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Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

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ClinicalTrials.gov Identifier: NCT01623167
Recruitment Status : Recruiting
First Posted : June 19, 2012
Results First Posted : June 5, 2020
Last Update Posted : June 30, 2020
Sponsor:
Collaborator:
Novartis
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: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Aplastic Anemia
Interventions Drug: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6
Drug: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3
Drug: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
Drug: Extension Cohort
Enrollment 207
Recruitment Details  
Pre-assignment Details Recruitment has yet to be completed for the Extension Cohort, which was added to better assess the secondary endpoints
Arm/Group Title hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Hide Arm/Group Description

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6

hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3

hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3

Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6

hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6

Period Title: Overall Study
Started 30 31 31
Completed 13 17 18
Not Completed 17 14 13
Arm/Group Title hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 Total
Hide Arm/Group Description

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6

hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3

hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3

Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6

hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6

Total of all reporting groups
Overall Number of Baseline Participants 30 31 31 92
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 31 participants 31 participants 92 participants
<=18 years
5
  16.7%
6
  19.4%
8
  25.8%
19
  20.7%
Between 18 and 65 years
20
  66.7%
23
  74.2%
18
  58.1%
61
  66.3%
>=65 years
5
  16.7%
2
   6.5%
5
  16.1%
12
  13.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 31 participants 31 participants 92 participants
Female
14
  46.7%
14
  45.2%
14
  45.2%
42
  45.7%
Male
16
  53.3%
17
  54.8%
17
  54.8%
50
  54.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 31 participants 31 participants 92 participants
Hispanic or Latino
6
  20.0%
2
   6.5%
9
  29.0%
17
  18.5%
Not Hispanic or Latino
24
  80.0%
29
  93.5%
22
  71.0%
75
  81.5%
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 30 participants 31 participants 31 participants 92 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
3
  10.0%
4
  12.9%
1
   3.2%
8
   8.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
5
  16.7%
7
  22.6%
4
  12.9%
16
  17.4%
White
16
  53.3%
19
  61.3%
23
  74.2%
58
  63.0%
More than one race
3
  10.0%
0
   0.0%
3
   9.7%
6
   6.5%
Unknown or Not Reported
3
  10.0%
1
   3.2%
0
   0.0%
4
   4.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 30 participants 31 participants 31 participants 92 participants
30 31 31 92
1.Primary Outcome
Title Rate of Complete Hematologic Response
Hide Description Rate of complete hematologic response at six months for cohorts 1, 2 and 3.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Hide Arm/Group Description:

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6

hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3

hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3

Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6

hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6

Overall Number of Participants Analyzed 30 31 31
Measure Type: Count of Participants
Unit of Measure: Participants
No Response
3
  10.0%
2
   6.5%
1
   3.2%
Partial Response
14
  46.7%
19
  61.3%
11
  35.5%
Complete Response
10
  33.3%
8
  25.8%
18
  58.1%
Off study/Not Evaluable
3
  10.0%
2
   6.5%
1
   3.2%
2.Secondary Outcome
Title Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24.
Hide Description Secondary endpoints will also be evaluated for the study to include: (a) hematological response at 3 and 12 months and yearly thereafter; (b) relapse (c) clonal evolution to PNH, clonal chromosomal population in bone marrow, myelodysplasia by morphology, or acute leukemia; (d) survival; (e) health-related quality of life; (f) hematological response of relapse subjects that re-start treatment; and (g) affects of a 2.0mg/kg/day CsA dose starting month 6 for 18 months until month 24 on the rate of relapse of subjects deemed responders at month 6.
Time Frame 3 months to 5 years
Outcome Measure Data Not Reported
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Hide Arm/Group Description

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6

hATG/CsA /eltrombopag -Cohort 1: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 6

Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3

hATG/CsA /eltrombopag Cohort 2: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months, eltrombopag (experimental) administered Day 14 to month 3

Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6

hATG/CsA /eltrombopag Cohort 3: hATG (standard of care) administered for 4 days, CsA (standard of care) administered starting day 1 for 6 months at higher dose, then reduced dose for 18 months, eltrombopag (experimental) administered Day 1 to month 6

