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Trial record 49 of 154 for:    "Skeletal Dysplasias"

Stem Cell Transplantation for Children Affected With Osteopetrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00145587
Recruitment Status : Terminated (Due to the principal investigator having left the institution.)
First Posted : September 5, 2005
Results First Posted : August 22, 2011
Last Update Posted : May 30, 2017
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Osteopetrosis
Interventions Procedure: Stem Cell Transplantation
Device: Miltenyi Biotec CliniMACS
Drug: Systemic chemotherapy and antibodies
Enrollment 15
Recruitment Details OPBMT2 was activated, July 2004. From September, 2004 through February, 2008, six transplant participants, five donors, and four genetic testing participants were recruited and enrolled on the study.
Pre-assignment Details Of the fifteen enrollments, the donors and genetic-testing participants did not receive transplants. Of the six transplant participants, five where eligible for and received a haploidentical hematopoietic stem cell transplant (HSCT) and one received a sibling donor HSCT.
Arm/Group Title Haplo Sibling
Hide Arm/Group Description Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT). Genetic testing
Period Title: Overall Study
Started 5 1
Completed 5 1
Not Completed 0 0
Arm/Group Title Haplo Sibling Total
Hide Arm/Group Description Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT). Genetic testing Total of all reporting groups
Overall Number of Baseline Participants 5 1 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 1 participants 6 participants
<=18 years
5
 100.0%
1
 100.0%
6
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 1 participants 6 participants
Female
1
  20.0%
1
 100.0%
2
  33.3%
Male
4
  80.0%
0
   0.0%
4
  66.7%
1.Primary Outcome
Title Engraftment
Hide Description To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient’s bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.
Time Frame 100 days post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
From September 2004 to March 2009, 5 consecutive MIOP patients were treated using mismatched family member donors. Favorable engraftment refers to the transplant patient not requiring blood or platelet transfusions and the presence of donor cells being present in the transplant recipient’s bone marrow or peripheral blood.
Arm/Group Title Haplo Sibling
Hide Arm/Group Description:
Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).
Genetic testing
Overall Number of Participants Analyzed 5 1
Measure Type: Number
Unit of Measure: Participants
Favorable engraftment 5 1
Un-favorable engraftment 0 0
Time Frame Adverse events were collected from September, 2004 through February, 2008.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Haplo Sibling
Hide Arm/Group Description Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT). Genetic testing
All-Cause Mortality
Haplo Sibling
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Haplo Sibling
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/5 (100.00%)      1/1 (100.00%)    
Gastrointestinal disorders     
Delayed gastric emptying * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
General disorders     
Fever without Neutropenia * 1  2/5 (40.00%)  4 0/1 (0.00%)  0
Infections and infestations     
Infection, CMV Pneumonitis * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Moraxella Catarrhalis, Upper Respiratory * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Respiratory Syncytial Virus * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Rotavirus, Stool * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Staph Aureus, Tracheostomy Site * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Methicillin Resistant Staphylococcus Aureus Sepsis * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Hemorrhage, Diffuse Alveolar * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Adult Respiratory Distress Syndrome Related To Aspiration Pneumonia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Aspiration Pneumonia Related to Tracheal Bleed * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hypoxia * 1  2/5 (40.00%)  2 1/1 (100.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (2.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Haplo Sibling
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/5 (100.00%)      1/1 (100.00%)    
Blood and lymphatic system disorders     
Anemia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Neutropenia * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Secondary Graft Failure * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Thrombocytopenia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Bilateral Subdural Hematomas * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Bleeding, Multisite: Oral, Nasal, Hickman Line Site * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Bleeding, Tracheal Granuloma * 1  1/5 (20.00%)  2 0/1 (0.00%)  0
Epistaxis * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hematemesis * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Petechiae * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Hematuria * 1  2/5 (40.00%)  2 1/1 (100.00%)  1
Cardiac disorders     
