Trial Outcomes & Findings for Stem Cell Transplant w/Laronidase for Hurler (NCT NCT00176891)
NCT ID: NCT00176891
Last Updated: 2019-07-11
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
one year
Results posted on
2019-07-11
Participant Flow
Participant milestones
| Measure |
Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Stem Cell Transplant w/Laronidase for Hurler
Baseline characteristics by cohort
| Measure |
Laronidase ERT Treatment
n=22 Participants
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
|
Age, Categorical
<=18 years
|
22 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
22 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: one yearOutcome measures
| Measure |
Laronidase ERT Treatment
n=22 Participants
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
|
Number of Patients Alive at One Year Post Transplant
|
18 Participants
|
PRIMARY outcome
Timeframe: one yearOutcome measures
| Measure |
Laronidase ERT Treatment
n=22 Participants
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
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|---|---|
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Number of Patients Requiring Ventilator Support at One Year Post Transplant
|
6 Participants
|
SECONDARY outcome
Timeframe: Day 100 post transplantOutcome measures
| Measure |
Laronidase ERT Treatment
n=22 Participants
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
|
Donor Engraftment
|
21 Participants
|
SECONDARY outcome
Timeframe: Day 100 post transplantOutcome measures
| Measure |
Laronidase ERT Treatment
n=22 Participants
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
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|---|---|
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Patients With Grade III-IV Acute GVHD
|
1 Participants
|
SECONDARY outcome
Timeframe: Prior to, During and After ERTPopulation: Data was not collected on this outcome measure and is not available for reporting.
Data was not collected on this outcome measure and is not available for reporting.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 year post transplantPopulation: Data was not collected on this outcome measure and is not available for reporting.
Data was not collected on this outcome measure and is not available for reporting.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 YearPopulation: Data was not collected on this outcome measure and is not available for reporting.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 12 weeks after laronidase, after transplantPopulation: Data was not collected on this outcome measure and is not available for reporting.
Outcome measures
Outcome data not reported
Adverse Events
Laronidase ERT Treatment
Serious events: 6 serious events
Other events: 21 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Laronidase ERT Treatment
n=22 participants at risk
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
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Cardiac disorders
Heart failure
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Cardiac disorders
Pericardial effusion
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
General disorders
Multiple system organ failure
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Other, Infection NOS
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Catheter-related infection
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Other, Upper respiratory Tract infection
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Nervous system disorders
Multifocal cerebral infarct
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
Other adverse events
| Measure |
Laronidase ERT Treatment
n=22 participants at risk
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Stem Cell Transplant: enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT: Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
|
|---|---|
|
Hepatobiliary disorders
Increased Liver Function Test
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Blood and lymphatic system disorders
Blood/Bone Marrow, NOS
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Nervous system disorders
Cerebrovascular Ischemia
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Cardiac disorders
Cardiac, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Hepatobiliary disorders
Cholecystitis
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Gastrointestinal disorders
Dehydration
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Gastrointestinal disorders
Diarrhea
|
18.2%
4/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
General disorders
Fatigue
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Musculoskeletal and connective tissue disorders
Fracture, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Blood and lymphatic system disorders
Hemoglobin, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Blood and lymphatic system disorders
Hemolysis
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, Pulmonary
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Infection with Grade 3 or 4 Neutrophils
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Infection, NOS
|
13.6%
3/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Infection with Normal Neutrophils
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Infections and infestations
Infection with Unknown Neutrophils
|
59.1%
13/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Mucositis, Pharynx
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Gastrointestinal disorders
Necrosis, GI
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Nervous system disorders
Neurology, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Renal and urinary disorders
Obstruction, Ureter
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Gastrointestinal disorders
Pain, Abdomen
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Cardiac disorders
Pain, Cardiac
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary, NOS
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Skin and subcutaneous tissue disorders
Rash/Desquamation
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Renal and urinary disorders
Renal, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Cardiac disorders
Supraventricular and Nodal Arrythmia
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Nervous system disorders
Syncope
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Vascular disorders
Thrombosis
|
9.1%
2/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
|
Vascular disorders
Vascular, NOS
|
4.5%
1/22
An SAE as defined by the protocol includes unexpected: 1) laronidase related toxicity which is life threatening, requires hospitalization, causes persistent or significant disability/incapacity, causes congenital anomaly, is a medical event that may jeopardize the patent and require medical/surgical intervention to prevent one of the other outcomes, or causes death; 2) graft failure; 3) severe acute GVHD; and 4) severe regimen related toxicity grade III-IV, which includes mortality.
|
Additional Information
Dr. Paul Orchard
Masonic Cancer Center, University of Minnesota
Phone: (612) 626-2313
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place