All-Cause Mortality
hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/30 (3.33%)      0/31 (0.00%)      0/31 (0.00%)    
Hide Serious Adverse Events
hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   17/30 (56.67%)      15/31 (48.39%)      14/31 (45.16%)    
Blood and lymphatic system disorders       
Anemia  1  0/30 (0.00%)  0 2/31 (6.45%)  2 0/31 (0.00%)  0
Idiopathic Thrombocytopenic Purpura  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Febrile neutropenia  1  4/30 (13.33%)  5 5/31 (16.13%)  8 1/31 (3.23%)  1
Cardiac disorders       
Heart failure  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Myocardial infarction  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Pericardial tamponade  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Gastrointestinal disorders       
Abdominal pain  1  1/30 (3.33%)  1 1/31 (3.23%)  1 1/31 (3.23%)  1
Appendicitis  1  2/30 (6.67%)  2 0/31 (0.00%)  0 0/31 (0.00%)  0
Gastritis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Paraesophageal hernia, post elective laparascopic surgery  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Viral gastroenteritis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Colitis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Ileal hemorrhage  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Lower gastrointestinal hemorrhage  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
General disorders       
Fever  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Edema limbs  1  0/30 (0.00%)  0 1/31 (3.23%)  1 1/31 (3.23%)  1
Pain (heel)  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Infusion related reaction  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Immune system disorders       
Anaphylaxis (Red blood cells transfusion reaction)  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Autoimmune Hemolyitic Anemia Disorder  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Infusion related reaction  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Serum sickness  1  0/30 (0.00%)  0 2/31 (6.45%)  2 4/31 (12.90%)  4
Infections and infestations       
Anorectal infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Catheter related infection  1  0/30 (0.00%)  0 2/31 (6.45%)  2 0/31 (0.00%)  0
Cellulitis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Stenotrophomanas maltophilia  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Periodontal abscess  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Lung Infection  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  2
Sinusitis (Invasive Fungal Infection: Dematiaceous of nare/sinus)  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Pseudomonas aeruginosa Bacteremia  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Staphylococcus Epidermidis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Lung infection  1  1/30 (3.33%)  1 1/31 (3.23%)  2 1/31 (3.23%)  1
Pneumonia  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Sepsis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Skin infection  1  1/30 (3.33%)  2 0/31 (0.00%)  0 0/31 (0.00%)  0
Tooth infection  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Upper Respiratory Infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Upper respiratory infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 1/31 (3.23%)  1
Urinary tract infection  1  0/30 (0.00%)  0 2/31 (6.45%)  2 0/31 (0.00%)  0
Injury, poisoning and procedural complications       
Humerus and Tibia Fracture  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Hip fracture, left  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Hip fracture  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Investigations       
Alanine aminotransferase increased  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Aspartate aminotransferase increased  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Hyperkalemia  1  1/30 (3.33%)  2 0/31 (0.00%)  0 0/31 (0.00%)  0
Hypoglycemia  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Muscle weakness lower limb  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Temperomandibular joint disease  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Rhabdomyolisis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Weakness  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Squamous Cell Carcinoma, keratocantoma type  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Basal Cell Carcinoma  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Nervous system disorders       
Amnesia  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Dizziness  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Encephalopathy  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Headache  1  2/30 (6.67%)  3 1/31 (3.23%)  2 0/31 (0.00%)  0
Syncope  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Psychiatric disorders       
Depression  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Renal and urinary disorders       
Acute kidney injury  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  2
Acute kidney injury  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Renal calculi  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Epistaxis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Pulmonary edema  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Cough  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Volume overload  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Acute Hypoxic Respiratory Failure  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Sinusitis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Skin and subcutaneous tissue disorders       
Rash maculo-papular  1  0/30 (0.00%)  0 1/31 (3.23%)  1 1/31 (3.23%)  1
Skin ulceration  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Laceration  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Vascular disorders       
Hematoma  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  1
Hypertension  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  1
Hypotension  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Thromboembolic event  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
hATG, CsA, EPAG Day 14 to Month 6 hATG, CsA, EPAG Day 14 to Month 3 hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   30/30 (100.00%)      31/31 (100.00%)      31/31 (100.00%)    
Blood and lymphatic system disorders       
Febrile neutropenia  1  0/30 (0.00%)  0 1/31 (3.23%)  1 5/31 (16.13%)  5
Cardiac disorders       
Atrial fibrillation  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Ear and labyrinth disorders       
Fluid in the mastoid and inner ear  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Pressure behind ear  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Ear pain  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
External ear inflammation  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Otitis media  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Eye disorders       
