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Stem Cell Transplant w/Laronidase for Hurler

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ClinicalTrials.gov Identifier: NCT00176891
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : July 11, 2019
Last Update Posted : July 11, 2019
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis I
Hurler Syndrome
Interventions Procedure: Stem Cell Transplant
Drug: Laronidase ERT
Enrollment 25
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 25
Completed 22
Not Completed 3
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description

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 Number of Baseline Participants 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
<=18 years
22
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
11
  50.0%
Male
11
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 22 participants
22
1.Primary Outcome
Title Number of Patients Alive at One Year Post Transplant
Hide Description [Not Specified]
Time Frame one year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
18
  81.8%
2.Primary Outcome
Title Number of Patients Requiring Ventilator Support at One Year Post Transplant
Hide Description [Not Specified]
Time Frame one year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
6
  27.3%
3.Secondary Outcome
Title Donor Engraftment
Hide Description [Not Specified]
Time Frame Day 100 post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
21
  95.5%
4.Secondary Outcome
Title Patients With Grade III-IV Acute GVHD
Hide Description [Not Specified]
Time Frame Day 100 post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
1
   4.5%
5.Secondary Outcome
Title Reduction in Glycosaminoglycans (GAG)
Hide Description Data was not collected on this outcome measure and is not available for reporting.
Time Frame Prior to, During and After ERT
Hide Outcome Measure Data
Hide Analysis Population Description
Data was not collected on this outcome measure and is not available for reporting.
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Toxicity (Adverse Events) Associated With Infusions of Laronidase
Hide Description Data was not collected on this outcome measure and is not available for reporting.
Time Frame 1 year post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
Data was not collected on this outcome measure and is not available for reporting.
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Development of Anti-iduronidase Antibodies in Serum
Hide Description [Not Specified]
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
Data was not collected on this outcome measure and is not available for reporting.
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography
Hide Description [Not Specified]
Time Frame Baseline, 12 weeks after laronidase, after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
Data was not collected on this outcome measure and is not available for reporting.
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description 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.
 
Arm/Group Title Laronidase ERT Treatment
Hide Arm/Group Description

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.

All-Cause Mortality
Laronidase ERT Treatment
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
Laronidase ERT Treatment
Affected / at Risk (%)
Total   6/22 (27.27%) 
Cardiac disorders   
Heart failure  1/22 (4.55%) 
Pericardial effusion  1/22 (4.55%) 
General disorders   
Multiple system organ failure  1/22 (4.55%) 
Infections and infestations   
Other, Infection NOS  2/22 (9.09%) 
Catheter-related infection  1/22 (4.55%) 
Other, Upper respiratory Tract infection  1/22 (4.55%) 
Nervous system disorders   
Multifocal cerebral infarct  1/22 (4.55%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Laronidase ERT Treatment
Affected / at Risk (%)
Total   21/22 (95.45%) 
Blood and lymphatic system disorders   
Blood/Bone Marrow, NOS  2/22 (9.09%) 
Hemoglobin, NOS  1/22 (4.55%) 
Hemolysis  1/22 (4.55%) 
Cardiac disorders   
Cardiac, NOS  1/22 (4.55%) 
Pain, Cardiac  1/22 (4.55%) 
Supraventricular and Nodal Arrythmia  1/22 (4.55%) 
Gastrointestinal disorders   
Dehydration  1/22 (4.55%) 
Diarrhea  4/22 (18.18%) 
Necrosis, GI  1/22 (4.55%) 
Pain, Abdomen  2/22 (9.09%) 
General disorders   
Fatigue  1/22 (4.55%) 
Hepatobiliary disorders   
Increased Liver Function Test  1/22 (4.55%) 
Hyperbilirubinemia  1/22 (4.55%) 
Cholecystitis  1/22 (4.55%) 
Infections and infestations   
Infection with Grade 3 or 4 Neutrophils  1/22 (4.55%) 
Infection, NOS  3/22 (13.64%) 
Infection with Normal Neutrophils  1/22 (4.55%) 
Infection with Unknown Neutrophils  13/22 (59.09%) 
Metabolism and nutrition disorders   
Hypoglycemia  1/22 (4.55%) 
Musculoskeletal and connective tissue disorders   
Fracture, NOS  1/22 (4.55%) 
Nervous system disorders   
Cerebrovascular Ischemia  1/22 (4.55%) 
Neurology, NOS  1/22 (4.55%) 
Syncope  1/22 (4.55%) 
Renal and urinary disorders   
Obstruction, Ureter  1/22 (4.55%) 
Renal, NOS  1/22 (4.55%) 
Respiratory, thoracic and mediastinal disorders   
Acute Respiratory Distress  1/22 (4.55%) 
Dyspnea  1/22 (4.55%) 
Hemorrhage, Pulmonary  1/22 (4.55%) 
Hypoxia  2/22 (9.09%) 
Mucositis, Pharynx  1/22 (4.55%) 
Pulmonary, NOS  2/22 (9.09%) 
Skin and subcutaneous tissue disorders   
Rash/Desquamation  1/22 (4.55%) 
Vascular disorders   
Thrombosis  2/22 (9.09%) 
Vascular, NOS  1/22 (4.55%) 
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.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Paul Orchard
Organization: Masonic Cancer Center, University of Minnesota
Phone: (612) 626-2313
EMail: orcha001@umn.edu
Layout table for additonal information
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176891    
Other Study ID Numbers: MT2004-09
0403M57728 ( Other Identifier: IRB, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: April 6, 2017
Results First Posted: July 11, 2019
Last Update Posted: July 11, 2019