Trial Outcomes & Findings for Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias (NCT NCT00244010)
NCT ID: NCT00244010
Last Updated: 2017-05-30
Results Overview
The primary objective of this study is to evaluate the safety of HAPLO HSCT for patients with refractory severe aplastic anemia (SAA) or refractory cytopenias. The treatment plan would be considered unsafe if we can demonstrate that it is associated with a significantly higher treatment failure rate. The treatment failure is defined as any occurrence of the following events, overall grade III-IV acute GVHD, graft failure or death due to any cause within 100 days post HSCT or after the last cellular product infusion, if required.
COMPLETED
NA
4 participants
100 days post transplant
2017-05-30
Participant Flow
Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed. The study was terminated due to the PI leaving St. Jude.
Participant milestones
| Measure |
Patients
Patients were enrolled to treat refractory severe aplastic anemia.
|
Donors
Parents of patients were enrolled onto the SAAHAP study to provide hematopoietic stem cells.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Baseline characteristics by cohort
| Measure |
Patients
n=2 Participants
Patients were enrolled to treat refractory severe aplastic anemia.
|
Donors
n=2 Participants
Parents of patients were enrolled onto the SAAHAP study to provide hematopoietic stem cells.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 100 days post transplantPopulation: Enrollment was terminated due to the PI leaving the institution. Insufficient data was generated to answer the objective.
The primary objective of this study is to evaluate the safety of HAPLO HSCT for patients with refractory severe aplastic anemia (SAA) or refractory cytopenias. The treatment plan would be considered unsafe if we can demonstrate that it is associated with a significantly higher treatment failure rate. The treatment failure is defined as any occurrence of the following events, overall grade III-IV acute GVHD, graft failure or death due to any cause within 100 days post HSCT or after the last cellular product infusion, if required.
Outcome measures
Outcome data not reported
Adverse Events
Patients
Donor
Serious adverse events
| Measure |
Patients
n=2 participants at risk
Patients were enrolled to treat refractory severe aplastic anemia.
|
Donor
Donors were not assessed for adverse events.
|
|---|---|---|
|
Infections and infestations
Cellulitis of skin
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Infections and infestations
Infection, Staphylococcus Aureus, Blood
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Investigations
Methemoglobinemia
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Gastrointestinal disorders
Pancreatitis
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Gastrointestinal disorders
Abdominal Pain (intermittent)
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
Other adverse events
| Measure |
Patients
n=2 participants at risk
Patients were enrolled to treat refractory severe aplastic anemia.
|
Donor
Donors were not assessed for adverse events.
|
|---|---|---|
|
Metabolism and nutrition disorders
Elevated Ferritin Level
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
50.0%
1/2 • Number of events 2 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
General disorders
Fever without Neutropenia
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Metabolism and nutrition disorders
Hemosiderosis
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Hepatobiliary disorders
Hepatomegaly (intermittent)
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Gastrointestinal disorders
Nausea and Vomiting (intermittent)
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Musculoskeletal and connective tissue disorders
Pain-Back
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
|
Metabolism and nutrition disorders
Iron overload (intermittent)
|
50.0%
1/2 • Number of events 1 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
—
0/0 • Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
|
Additional Information
Kimberly Kasow, DO
St. Jude Children's Research Hospital
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place