Blurred vision  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Conjunctivitis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Eye discoloration  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Subconjunctival hemorrhage eye  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Blindness transient  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Photophobia  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Retinopathy  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Scleral disorder  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Vitreous hemorrhage  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Gastrointestinal disorders       
Abdominal pain  1  2/30 (6.67%)  2 1/31 (3.23%)  1 1/31 (3.23%)  1
Cholecystitis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Colitis, intermittent (C. difficile)  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Constipation  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Diarrhea  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Gastroesophageal reflux disease  1  2/30 (6.67%)  2 1/31 (3.23%)  1 0/31 (0.00%)  0
Gallstones  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Mucositis oral  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  1
Nausea  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Oral hemorrhage  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Toothache  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Vomiting  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
General disorders       
Edema limbs  1  2/30 (6.67%)  2 0/31 (0.00%)  0 0/31 (0.00%)  0
Fever  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Infusion related reaction  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Non-cardiac chest pain  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Immune system disorders       
Allergic reaction  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Infections and infestations       
Catheter related infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 1/31 (3.23%)  1
HSV mucositis: Mouth sore (aphthous ulcer)  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Oral candidiasis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Parainfluenzae virus infection  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Staphylococcus test positive  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Culture stool positive  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Lip infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Lung infection  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Meningitis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Paronychia  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Pharyngitis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Skin infection  1  0/30 (0.00%)  0 1/31 (3.23%)  1 1/31 (3.23%)  1
Skin infection (Folliculitis)  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Upper respiratory infection  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Urinary tract infection  1  1/30 (3.33%)  1 1/31 (3.23%)  1 0/31 (0.00%)  0
Investigations       
Alanine aminotransferase increased  1  3/30 (10.00%)  3 8/31 (25.81%)  17 11/31 (35.48%)  17
Alanine aminotransferase increased (intermittent)  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Aspartate aminotransferase increased  1  2/30 (6.67%)  2 3/31 (9.68%)  4 7/31 (22.58%)  8
Blood bilirubin increased  1  9/30 (30.00%)  10 9/31 (29.03%)  10 5/31 (16.13%)  8
Blood bilirubin increased (intermittent)  1  2/30 (6.67%)  3 4/31 (12.90%)  4 1/31 (3.23%)  1
CPK increased  1  1/30 (3.33%)  1 1/31 (3.23%)  1 0/31 (0.00%)  0
Hypokalemia  1  0/30 (0.00%)  0 0/31 (0.00%)  0 2/31 (6.45%)  2
Hyponatremia  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Weight gain  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Metabolism and nutrition disorders       
Gout  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Musculoskeletal and connective tissue disorders       
Back pain  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Smear cervix abnormal  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Basal Cell Carcinoma  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Squamous Cell Carcinoma  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Nervous system disorders       
Headache  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Memory impairment  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Radiculitis  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Seizure  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Psychiatric disorders       
Depression  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Insomnia  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Suicidal ideation  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Renal and urinary disorders       
Acute kidney injury  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Urine discoloration  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Reproductive system and breast disorders       
Gynecomastia  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Ovarian hemorrhage  1  0/30 (0.00%)  0 1/31 (3.23%)  1 0/31 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Epistaxis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Nasal congestion  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Pleural effusion  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Sore throat  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  1
Skin and subcutaneous tissue disorders       
Hirsutism  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Papulopustular rash  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Pruritus  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Rash acneiform  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Actinic keratosis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Actinic elastosis  1  1/30 (3.33%)  1 0/31 (0.00%)  0 0/31 (0.00%)  0
Onychocryptosis  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Skin hyperpigmentation  1  0/30 (0.00%)  0 0/31 (0.00%)  0 2/31 (6.45%)  2
Surgical and medical procedures       
Debridement of nasal septum  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
Vascular disorders       
Hypertension  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  3
Hypotension  1  1/30 (3.33%)  1 0/31 (0.00%)  0 1/31 (3.23%)  1
Thromboembolic event  1  0/30 (0.00%)  0 0/31 (0.00%)  0 1/31 (3.23%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
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: Neal S. Young, M.D, NIH Principal Investigator
Organization: National Heart Lung and Blood Institute (NHLBI)
Phone: 301-496-5093
EMail: youngns@nhlbi.nih.gov
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01623167    
Other Study ID Numbers: 120150
12-H-0150
First Submitted: June 14, 2012
First Posted: June 19, 2012
Results First Submitted: May 18, 2020
Results First Posted: June 5, 2020
Last Update Posted: June 30, 2020