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A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

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ClinicalTrials.gov Identifier: NCT00001962
Recruitment Status : Terminated (DSMB recommended closing the study and publishing the results)
First Posted : January 19, 2000
Results First Posted : May 14, 2015
Last Update Posted : May 14, 2015
Sponsor:
Information provided by (Responsible Party):
Neal Young, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Aplastic Anemia
Pure Red Cell Aplasia
Diamond Blackfan Anemia
Intervention Drug: Daclizumab
Enrollment 100
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Daclizumab Hematologic Response
Hide Arm/Group Description daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period.The Diamond Blackfan anemia arm was closed due to lack of accrual.
Period Title: Overall Study
Started 100
Completed 72
Not Completed 28
Reason Not Completed
Lack of Efficacy             18
Lack of Accrual             6
Death             1
Lost to Follow-up             2
Adverse Event             1
Arm/Group Title Daclizumab Hematologic Response
Hide Arm/Group Description daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period. The Diamond Blackfan anemia arm was closed due to lack of accrual.
Overall Number of Baseline Participants 100
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 100 participants
<=18 years
27
  27.0%
Between 18 and 65 years
57
  57.0%
>=65 years
16
  16.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 100 participants
Female
49
  49.0%
Male
51
  51.0%
1.Primary Outcome
Title Daclizumab Hematologic Response
Hide Description

Daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions to subjects diagnosed with moderate aplastic anemia (MAA), pure red cell aplasia (PRCA), Diamond Blackfan anemia (DBA), relapse and refractory severe aplastic anemia (SAA) will receive treatment. The Diamond Blackfan anemia arm was closed due to the lack of accrual. The hematologic response will be evaluated at 3 months.

A complete hematologic response will be considered an achievement of normal blood counts. A partial response was defined as any response less than a complete response. The primary endpoint was a hematologic response in at least one affected peripheral blood count parameter, as determined by 3 separate measurements in the first 12 weeks after completion of the infusion.

