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Haploidentical PBMC Transplant for Severe Congenital Anemias

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ClinicalTrials.gov Identifier: NCT00977691
Recruitment Status : Active, not recruiting
First Posted : September 16, 2009
Results First Posted : October 14, 2019
Last Update Posted : March 29, 2021
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Anemia
Interventions Procedure: PBSC Transplant
Drug: Alemtuzumab
Drug: Sirolimus
Drug: Cyclophosphamide
Procedure: Low Dose Irradiation
Enrollment 23
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and no posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 50 mg/kg posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 100 mg/kg posttransplant cyclophosphamide (PT-Cy).
Period Title: Overall Study
Started 3 8 12
Completed 3 7 12
Not Completed 0 1 0
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Total
Hide Arm/Group Description Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and no posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 50 mg/kg posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 100 mg/kg posttransplant cyclophosphamide (PT-Cy). Total of all reporting groups
Overall Number of Baseline Participants 3 8 12 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 12 participants 23 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
3
 100.0%
8
 100.0%
12
 100.0%
23
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 12 participants 23 participants
Female
2
  66.7%
2
  25.0%
5
  41.7%
9
  39.1%
Male
1
  33.3%
6
  75.0%
7
  58.3%
14
  60.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 12 participants 23 participants
Hispanic or Latino
0
   0.0%
1
  12.5%
1
   8.3%
2
   8.7%
Not Hispanic or Latino
3
 100.0%
7
  87.5%
11
  91.7%
21
  91.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 12 participants 23 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
3
 100.0%
6
  75.0%
11
  91.7%
20
  87.0%
White
0
   0.0%
2
  25.0%
0
   0.0%
2
   8.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
1
   8.3%
1
   4.3%
1.Primary Outcome
Title Patients With Donor Type Hemoglobin
Hide Description Percentage of patients post transplant with sustained donor type hemoglobin on hemoglobin electrophoresis
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and no posttransplant cyclophosphamide (PT-Cy).
Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 50 mg/kg posttransplant cyclophosphamide (PT-Cy).
Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 100 mg/kg posttransplant cyclophosphamide (PT-Cy).
Overall Number of Participants Analyzed 3 8 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
3
  37.5%
6
  50.0%
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and no posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 50 mg/kg posttransplant cyclophosphamide (PT-Cy). Patients received a regimen consisting of alemtuzumab, 400 cGy total body irradiation (TBI), sirolimus, and 100 mg/kg posttransplant cyclophosphamide (PT-Cy).
All-Cause Mortality
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   1/8 (12.50%)   1/12 (8.33%) 
Hide Serious Adverse Events
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   7/8 (87.50%)   11/12 (91.67%) 
Blood and lymphatic system disorders       
Anaemia   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
myelodysplastic syndrome   1/3 (33.33%)  0/8 (0.00%)  1/12 (8.33%) 
Sickle cell anaemia with crisis   0/3 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
Cardiac disorders       
Cardiovascular disorder   0/3 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Syncope   0/3 (0.00%)  0/8 (0.00%)  2/12 (16.67%) 
ventricular tachycardia   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Endocrine disorders       
hypothyroidism   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Gastrointestinal disorders       
Abdominal pain   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
colitis   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Upper gastrointestinal haemorrhage   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
General disorders       
Device related infection   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Device related thrombosis   1/3 (33.33%)  1/8 (12.50%)  0/12 (0.00%) 
Extravasation   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Inflammation   0/3 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
pain   1/3 (33.33%)  1/8 (12.50%)  2/12 (16.67%) 
Hepatobiliary disorders       
Hepatic haemorrhage   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
portal hypertension   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Immune system disorders       
Hypersensitivity   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Transplant rejection   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Infections and infestations       
Cytomegalovirus infection   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Infection   0/3 (0.00%)  4/8 (50.00%)  6/12 (50.00%) 
Infective Myositis   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Lung infection   1/3 (33.33%)  0/8 (0.00%)  2/12 (16.67%) 
Sepsis   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Upper respiratory tract infection   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Injury, poisoning and procedural complications       
Fall   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Metabolism and nutrition disorders       
Dehydration   1/3 (33.33%)  1/8 (12.50%)  0/12 (0.00%) 
Musculoskeletal and connective tissue disorders       
arthralgia   1/3 (33.33%)  0/8 (0.00%)  0/12 (0.00%) 
Musculoskeletal disorder   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Nervous system disorders       
Cerebrovascular accident   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Headache   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
memory impairment   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Seizure   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Pregnancy, puerperium and perinatal conditions       
pregnancy   0/3 (0.00%)  0/8 (0.00%)  1/12 (8.33%) 
Respiratory, thoracic and mediastinal disorders       
Pneumothorax   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Pulmonary Embolism   1/3 (33.33%)  2/8 (25.00%)  0/12 (0.00%) 
Pulmonary Hypertension   1/3 (33.33%)  0/8 (0.00%)  0/12 (0.00%) 
Pulmonary mass   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Vascular disorders       
Embolism   0/3 (0.00%)  1/8 (12.50%)  1/12 (8.33%) 
Thrombosis   0/3 (0.00%)  1/8 (12.50%)  0/12 (0.00%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/8 (0.00%)   0/12 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Fitzhugh, Courtney
Organization: National Heart Lung and Blood Institute
Phone: +1 301 402 6496
EMail: courtney.fitzhugh@nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT00977691    
Other Study ID Numbers: 090225
09-H-0225
First Submitted: September 15, 2009
First Posted: September 16, 2009
Results First Submitted: August 23, 2019
Results First Posted: October 14, 2019
Last Update Posted: March 29, 2021