Bradycardia, Asymptomatic * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Prolonged QT interval * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Edema, Lower Extremity * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hypertension * 1  4/5 (80.00%)  5 1/1 (100.00%)  1
Hypotension * 1  2/5 (40.00%)  3 1/1 (100.00%)  1
Pulmonary Hypertension * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Ear and labyrinth disorders     
Bleeding from ear * 1  1/5 (20.00%)  2 0/1 (0.00%)  0
Gastrointestinal disorders     
Ascites * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Constipation * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Diarrhea * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
GI Reflux * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Mucositis * 1  3/5 (60.00%)  3 1/1 (100.00%)  1
Nausea * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Nausea and Vomiting * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Stomatitis * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Vomiting * 1  4/5 (80.00%)  5 1/1 (100.00%)  1
Loss of appetite * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
General disorders     
Fever without Neutropenia * 1  3/5 (60.00%)  6 1/1 (100.00%)  2
Otalgia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hepatobiliary disorders     
Elevated GGT * 1  1/5 (20.00%)  1 1/1 (100.00%)  2
Hyperbilirubinemia * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Hypoalbuminemia * 1  1/5 (20.00%)  1 1/1 (100.00%)  2
SGOT Measurement, Abnormal Level * 1  2/5 (40.00%)  3 1/1 (100.00%)  1
SGPT Measurement, Abnormal Level * 1  2/5 (40.00%)  3 1/1 (100.00%)  1
Veno-occlusive disease of the liver * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Immune system disorders     
Allergic Drug Reaction, OKT3 * 1  3/5 (60.00%)  3 0/1 (0.00%)  0
Allergic Drug Reaction, Vancomycin * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Allergic Reaction, Platelet Transfusion * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infections and infestations     
Infection, Adenovirus, Stool * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Infection, Adenovirus, Tracheal Aspirate * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Infection, Aspergillus, Endotracheal Tube * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Febrile Neutropenia * 1  3/5 (60.00%)  4 0/1 (0.00%)  0
Infection, Bacillus, Hickman Line * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Coagulase Negative Staphylococcus, Hickman Line Site * 1  1/5 (20.00%)  2 0/1 (0.00%)  0
Infection, Coagulase Negative Staphylococcus, Tracheostomy * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Infection, Cytomegalovirus, Tracheal Aspirate * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Infection, Enterococcus Faecalis, Urinary Tract * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Infection, Haemophilus Influenzae, Tracheal Aspirate * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Metabolism and nutrition disorders     
Hypercalcemia * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Hyperglycemia * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Hypokalemia * 1  3/5 (60.00%)  4 1/1 (100.00%)  2
Hypomagnesemia * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Hyponatremia * 1  1/5 (20.00%)  1 1/1 (100.00%)  2
Hypophosphatemia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hypertriglyceridemia * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Hypocalcemia * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hypoglycemia * 1  0/5 (0.00%)  0 1/1 (100.00%)  3
Nervous system disorders     
Twitching/Staring Episode * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Renal and urinary disorders     
Nephromegaly * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Renal Insufficiency * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Atelectasis * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Dyspnea * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Hypoxia * 1  1/5 (20.00%)  1 1/1 (100.00%)  1
Infiltrates, Pulmonary * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Pneumomediastinum * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Skin and subcutaneous tissue disorders     
Contact dermatitis * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Decubitus ulcer * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Erythema * 1  1/5 (20.00%)  1 0/1 (0.00%)  0
Graft Versus Host Disease (GVHD), Chronic, Skin; Limited/ * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
Rash, Generalized * 1  4/5 (80.00%)  7 0/1 (0.00%)  0
Transient Erythematous Rash * 1  0/5 (0.00%)  0 1/1 (100.00%)  1
Graft Versus Host Disease (GVHD), Acute, Skin; Maximum Stage 3, Overall Grade 2 * 1  2/5 (40.00%)  2 0/1 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (2.0)
This study closed prior to completion of enrollment due to the principal investigator having left the institution.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Kimberly Kasow, DO
Organization: St. Jude Children's Research Hospital
Phone: 901-595-3300
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00145587     History of Changes
Other Study ID Numbers: OPBMT2
First Submitted: September 1, 2005
First Posted: September 5, 2005
Results First Submitted: June 22, 2011
Results First Posted: August 22, 2011
Last Update Posted: May 30, 2017