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Trial record 58 of 141 for:    MPL

A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients

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ClinicalTrials.gov Identifier: NCT00922883
Recruitment Status : Active, not recruiting
First Posted : June 17, 2009
Results First Posted : July 16, 2018
Last Update Posted : March 20, 2019
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 Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Anemia, Aplastic
Anemia, Hypoplastic
Thrombocytopenia
Intervention Drug: Eltrombopag
Enrollment 43
Recruitment Details  
Pre-assignment Details 44 patients were consented, but 1 patient was removedl before treatment with the investigational agent was initiated because an additional review of baseline bone marrow revealed that the patient was not eligible, because the patient had changes in the bone marrow inconsistent with a diagnosis of aplastic anemia.
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
Period Title: Overall Study
Started 43
Completed 43
Not Completed 0
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
Overall Number of Baseline Participants 44
Hide Baseline Analysis Population Description
Note that baseline information was available on the 44 patients consented and enrolled, but one patient was removed from protocol before initiation of the investigative agent, and thus adverse events and results information refers only to 43 patients
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 44 participants
<=18 years
4
   9.1%
Between 18 and 65 years
27
  61.4%
>=65 years
13
  29.5%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 44 participants
Female
19
  43.2%
Male
25
  56.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 44 participants
Hispanic or Latino
11
  25.0%
Not Hispanic or Latino
33
  75.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 44 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   2.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
11
  25.0%
White
21
  47.7%
More than one race
3
   6.8%
Unknown or Not Reported
8
  18.2%
1.Primary Outcome
Title The Portion of Drug Responders as Defined by Hematologic Improvements
Hide Description Defined as unilineage or multilineage recovery by 1 or more of the following: 1) platelet response (increase to 20 × 103/μL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks in those who were transfusion dependent on entry into the protocol); (2) erythroid response (when pretreatment hemoglobin was <9 g/dL, defined as an increase in hemoglobin by 1.5 g/dL or, in transfused patients, a reduction in the units of packed red blood cell transfusions by an absolute number of at least 4 transfusions for 8 consecutive weeks, compared with the pretreatment transfusion number in the previous 8 weeks); and (3) neutrophil response (when pretreatment absolute neutrophil count [ANC] of <0.5 × 103/μL as at least a 100% increase in ANC, or an ANC increase >0.5 × 103/μL, and the toxicity profile as measured using Common Terminology Criteria for Adverse Events).
Time Frame 12-16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who received Eltrombopag were analyzed.
Arm/Group Title Eltrombopag
Hide Arm/Group Description:
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
Overall Number of Participants Analyzed 43
Measure Type: Count of Participants
Unit of Measure: Participants
17
  39.5%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
All-Cause Mortality
Eltrombopag
Affected / at Risk (%)
Total   4/43 (9.30%) 
Show Serious Adverse Events Hide Serious Adverse Events
Eltrombopag
Affected / at Risk (%)
Total   16/43 (37.21%) 
Blood and lymphatic system disorders   
Anaemia   1/43 (2.33%) 
Febrile neutropenia   7/43 (16.28%) 
Neutropenia   1/43 (2.33%) 
Thrombocytopenia   1/43 (2.33%) 
Eye disorders   
Conjunctival haemorrhage   2/43 (4.65%) 
Gastrointestinal disorders   
Abdominal pain   1/43 (2.33%) 
Clostridium difficile colitis   1/43 (2.33%) 
Gingivitis ulcerative   1/43 (2.33%) 
General disorders   
Drug eruption   1/43 (2.33%) 
Pyrexia   1/43 (2.33%) 
Hepatobiliary disorders   
Hepatic neoplasm   1/43 (2.33%) 
Infections and infestations   
Bacteraemia   1/43 (2.33%) 
Influenza   1/43 (2.33%) 
Sepsis   3/43 (6.98%) 
Urinary tract infection   1/43 (2.33%) 
Investigations   
Aspartate aminotransferase increased   1/43 (2.33%) 
Vascular disorders   
Deep vein thrombosis   1/43 (2.33%) 
Hypotension   1/43 (2.33%) 
