, Phase 1, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2020-03-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Administration of IV Laronidase Post Bone Marrow Transplant in Hurler. Trials, record NCT01173016. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01173016
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
- NCT number
- NCT01173016
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 11 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Hurler Syndrome
- Interventions
- Laronidase
- First posted
- 2010-07-30
- Last updated
- 2020-03-20
- Results first posted
- 2020-03-20
- Limitations and caveats
- All listed serious and non-serious adverse events are infusion related except for a seizure that was thought to be unrelated to the study drug.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Adherence to the Scheduled Weekly Infusion by the Participants | 24 months | To determine the feasibility of giving weekly Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation, compliance throughout the study with drug administration, the percentage of adherence to the scheduled weekly infusion for each participant is measured. |
| Primary | Number of Participants Experiencing Severe Adverse Events | 24 months | Number of participants experiencing severe adverse events that occur after administration with Laronidase to determine the feasibility of giving weekly Laronidase |
| Secondary | Changes in Growth Velocity | Baseline, Month 24 | difference between baseline and month 24 growth velocities |
| Secondary | Change in Muscle Strength | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | Handgrip strength is measured three times in both hands with a mechanical handheld Biodex System 3 dynamometer (Biodex medical Systems, Inc., Shirley, NY) with the subject in a seated position at each visit; the average for each hand is presented. |
| Secondary | Change in Peak Heart Rate to Monitor "Fitness" | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | A modified Balke Treadmill Test was performed. Briefly, patients began walking at 2.0 mph with a 2% increase in grade every 2 min. A 12-lead electrocardiogram was monitored continuously throughout the test for the determination of heart rate and dysrhythmias or ischemic changes. Heart rate was measured at the end of each stage (i.e. every 2 min) . |
| Secondary | Number of Participants Showing Improvements in Joint Range of Motion (ROM) | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | Bilateral shoulder flexion, elbow extension, and hip extension were measured using goniometry. Improvements are defined for all joints as \>5°. |
| Secondary | Shortening Fraction to Determine Systolic Cardiac Function | Baseline and month 24 | Cardiac ultrasounds were obtained at baseline and month 24. Two-dimensional imaging was obtained for determination of anatomy. Shortening fraction (SF \[normal \> 27%\]) was calculated by standard methods to determine the normal systolic cardiac function |
| Secondary | Number of Participants With Changes in Cardiac Echo Structural Parameters | Baseline and month 24 | Pulse-wave and color Doppler interrogation of cardiac valves was performed for determination of valve regurgitation |
| Secondary | Correlation of 6 Minute Walk Test With Anti-laronidase Antibody + Status | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | 6 minute walk test (6MWT) was performed to assess overall physical function and health status. In brief, a 30m hospital corridor marked by colored tape at each end was used. Subjects were instructed to walk from end to end at their self-selected pace, while attempting to cover as much distance as possible in the 6 min. The patients were instructed to walk around the mark as they changed direction. The time and distance covered was recorded, as was the heart rate prior to and immediately after completion of the walk test. To find the association between the rate of change in 6MWT, and anti drug antibody (ADA) titer, a statistical test is performed adjusting for age at the time of enrollment. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Adherence to the Scheduled Weekly Infusion by the Participants | 24 months | percentage | MEDIAN | |
| PRIMARY | Number of Participants Experiencing Severe Adverse Events | 24 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Changes in Growth Velocity | Baseline, Month 24 | Baseline growth velocity value was available in six of ten participants. One female participant was excluded from the within-group growth analyses due to a bone age of 14.5 years | cm/year | MEAN |
| SECONDARY | Change in Muscle Strength | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | kg | MEAN | |
| SECONDARY | Change in Peak Heart Rate to Monitor Fitness | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | bpm | MEAN | |
| SECONDARY | Number of Participants Showing Improvements in Joint Range of Motion (ROM) | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Shortening Fraction to Determine Systolic Cardiac Function | Baseline and month 24 | two subjects didn't have both baseline and month 24 echo data | percentage of systolic cardiac function | MEAN |
| SECONDARY | Number of Participants With Changes in Cardiac Echo Structural Parameters | Baseline and month 24 | Two subjects didn't have both baseline and month 24 echo data | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Correlation of 6 Minute Walk Test With Anti-laronidase Antibody + Status | Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months | meters | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Adherence to the Scheduled Weekly Infusion by the Participants | Laronidase After Transplantation | 99 | Full Range 79 to 100 | percentage | |
| Number of Participants Experiencing Severe Adverse Events | Laronidase After Transplantation | 1 | Participants | ||
| Changes in Growth Velocity | Laronidase After Transplantation | -1.1 | Standard Deviation 3.8 | cm/year | |
| Change in Muscle Strength | Laronidase After Transplantation | 1.1 | Standard Deviation 1.9 | kg | |
| Change in Peak Heart Rate to Monitor Fitness | Laronidase After Transplantation | 23 | Standard Deviation 18 | bpm | |
| Number of Participants Showing Improvements in Joint Range of Motion (ROM) | Laronidase After Transplantation | Left or right shoulder ROM improvement | 4 | Participants | |
| Number of Participants Showing Improvements in Joint Range of Motion (ROM) | Laronidase After Transplantation | Left or right elbow ROM improvement | 3 | Participants | |
| Number of Participants Showing Improvements in Joint Range of Motion (ROM) | Laronidase After Transplantation | Left or right hip ROM improvement | 5 | Participants | |
| Shortening Fraction to Determine Systolic Cardiac Function | Laronidase After Transplantation | 38 | Full Range 34 to 52 | percentage of systolic cardiac function | |
| Number of Participants With Changes in Cardiac Echo Structural Parameters | Laronidase After Transplantation | Mitral regurgitation - unchaged | 7 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Laronidase After Transplantation | 11 | |
| Overall Study | COMPLETED | Laronidase After Transplantation | 10 | |
| Overall Study | NOT COMPLETED | Laronidase After Transplantation | 1 |
Source ClinicalTrials.gov
Adverse events10
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 | Allergy Reaction/Hypersensitivity | Immune system disorders | Laronidase After Transplantation | 1 | 11 | 4 |
| other | Fever | General disorders | Laronidase After Transplantation | 1 | 11 | 1 |
| other | Headache | Nervous system disorders | Laronidase After Transplantation | 1 | 11 | 1 |
| other | Hives | Immune system disorders | Laronidase After Transplantation | 1 | 11 | 3 |
| other | Hypertension | Vascular disorders | Laronidase After Transplantation | 3 | 11 | 47 |
| other | Pain, Right Shoulder | General disorders | Laronidase After Transplantation | 1 | 11 | 1 |
| other | Sinus Tachycardia | Cardiac disorders | Laronidase After Transplantation | 3 | 11 | 36 |
| other | Hypotension | Cardiac disorders | Laronidase After Transplantation | 1 | 11 | 1 |
| serious | Syncope | Nervous system disorders | Laronidase After Transplantation | 1 | 11 | |
| serious | Seizure | Nervous system disorders | Laronidase After Transplantation | 1 | 11 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Laronidase After Transplantation | ||
| Total, other adverse events | Laronidase After Transplantation | 7 | 11 |
| Total, serious adverse events | Laronidase After Transplantation | 1 | 11 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31575939 | 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 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/NCT01173016
GET https://conductscience.org/api/trials/v1/NCT01173016/registered