Time Frame 3 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The daclizumab hematologic response was evaluated for subjects diagnosed with moderate aplastic anemia (MAA) and pure red cell aplasia (PRCA). The Diamond Blackfan arm was closed due to lack of accrual.
Arm/Group Title Daclizumab Hematologic Response
Hide Arm/Group Description:
daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions to subjects diagnosed with moderate aplastic anemia (MAA), pure red cell aplasia (PRCA), Diamond Blackfan anemia (DBA), relapse and refractory severe aplastic anemia (SAA) will receive treatment.
Overall Number of Participants Analyzed 72
Measure Type: Number
Unit of Measure: participants
MAA Complete response 6
MAA Partial response 13
MAA No response 26
PRCA Complete response 6
PRCA Partial response 4
PRCA No response 17
2.Secondary Outcome
Title Change in the Transfusion Requirements, Overall Survival.
Hide Description [Not Specified]
Time Frame 3 and 6 months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Daclizumab Hematologic Response
Hide Arm/Group Description daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period. The Diamond Blackfan anemia arm was closed due to lack of accrual.
All-Cause Mortality
Daclizumab Hematologic Response
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Daclizumab Hematologic Response
Affected / at Risk (%) # Events
Total   9/100 (9.00%)    
Blood and lymphatic system disorders   
polycythemia *  1/100 (1.00%)  1
Cardiac disorders   
angina *  1/100 (1.00%)  1
Hepatobiliary disorders   
elevated liver function tests *  1/100 (1.00%)  1
Immune system disorders   
infection *  2/100 (2.00%)  2
Infections and infestations   
sinusitis *  1/100 (1.00%)  1
gastroenteritis * [1]  1/100 (1.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
hepatocellular carcinoma * [2]  1/100 (1.00%)  1
myeloma *  1/100 (1.00%)  1
Renal and urinary disorders   
renal failure *  1/100 (1.00%)  1
*
Indicates events were collected by non-systematic assessment
[1]
wound- infectious
[2]
Herpes zoster
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Daclizumab Hematologic Response
Affected / at Risk (%) # Events
Total   40/100 (40.00%)    
Blood and lymphatic system disorders   
gum bleeding *  9/100 (9.00%)  9
petechiae *  13/100 (13.00%)  13
elevated hemoglobin *  2/100 (2.00%)  2
hemolysis *  1/100 (1.00%)  1
ear bleeding *  1/100 (1.00%)  1
trauma bleeding *  1/100 (1.00%)  1
vaginal bleeding *  2/100 (2.00%)  2
blood blisters *  1/100 (1.00%)  1
blood in stool *  3/100 (3.00%)  3
bruising *  9/100 (9.00%)  9
epistaxis *  6/100 (6.00%)  6
heavy menses *  2/100 (2.00%)  2
retinal hemorrhage *  1/100 (1.00%)  1
subconjunctival hemorrhage *  1/100 (1.00%)  1
Cardiac disorders   
edema * [1]  14/100 (14.00%)  14
palpitations *  2/100 (2.00%)  2
lower extremity edema *  1/100 (1.00%)  1
peripheral vascular disease *  1/100 (1.00%)  1
Ear and labyrinth disorders   
hearing loss *  1/100 (1.00%)  1
earache *  2/100 (2.00%)  2
Endocrine disorders   
gynecomastia *  1/100 (1.00%)  1
hyperparathyroid *  1/100 (1.00%)  1
hypothyroidism *  1/100 (1.00%)  1
Eye disorders   
blurred vision *  3/100 (3.00%)  3
Gastrointestinal disorders   
diarrhea *  7/100 (7.00%)  7
abdominal pain *  2/100 (2.00%)  2
nausea *  11/100 (11.00%)  11
mouth sores *  7/100 (7.00%)  7
constipation *  5/100 (5.00%)  5
anorexia *  1/100 (1.00%)  1
esophageal stricture * [2]  1/100 (1.00%)  1
abdominal discomfort *  3/100 (3.00%)  3
duodenal ulcer *  1/100 (1.00%)  1
gingival hyperplasia *  1/100 (1.00%)  1
heartburn *  1/100 (1.00%)  1
General disorders   
headache *  11/100 (11.00%)  12
fatigue *  5/100 (5.00%)  5
fever *  4/100 (4.00%)  4
pallor *  4/100 (4.00%)  4
back pain *  6/100 (6.00%)  6
knee pain *  3/100 (3.00%)  3
hepatic pain *  3/100 (3.00%)  3
myalgia *  6/100 (6.00%)  6
dizziness *  3/100 (3.00%)  3
weight gain *  3/100 (3.00%)  3
diaphoresis *  1/100 (1.00%)  1
bone pain *  4/100 (4.00%)  4
joint pain * [3]  1/100 (1.00%)  2
leg pain *  3/100 (3.00%)  3
Hepatobiliary disorders   
elevated liver function tests *  3/100 (3.00%)  3
Immune system disorders   
allergic reaction *  7/100 (7.00%)  7
post nasal drip *  1/100 (1.00%)  1
Infections and infestations   
shortness of breath *  9/100 (9.00%)  9
upper respiratory tract infection *  40/100 (40.00%)  48
urinary tract infection *  6/100 (6.00%)  6
viral infection * [4]  1/100 (1.00%)  1
strept throat *  1/100 (1.00%)  2
catheter related sepsis *  1/100 (1.00%)  2
gastritis *  1/100 (1.00%)  1
localized infection *  4/100 (4.00%)  4
strep throat *  1/100 (1.00%)  2
localized infection * [5]  1/100 (1.00%)  1
Metabolism and nutrition disorders   
decreased potassium *  1/100 (1.00%)  1
Musculoskeletal and connective tissue disorders   
pain *  6/100 (6.00%)  6
tendonitis *  1/100 (1.00%)  1
arthritis *  2/100 (2.00%)  2
cramps *  4/100 (4.00%)  4
neck stiffness *  1/100 (1.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
endometrial benign polyp *  1/100 (1.00%)  1
prostate malignancy *  1/100 (1.00%)  1
Nervous system disorders   
lightheadness *  4/100 (4.00%)  4
depression *  1/100 (1.00%)  1
anxiety *  1/100 (1.00%)  1
insomnia *  2/100 (2.00%)  2
sensory neuropathy *  1/100 (1.00%)  1
vertigo *  1/100 (1.00%)  1
Respiratory, thoracic and mediastinal disorders   
cough *  2/100 (2.00%)  4
Skin and subcutaneous tissue disorders   
dry skin *  1/100 (1.00%)  1
eczema *  1/100 (1.00%)  1
alopecia *  1/100 (1.00%)  1
rash *  14/100 (14.00%)  15
sclerodactyly *  1/100 (1.00%)  1
rosacea *  1/100 (1.00%)  1
acne *  1/100 (1.00%)  1
furuncle *  1/100 (1.00%)  1
nail changes *  1/100 (1.00%)  1
skin lesions *  3/100 (3.00%)  3
*
Indicates events were collected by non-systematic assessment
[1]
periorbital, lower extremity
[2]
Barretts esophageal stricture
[3]
arthralgia
[4]
localized earache
[5]
swollen lymph nodes
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Neal Young MD
Organization: NIH National Heart, Lung and Blood Institute
Phone: 301-496-5093
Responsible Party: Neal Young, M.D., National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00001962     History of Changes
Other Study ID Numbers: 000029
00-H-0029 ( Other Identifier: NIH NHLBI )
First Submitted: January 18, 2000
First Posted: January 19, 2000
Results First Submitted: April 20, 2011
Results First Posted: May 14, 2015
Last Update Posted: May 14, 2015