Trial Outcomes & Findings for A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure (NCT NCT00001962)
NCT ID: NCT00001962
Last Updated: 2021-07-09
Results Overview
Number of participants with hematologic response at 3 months following Daclizumab, 1 mg/kg, 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. 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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
100 participants
Primary outcome timeframe
3 months
Results posted on
2021-07-09
Participant Flow
Participant milestones
| Measure |
Daclizumab in Bone Marrow Failure Syndromes
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Overall Study
STARTED
|
100
|
|
Overall Study
COMPLETED
|
72
|
|
Overall Study
NOT COMPLETED
|
28
|
Reasons for withdrawal
| Measure |
Daclizumab in Bone Marrow Failure Syndromes
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Overall Study
Lack of Efficacy
|
18
|
|
Overall Study
Lack of Accrual
|
6
|
|
Overall Study
Death
|
1
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Baseline characteristics by cohort
| Measure |
Daclizumab in Bone Marrow Failure Syndromes
n=100 Participants
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Age, Categorical
<=18 years
|
27 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
57 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
16 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
49 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
51 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 3 monthsPopulation: 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. Relapse and Refractory Severe Aplastic Anemia (SAA) was closed to lack of efficacy.
Number of participants with hematologic response at 3 months following Daclizumab, 1 mg/kg, 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. 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.
Outcome measures
| Measure |
Daclizumab in Participants With a Bone Marrow Failure Syndrome
n=72 Participants
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
MAA Complete response
|
6 participants
|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
MAA Partial response
|
13 participants
|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
MAA No response
|
26 participants
|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
PRCA Complete response
|
6 participants
|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
PRCA Partial response
|
4 participants
|
|
Number of Participants With Hematologic Response Following Daclizumab in Patients With Moderate Aplastic Anemia (MAA) and Pure Red Cell Aplasia (PRCA).
PRCA No response
|
17 participants
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: This was evaluated for subjects diagnosed with moderate aplastic anemia (MAA) and pure red cell aplasia (PRCA). The Diamond Blackfan anemia arm was closed due to the lack of accrual. The Relapse and Refractory Severe Aplastic Anemia (SAA) was closed due to lack of efficacy.
Number of participants that no longer required blood transfusion after receiving Daclizumab, 1 mg/kg, 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.
Outcome measures
| Measure |
Daclizumab in Participants With a Bone Marrow Failure Syndrome
n=72 Participants
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Number of Participants That no Longer Required Blood Transfusion
MAA
|
7 participants
|
|
Number of Participants That no Longer Required Blood Transfusion
PRCA
|
28 participants
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: This was evaluated for subjects diagnosed with moderate aplastic anemia (MAA) and pure red cell aplasia (PRCA). The Diamond Blackfan anemia arm was closed due to the lack of accrual. The Relapse and Refractory Severe Aplastic Anemia (SAA) was closed due to lack of efficacy.
Overall Survival at end of study after receiving Daclizumab, 1 mg/kg, 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.
Outcome measures
| Measure |
Daclizumab in Participants With a Bone Marrow Failure Syndrome
n=72 Participants
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Overall Survival
Deaths
|
7 Participants
|
|
Overall Survival
Alive
|
65 Participants
|
Adverse Events
Daclizumab in Participants With a Bone Marrow Failure Syndrome
Serious events: 9 serious events
Other events: 40 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Daclizumab in Participants With a Bone Marrow Failure Syndrome
n=100 participants at risk
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Immune system disorders
infection
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Renal and urinary disorders
renal failure
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
hepatocellular carcinoma
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Hepatobiliary disorders
elevated liver function tests
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
polycythemia
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Cardiac disorders
angina
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Infections and infestations
sinusitis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Infections and infestations
gastroenteritis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
myeloma
|
1.0%
1/100 • Number of events 1 • 5 years
|
Other adverse events
| Measure |
Daclizumab in Participants With a Bone Marrow Failure Syndrome
n=100 participants at risk
Daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions in participants with a bone marrow failure syndrome. These subjects may be 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.
