Trial Outcomes & Findings for Stem Cell Transplantation for Children Affected With Osteopetrosis (NCT NCT00145587)

Last Updated: 2017-05-30

Results Overview

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.

Recruitment status

TERMINATED

Study phase

Target enrollment

15 participants

Primary outcome timeframe

100 days post-transplant

Results posted on

2017-05-30

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	Haplo Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).	Sibling Genetic testing
Overall Study STARTED	5	1
Overall Study COMPLETED	5	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation for Children Affected With Osteopetrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Haplo n=5 Participants Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).	Sibling n=1 Participants Genetic testing	Total n=6 Participants Total of all reporting groups
Age, Categorical <=18 years	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days post-transplant

Population: 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.

Outcome measures

Outcome measures
Measure	Haplo n=5 Participants Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).	Sibling n=1 Participants Genetic testing
Engraftment Favorable engraftment	5 Participants	1 Participants
Engraftment Un-favorable engraftment	0 Participants	0 Participants

Adverse Events

Haplo

Serious events: 5 serious events

Other events: 5 other events

Deaths: 0 deaths

Sibling

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Haplo n=5 participants at risk Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).	Sibling n=1 participants at risk Genetic testing
General disorders Fever without Neutropenia	40.0% 2/5 • Number of events 4 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Delayed gastric emptying	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Hemorrhage, Diffuse Alveolar	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, CMV Pneumonitis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Moraxella Catarrhalis, Upper Respiratory	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Respiratory Syncytial Virus	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Rotavirus, Stool	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Staph Aureus, Tracheostomy Site	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome Related To Aspiration Pneumonia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Aspiration Pneumonia Related to Tracheal Bleed	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Hypoxia	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Methicillin Resistant Staphylococcus Aureus Sepsis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.

Other adverse events

Additional Information

Kimberly Kasow, DO

St. Jude Children's Research Hospital

Phone: 901-595-3300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Haplo n=5 participants at risk Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).	Sibling n=1 participants at risk Genetic testing
Immune system disorders Allergic Drug Reaction, OKT3	60.0% 3/5 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Immune system disorders Allergic Drug Reaction, Vancomycin	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Immune system disorders Allergic Reaction, Platelet Transfusion	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Anemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Neutropenia	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Secondary Graft Failure	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Thrombocytopenia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Bradycardia, Asymptomatic	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Prolonged QT interval	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Edema, Lower Extremity	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Hypertension	80.0% 4/5 • Number of events 5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Hypotension	40.0% 2/5 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Cardiac disorders Pulmonary Hypertension	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
General disorders Fever without Neutropenia	60.0% 3/5 • Number of events 6 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Contact dermatitis	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Decubitus ulcer	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Erythema	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Graft Versus Host Disease (GVHD), Chronic, Skin; Limited/	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Rash, Generalized	80.0% 4/5 • Number of events 7 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Transient Erythematous Rash	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Ascites	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Constipation	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Diarrhea	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders GI Reflux	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Mucositis	60.0% 3/5 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Nausea	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Nausea and Vomiting	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Stomatitis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Vomiting	80.0% 4/5 • Number of events 5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Bilateral Subdural Hematomas	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Ear and labyrinth disorders Bleeding from ear	20.0% 1/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Bleeding, Multisite: Oral, Nasal, Hickman Line Site	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Bleeding, Tracheal Granuloma	20.0% 1/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Epistaxis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Hematemesis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Petechiae	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders Elevated GGT	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders Hyperbilirubinemia	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders Hypoalbuminemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders SGOT Measurement, Abnormal Level	40.0% 2/5 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders SGPT Measurement, Abnormal Level	40.0% 2/5 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Hepatobiliary disorders Veno-occlusive disease of the liver	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Adenovirus, Stool	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Adenovirus, Tracheal Aspirate	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Aspergillus, Endotracheal Tube	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Febrile Neutropenia	60.0% 3/5 • Number of events 4 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Bacillus, Hickman Line	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Gastrointestinal disorders Loss of appetite	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Coagulase Negative Staphylococcus, Hickman Line Site	20.0% 1/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Coagulase Negative Staphylococcus, Tracheostomy	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Cytomegalovirus, Tracheal Aspirate	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Enterococcus Faecalis, Urinary Tract	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Infections and infestations Infection, Haemophilus Influenzae, Tracheal Aspirate	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypercalcemia	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hyperglycemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypokalemia	60.0% 3/5 • Number of events 4 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypomagnesemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hyponatremia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypophosphatemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Nervous system disorders Twitching/Staring Episode	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
General disorders Otalgia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Atelectasis	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Dyspnea	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Hypoxia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Infiltrates, Pulmonary	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Respiratory, thoracic and mediastinal disorders Pneumomediastinum	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Renal and urinary disorders Nephromegaly	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Renal and urinary disorders Renal Insufficiency	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypertriglyceridemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypocalcemia	20.0% 1/5 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Metabolism and nutrition disorders Hypoglycemia	0.00% 0/5 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 3 • Adverse events were collected from September, 2004 through February, 2008.
Skin and subcutaneous tissue disorders Graft Versus Host Disease (GVHD), Acute, Skin; Maximum Stage 3, Overall Grade 2	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	0.00% 0/1 • Adverse events were collected from September, 2004 through February, 2008.
Blood and lymphatic system disorders Hematuria	40.0% 2/5 • Number of events 2 • Adverse events were collected from September, 2004 through February, 2008.	100.0% 1/1 • Number of events 1 • Adverse events were collected from September, 2004 through February, 2008.