Trial Outcomes & Findings for Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia (NCT NCT01623167)
NCT ID: NCT01623167
Last Updated: 2025-03-11
Results Overview
Rate of complete hematologic response at six months for cohorts 1, 2 and 3.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE1/PHASE2
Target enrollment
207 participants
Primary outcome timeframe
6 months
Results posted on
2025-03-11
Participant Flow
Recruitment has yet to be completed for the Extension Cohort, which was added to better assess the secondary endpoints
Participant milestones
| Measure |
hATG, CsA, EPAG 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 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
|
hATG, CsA, EPAG Day 14 to Month 3
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
|
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
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 Study
STARTED
|
30
|
31
|
31
|
|
Overall Study
COMPLETED
|
13
|
17
|
18
|
|
Overall Study
NOT COMPLETED
|
17
|
14
|
13
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
Baseline characteristics by cohort
| Measure |
hATG, CsA, EPAG Day 14 to Month 6
n=30 Participants
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
|
hATG, CsA, EPAG Day 14 to Month 3
n=31 Participants
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
|
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
n=31 Participants
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
n=92 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
19 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
61 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
42 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
50 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
17 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
24 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
75 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
16 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
58 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=5 Participants
|
31 participants
n=7 Participants
|
31 participants
n=5 Participants
|
92 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 6 monthsRate of complete hematologic response at six months for cohorts 1, 2 and 3.
Outcome measures
| Measure |
hATG, CsA, EPAG Day 14 to Month 6
n=30 Participants
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
|
hATG, CsA, EPAG Day 14 to Month 3
n=31 Participants
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
|
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
n=31 Participants
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
|
|---|---|---|---|
|
Rate of Complete Hematologic Response
Off study/Not Evaluable
|
3 Participants
|
2 Participants
|
1 Participants
|
|
Rate of Complete Hematologic Response
No Response
|
3 Participants
|
2 Participants
|
1 Participants
|
|
Rate of Complete Hematologic Response
Partial Response
|
14 Participants
|
19 Participants
|
11 Participants
|
|
Rate of Complete Hematologic Response
Complete Response
|
10 Participants
|
8 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: 3 months to 5 yearsSecondary 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.
Outcome measures
Outcome data not reported
Adverse Events
hATG, CsA, EPAG Day 14 to Month 6
Serious events: 17 serious events
Other events: 30 other events
Deaths: 1 deaths
hATG, CsA, EPAG Day 14 to Month 3
Serious events: 15 serious events
Other events: 31 other events
Deaths: 0 deaths
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
Serious events: 14 serious events
Other events: 31 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
hATG, CsA, EPAG Day 14 to Month 6
n=30 participants at risk
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
|
hATG, CsA, EPAG Day 14 to Month 3
n=31 participants at risk
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
|
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
n=31 participants at risk
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
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
3.3%
1/30 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Amnesia
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Immune system disorders
Anaphylaxis (Red blood cells transfusion reaction)
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/30 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Anorectal infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Appendicitis
|
6.7%
2/30 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Blood and lymphatic system disorders
Idiopathic Thrombocytopenic Purpura
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Catheter related infection
|
0.00%
0/30 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
|
Psychiatric disorders
Depression
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Dizziness
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Encephalopathy
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
13.3%
4/30 • Number of events 5 • 6 months
|
16.1%
5/31 • Number of events 8 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Fever
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Gastritis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Paraesophageal hernia, post elective laparascopic surgery
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Viral gastroenteritis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Edema limbs
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Pain (heel)
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Infusion related reaction
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Headache
|
6.7%
2/30 • Number of events 3 • 6 months
|
3.2%
1/31 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
|
Cardiac disorders
Heart failure
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Vascular disorders
Hematoma
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Investigations
Hyperkalemia
|
3.3%
1/30 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Vascular disorders
Hypertension
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Investigations
Hypoglycemia
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Vascular disorders
Hypotension
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Ileal hemorrhage
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Immune system disorders
Autoimmune Hemolyitic Anemia Disorder
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Cellulitis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Stenotrophomanas maltophilia
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Periodontal abscess
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Lung Infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 2 • 6 months
|
|
Infections and infestations
Sinusitis (Invasive Fungal Infection: Dematiaceous of nare/sinus)
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Pseudomonas aeruginosa Bacteremia
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Staphylococcus Epidermidis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Immune system disorders
Infusion related reaction
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Injury, poisoning and procedural complications
Humerus and Tibia Fracture
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Injury, poisoning and procedural complications
Hip fracture, left
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Lung infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 2 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Temperomandibular joint disease
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolisis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Cardiac disorders
Myocardial infarction
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous Cell Carcinoma, keratocantoma type
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Cardiac disorders
Pericardial tamponade
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Pneumonia
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Renal calculi
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Volume overload
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Acute Hypoxic Respiratory Failure
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Sepsis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Immune system disorders
Serum sickness