|
|---|---|
|
Infections and infestations
shortness of breath
|
9.0%
9/100 • Number of events 9 • 5 years
|
|
General disorders
headache
|
11.0%
11/100 • Number of events 12 • 5 years
|
|
Gastrointestinal disorders
diarrhea
|
7.0%
7/100 • Number of events 7 • 5 years
|
|
Musculoskeletal and connective tissue disorders
pain
|
6.0%
6/100 • Number of events 6 • 5 years
|
|
Skin and subcutaneous tissue disorders
dry skin
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Cardiac disorders
edema
|
14.0%
14/100 • Number of events 14 • 5 years
|
|
General disorders
fatigue
|
5.0%
5/100 • Number of events 5 • 5 years
|
|
General disorders
fever
|
4.0%
4/100 • Number of events 4 • 5 years
|
|
Skin and subcutaneous tissue disorders
eczema
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Eye disorders
blurred vision
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Skin and subcutaneous tissue disorders
alopecia
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
rash
|
14.0%
14/100 • Number of events 15 • 5 years
|
|
General disorders
pallor
|
4.0%
4/100 • Number of events 4 • 5 years
|
|
Gastrointestinal disorders
abdominal pain
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Endocrine disorders
gynecomastia
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
nausea
|
11.0%
11/100 • Number of events 11 • 5 years
|
|
Gastrointestinal disorders
mouth sores
|
7.0%
7/100 • Number of events 7 • 5 years
|
|
Blood and lymphatic system disorders
gum bleeding
|
9.0%
9/100 • Number of events 9 • 5 years
|
|
General disorders
back pain
|
6.0%
6/100 • Number of events 6 • 5 years
|
|
Musculoskeletal and connective tissue disorders
tendonitis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Nervous system disorders
lightheadness
|
4.0%
4/100 • Number of events 4 • 5 years
|
|
Nervous system disorders
depression
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
General disorders
knee pain
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Infections and infestations
upper respiratory tract infection
|
40.0%
40/100 • Number of events 48 • 5 years
|
|
Infections and infestations
urinary tract infection
|
6.0%
6/100 • Number of events 6 • 5 years
|
|
General disorders
hepatic pain
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Musculoskeletal and connective tissue disorders
arthritis
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
2.0%
2/100 • Number of events 4 • 5 years
|
|
Infections and infestations
viral infection
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
General disorders
myalgia
|
6.0%
6/100 • Number of events 6 • 5 years
|
|
Gastrointestinal disorders
constipation
|
5.0%
5/100 • Number of events 5 • 5 years
|
|
Cardiac disorders
palpitations
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
General disorders
dizziness
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
General disorders
weight gain
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Skin and subcutaneous tissue disorders
sclerodactyly
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Endocrine disorders
hyperparathyroid
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
petechiae
|
13.0%
13/100 • Number of events 13 • 5 years
|
|
Musculoskeletal and connective tissue disorders
cramps
|
4.0%
4/100 • Number of events 4 • 5 years
|
|
Metabolism and nutrition disorders
decreased potassium
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Ear and labyrinth disorders
hearing loss
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
elevated hemoglobin
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Blood and lymphatic system disorders
hemolysis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Cardiac disorders
lower extremity edema
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Cardiac disorders
peripheral vascular disease
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
rosacea
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Ear and labyrinth disorders
earache
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Immune system disorders
allergic reaction
|
7.0%
7/100 • Number of events 7 • 5 years
|
|
Hepatobiliary disorders
elevated liver function tests
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Infections and infestations
strept throat
|
1.0%
1/100 • Number of events 2 • 5 years
|
|
Immune system disorders
post nasal drip
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
General disorders
diaphoresis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
acne
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
furuncle
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
nail changes
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
skin lesions
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Endocrine disorders
hypothyroidism
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
anorexia
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
esophageal stricture
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
abdominal discomfort
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Gastrointestinal disorders
duodenal ulcer
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
gingival hyperplasia
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
heartburn
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
endometrial benign polyp
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
ear bleeding
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
trauma bleeding
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
vaginal bleeding
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Blood and lymphatic system disorders
blood blisters
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
blood in stool
|
3.0%
3/100 • Number of events 3 • 5 years
|
|
Blood and lymphatic system disorders
bruising
|
9.0%
9/100 • Number of events 9 • 5 years
|
|
Blood and lymphatic system disorders
epistaxis
|
6.0%
6/100 • Number of events 6 • 5 years
|
|
Blood and lymphatic system disorders
heavy menses
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Blood and lymphatic system disorders
retinal hemorrhage
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
subconjunctival hemorrhage
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Infections and infestations
catheter related sepsis
|
1.0%
1/100 • Number of events 2 • 5 years
|
|
Infections and infestations
gastritis
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Infections and infestations
localized infection
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Infections and infestations
strep throat
|
1.0%
1/100 • Number of events 2 • 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
prostate malignancy
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Musculoskeletal and connective tissue disorders
neck stiffness
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Nervous system disorders
anxiety
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Nervous system disorders
insomnia
|
2.0%
2/100 • Number of events 2 • 5 years
|
|
Nervous system disorders
sensory neuropathy
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
Nervous system disorders
vertigo
|
1.0%
1/100 • Number of events 1 • 5 years
|
|
General disorders
bone pain
|
4.0%
4/100 • Number of events 4 • 5 years
|
|
General disorders
joint pain
|
1.0%
1/100 • Number of events 2 • 5 years
|
|
General disorders
leg pain
|
3.0%
3/100 • Number of events 3 • 5 years
|
Additional Information
Neal Young MD
NIH National Heart, Lung and Blood Institute
Phone: 301-496-5093
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place