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Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

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ClinicalTrials.gov Identifier: NCT00244010
Recruitment Status : Completed
First Posted : October 25, 2005
Results First Posted : May 2, 2012
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
Conditions Anemia, Aplastic
Amegakaryocytic Thrombocytopenia
Diamond-Blackfan Anemia
Kostmann Syndrome
Intervention Device: Allogeneic stem cell transplant
Enrollment 4

Recruitment Details 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.
Pre-assignment Details  
Arm/Group Title Patients Donors
Hide Arm/Group Description Patients were enrolled to treat refractory severe aplastic anemia. Parents of patients were enrolled onto the SAAHAP study to provide hematopoietic stem cells.
Period Title: Overall Study
Started 2 2
Completed 2 2
Not Completed 0 0
Arm/Group Title Patients Donors Total
Hide Arm/Group Description Patients were enrolled to treat refractory severe aplastic anemia. Parents of patients were enrolled onto the SAAHAP study to provide hematopoietic stem cells. Total of all reporting groups
Overall Number of Baseline Participants 2 2 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 4 participants
<=18 years
2
 100.0%
0
   0.0%
2
  50.0%
Between 18 and 65 years
0
   0.0%
2
 100.0%
2
  50.0%
>=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 2 participants 2 participants 4 participants
Female
2
 100.0%
1
  50.0%
3
  75.0%
Male
0
   0.0%
1
  50.0%
1
  25.0%
1.Primary Outcome
Title Treatment Failures
Hide Description 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.
Time Frame 100 days post transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Enrollment was terminated due to the PI leaving the institution. Insufficient data was generated to answer the objective.
Arm/Group Title Patients Donors
Hide Arm/Group Description:
Patients were enrolled to treat refractory severe aplastic anemia.
Parents of patients were enrolled onto the SAAHAP study to provide hematopoietic stem cells.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Two patients and two donors were enrolled between 10/24/2005 and 2/24/2009 when the study was closed.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Patients Donor
Hide Arm/Group Description Patients were enrolled to treat refractory severe aplastic anemia. Donors were not assessed for adverse events.
All-Cause Mortality
Patients Donor
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Patients Donor
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      0/0    
Gastrointestinal disorders     
Pancreatitis * 1  1/2 (50.00%)  1 0/0  0
Abdominal Pain (intermittent) * 1  1/2 (50.00%)  1 0/0  0
Infections and infestations     
Cellulitis of skin * 1  1/2 (50.00%)  1 0/0  0
Infection, Staphylococcus Aureus, Blood * 1  1/2 (50.00%)  1 0/0  0
Investigations     
Methemoglobinemia * 1  1/2 (50.00%)  1 0/0  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Patients Donor
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      0/0    
Blood and lymphatic system disorders     
Febrile Neutropenia * 1  1/2 (50.00%)  2 0/0 
Gastrointestinal disorders     
Nausea and Vomiting (intermittent) * 1  1/2 (50.00%)  1 0/0 
General disorders     
Fever without Neutropenia * 1  1/2 (50.00%)  1 0/0 
Hepatobiliary disorders     
Hepatomegaly (intermittent) * 1  1/2 (50.00%)  1 0/0 
Metabolism and nutrition disorders     
Elevated Ferritin Level * 1  1/2 (50.00%)  1 0/0 
Hemosiderosis * 1  1/2 (50.00%)  1 0/0 
Iron overload (intermittent) * 1  1/2 (50.00%)  1 0/0 
Musculoskeletal and connective tissue disorders     
Pain-Back * 1  1/2 (50.00%)  1 0/0 
Nervous system disorders     
Headache * 1  1/2 (50.00%)  1 0/0 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (3.0)
This study has terminated due to the PI leaving the institution. An insufficient number of subjects were enrolled to answer the primary objective.
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: NCT00244010     History of Changes
Other Study ID Numbers: SAAHAP
Severe Aplastic Anemia
Cytopenias
First Submitted: October 24, 2005
First Posted: October 25, 2005
Results First Submitted: February 2, 2012
Results First Posted: May 2, 2012
Last Update Posted: May 30, 2017