|
0.00%
0/30 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
12.9%
4/31 • Number of events 4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal Cell Carcinoma
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Skin infection
|
3.3%
1/30 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Laceration
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Nervous system disorders
Syncope
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Vascular disorders
Thromboembolic event
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Tooth infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/30 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Weakness
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
Other adverse events
| Measure |
hATG, CsA, EPAG Day 14 to Month 6
n=30 participants at risk
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
|
hATG, CsA, EPAG Day 14 to Month 3
n=31 participants at risk
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
|
hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6
n=31 participants at risk
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
|
|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
10.0%
3/30 • Number of events 3 • 6 months
|
25.8%
8/31 • Number of events 17 • 6 months
|
35.5%
11/31 • Number of events 17 • 6 months
|
|
Investigations
Alanine aminotransferase increased (intermittent)
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Immune system disorders
Allergic reaction
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Abdominal pain
|
6.7%
2/30 • Number of events 2 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Investigations
Aspartate aminotransferase increased
|
6.7%
2/30 • Number of events 2 • 6 months
|
9.7%
3/31 • Number of events 4 • 6 months
|
22.6%
7/31 • Number of events 8 • 6 months
|
|
Cardiac disorders
Atrial fibrillation
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Investigations
Blood bilirubin increased
|
30.0%
9/30 • Number of events 10 • 6 months
|
29.0%
9/31 • Number of events 10 • 6 months
|
16.1%
5/31 • Number of events 8 • 6 months
|
|
Investigations
Blood bilirubin increased (intermittent)
|
6.7%
2/30 • Number of events 3 • 6 months
|
12.9%
4/31 • Number of events 4 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Eye disorders
Blurred vision
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Investigations
CPK increased
|
3.3%
1/30 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Catheter related infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Cholecystitis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Colitis, intermittent (C. difficile)
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Eye disorders
Conjunctivitis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Constipation
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Psychiatric disorders
Depression
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Ear and labyrinth disorders
Fluid in the mastoid and inner ear
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Ear and labyrinth disorders
Pressure behind ear
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Ear and labyrinth disorders
Ear pain
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Edema limbs
|
6.7%
2/30 • Number of events 2 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Ear and labyrinth disorders
External ear inflammation
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Eye disorders
Eye discoloration
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Eye disorders
Subconjunctival hemorrhage eye
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Eye disorders
Blindness transient
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
16.1%
5/31 • Number of events 5 • 6 months
|
|
General disorders
Fever
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
6.7%
2/30 • Number of events 2 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Reproductive system and breast disorders
Gynecomastia
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
HSV mucositis: Mouth sore (aphthous ulcer)
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Headache
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Gallstones
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Hirsutism
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Vascular disorders
Hypertension
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 3 • 6 months
|
|
Investigations
Hypokalemia
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
|
Investigations
Hyponatremia
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Vascular disorders
Hypotension
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Oral candidiasis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Parainfluenzae virus infection
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Staphylococcus test positive
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Culture stool positive
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
General disorders
Infusion related reaction
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Psychiatric disorders
Insomnia
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Lip infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Lung infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Meningitis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Metabolism and nutrition disorders
Gout
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Mucositis oral
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Smear cervix abnormal
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal Cell Carcinoma
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous Cell Carcinoma
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
General disorders
Non-cardiac chest pain
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Oral hemorrhage
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Ear and labyrinth disorders
Otitis media
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Reproductive system and breast disorders
Ovarian hemorrhage
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Papulopustular rash
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Paronychia
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Pharyngitis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Eye disorders
Photophobia
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Nervous system disorders
Radiculitis
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Eye disorders
Retinopathy
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Eye disorders
Scleral disorder
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Nervous system disorders
Seizure
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Actinic keratosis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Actinic elastosis
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Skin and subcutaneous tissue disorders
Onychocryptosis
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
6.5%
2/31 • Number of events 2 • 6 months
|
|
Infections and infestations
Skin infection
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Skin infection (Folliculitis)
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Surgical and medical procedures
Debridement of nasal septum
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Vascular disorders
Thromboembolic event
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Infections and infestations
Upper respiratory infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Infections and infestations
Urinary tract infection
|
3.3%
1/30 • Number of events 1 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
|
Renal and urinary disorders
Urine discoloration
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Eye disorders
Vitreous hemorrhage
|
3.3%
1/30 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
0.00%
0/31 • 6 months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/30 • 6 months
|
0.00%
0/31 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
|
Investigations
Weight gain
|
0.00%
0/30 • 6 months
|
3.2%
1/31 • Number of events 1 • 6 months
|
0.00%
0/31 • 6 months
|
Additional Information
Neal S. Young, M.D, NIH Principal Investigator
National Heart Lung and Blood Institute (NHLBI)
Phone: 301-496-5093
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place