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Eltrombopag
Affected / at Risk (%)
Total   38/43 (88.37%) 
Blood and lymphatic system disorders   
Febrile neutropenia   3/43 (6.98%) 
Hyperbilirubinaemia   1/43 (2.33%) 
Jaundice   1/43 (2.33%) 
Petechiae   3/43 (6.98%) 
Purpura   1/43 (2.33%) 
Cardiac disorders   
Chest pain   2/43 (4.65%) 
Dizziness   4/43 (9.30%) 
Dyspnoea   1/43 (2.33%) 
Dyspnoea exertional   2/43 (4.65%) 
Eye disorders   
Cataract   1/43 (2.33%) 
Conjunctival haemorrhage   2/43 (4.65%) 
Dry eye   2/43 (4.65%) 
Eye contusion   1/43 (2.33%) 
Eye irritation   1/43 (2.33%) 
Myopia   1/43 (2.33%) 
Ocular hyperaemia   1/43 (2.33%) 
Vision blurred   1/43 (2.33%) 
Vitreous floaters   1/43 (2.33%) 
Gastrointestinal disorders   
Abdominal discomfort   2/43 (4.65%) 
Abdominal injury   3/43 (6.98%) 
Abdominal pain   4/43 (9.30%) 
Anal fissure   1/43 (2.33%) 
Anorectal discomfort   1/43 (2.33%) 
Constipation   1/43 (2.33%) 
Diarrhoea   4/43 (9.30%) 
Dyspepsia   1/43 (2.33%) 
Gastrointestinal disorder   1/43 (2.33%) 
Gingival bleeding   2/43 (4.65%) 
Gingival disorder   1/43 (2.33%) 
Gingival swelling   1/43 (2.33%) 
Lip ulceration   1/43 (2.33%) 
Nausea   11/43 (25.58%) 
Oral candidiasis   1/43 (2.33%) 
Oropharyngeal pain   4/43 (9.30%) 
Pelvic organ injury   1/43 (2.33%) 
Pericoronitis   1/43 (2.33%) 
Periodontal disease   1/43 (2.33%) 
Tooth abscess   1/43 (2.33%) 
Tooth injury   1/43 (2.33%) 
Toothache   1/43 (2.33%) 
Vomiting   3/43 (6.98%) 
General disorders   
Asthenia   1/43 (2.33%) 
Axillary pain   1/43 (2.33%) 
Catheter site pain   1/43 (2.33%) 
Fatigue   11/43 (25.58%) 
Induration   1/43 (2.33%) 
Injection site infection   1/43 (2.33%) 
Night sweats   1/43 (2.33%) 
Pain   2/43 (4.65%) 
Pyrexia   4/43 (9.30%) 
Hepatobiliary disorders   
Cholecystitis   1/43 (2.33%) 
Hepatitis viral   1/43 (2.33%) 
Immune system disorders   
Hypersensitivity   1/43 (2.33%) 
Rhinitis allergic   2/43 (4.65%) 
Urticaria   2/43 (4.65%) 
Infections and infestations   
Cellulitis   1/43 (2.33%) 
Herpes zoster   1/43 (2.33%) 
Influenza   1/43 (2.33%) 
Kidney infection   1/43 (2.33%) 
Upper respiratory tract infection   8/43 (18.60%) 
Viral infection   1/43 (2.33%) 
Investigations   
Alanine aminotransferase increased   3/43 (6.98%) 
Aspartate aminotransferase increased   2/43 (4.65%) 
Blood creatine phosphokinase increased   1/43 (2.33%) 
Neutrophil count decreased   1/43 (2.33%) 
Weight decreased   1/43 (2.33%) 
Metabolism and nutrition disorders   
Gout   1/43 (2.33%) 
Osteoporosis   1/43 (2.33%) 
Musculoskeletal and connective tissue disorders   
Arthralgia   2/43 (4.65%) 
Back pain   2/43 (4.65%) 
Bone pain   1/43 (2.33%) 
Muscle spasms   5/43 (11.63%) 
Musculoskeletal disorder   1/43 (2.33%) 
Musculoskeletal pain   2/43 (4.65%) 
Pain in extremity   2/43 (4.65%) 
Piriformis syndrome   1/43 (2.33%) 
Synovial cyst   1/43 (2.33%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Ovarian cyst   1/43 (2.33%) 
Seborrhoeic keratosis   1/43 (2.33%) 
Skin cancer   1/43 (2.33%) 
Nervous system disorders   
Headache   7/43 (16.28%) 
Insomnia   3/43 (6.98%) 
Peripheral sensory neuropathy   1/43 (2.33%) 
Psychiatric disorders   
Anxiety   1/43 (2.33%) 
Depressed mood   1/43 (2.33%) 
Depression   1/43 (2.33%) 
Libido decreased   1/43 (2.33%) 
Mood altered   1/43 (2.33%) 
Nightmare   1/43 (2.33%) 
Personality disorder   1/43 (2.33%) 
Renal and urinary disorders   
Chromaturia   1/43 (2.33%) 
Dysuria   1/43 (2.33%) 
Pollakiuria   1/43 (2.33%) 
Respiratory, thoracic and mediastinal disorders   
Cough   3/43 (6.98%) 
Epistaxis   2/43 (4.65%) 
Nasal disorder   1/43 (2.33%) 
Pulmonary haemorrhage   1/43 (2.33%) 
Rhinorrhoea   1/43 (2.33%) 
Skin and subcutaneous tissue disorders   
Ingrowing nail   1/43 (2.33%) 
Pruritus   2/43 (4.65%) 
Rash   3/43 (6.98%) 
Skin discolouration   1/43 (2.33%) 
Skin lesion   1/43 (2.33%) 
Skin ulcer   1/43 (2.33%) 
Surgical and medical procedures   
Platelet transfusion   1/43 (2.33%) 
Tooth extraction   1/43 (2.33%) 
Vascular disorders   
Haematoma   1/43 (2.33%) 
Hypotension   1/43 (2.33%) 
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Cynthia E Dunbar
Organization: NHLBI
Phone: 301-496-5093
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT00922883     History of Changes
Other Study ID Numbers: 090154
09-H-0154 ( Other Identifier: The National Institutes of Health )
First Submitted: June 16, 2009
First Posted: June 17, 2009
Results First Submitted: June 18, 2018
Results First Posted: July 16, 2018
Last Update Posted: March 20, 2019