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A Three-part Study of Eltrombopag in Thrombocytopenic Subjects With Myelodysplastic Syndromes or Acute Myeloid Leukemia (ASPIRE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis
ClinicalTrials.gov Identifier:
NCT01440374
First received: September 15, 2011
Last updated: May 6, 2016
Last verified: May 2016
Results First Received: March 10, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Supportive Care
Condition: Thrombocytopaenia
Interventions: Drug: eltrombopag
Drug: placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The results presented are based on the Interim Analysis (Part 3 results are not included).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) and Grade 4 thrombocytopenia due to bone marrow insufficiency and had at least one of the following: platelet count <10 Giga cells per liter, platelet transfusion or symptomatic hemorrhagic event during the 4 weeks prior to enrollment, were enrolled in the study.

Reporting Groups
  Description
Part 1: Eltrombopag The eltrombopag starting dose the participants received was 100 milligrams (mg) daily (50 mg for participants of East Asian heritage). The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. After all participants finished the 8 week treatment period, platelet response and safety were analyzed before initiating Part 2 of the study. Supportive standard of care was allowed as needed. The duration of Part 1 was 8 weeks.
Part 2: Placebo Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study.
Part 2: Eltrombopag The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The duration of treatment with the initial dose level prior to first dose escalation was determined based on the platelet response and toxicity observed in Part 1. The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. Supportive standard of care was allowed as needed throughout the study.

Participant Flow for 2 periods

Period 1:   Part 1: Open-label Phase
    Part 1: Eltrombopag   Part 2: Placebo   Part 2: Eltrombopag
STARTED   17   0   0 
COMPLETED   14   0   0 
NOT COMPLETED   3   0   0 
Withdrawal by Subject                3                0                0 

Period 2:   Part 2; Double-blind Phase
    Part 1: Eltrombopag   Part 2: Placebo   Part 2: Eltrombopag
STARTED   0   47   98 
Ongoing   0   4   11 
COMPLETED   0   35   72 
NOT COMPLETED   0   12   26 
Lost to Follow-up                0                0                1 
Physician Decision                0                1                1 
Withdrawal by Subject                0                7                13 
Ongoing                0                4                11 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Part 1: Eltrombopag The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. After all participants finished the 8 week treatment period, platelet response and safety were analyzed before initiating Part 2 of the study. Supportive standard of care was allowed as needed. The duration of Part 1 was 8 weeks.
Part 2: Placebo Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study.
Part 2: Eltrombopag The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The duration of treatment with the initial dose level prior to first dose escalation was determined based on the platelet response and toxicity observed in Part 1. The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. Supportive standard of care was allowed as needed throughout the study.
Total Total of all reporting groups

Baseline Measures
   Part 1: Eltrombopag   Part 2: Placebo   Part 2: Eltrombopag   Total 
Overall Participants Analyzed 
[Units: Participants]
 17   47   98   162 
Age 
[Units: Years]
Mean (Standard Deviation)
 71.5  (10.78)   70.6  (10.72)   72.3  (8.94)   71.7  (9.66) 
Gender 
[Units: Participants]
       
Female   7   16   32   55 
Male   10   31   66   107 
Race/Ethnicity, Customized 
[Units: Participants]
       
African American/African Heritage   0   0   1   1 
Asian - East Asian Heritage   3   5   9   17 
Asian - South East Asian Heritage   0   2   2   4 
White - Arabic/North African Heritage   0   1   5   6 
White - White/Caucasian/European Heritage   14   39   81   134 


  Outcome Measures
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1.  Primary:   Number of Participants With Platelet Response up to Week 8 During Part 1   [ Time Frame: From Baseline up to Week 8 during Part 1 ]

2.  Primary:   Percentage of Participants With Clinically Relevant Thrombocytopenic Events (CRTE) From Week 5 up to Week 12 During Part 2   [ Time Frame: From Week 5 up to Week 12 during Part 2 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Clinical Disclosure Office
Organization: Novartis Pharmaceuticals
phone: 862-778-8300



Responsible Party: Novartis
ClinicalTrials.gov Identifier: NCT01440374     History of Changes
Other Study ID Numbers: 114968
Study First Received: September 15, 2011
Results First Received: March 10, 2016
Last Updated: May 6, 2016
Health Authority: Spain: Agencia Espanola de Medicamentos y Productos Sanitarios
Netherlands: Centrale Commissie Mensgesbonden Onderzoek (Central Committee on Research Involving Human Subjects) Den Haag (The Hague), The Netherlands
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Korea: Food and Drug Administration
Mexico: Secretaría de Salud
Taiwan: Food and Drug Administration, Department of Health, Executive Yuan
Greece: National Organization of Medicines
Hungary: National Institute of Pharmacy
Thailand: Food and Drug Administration
United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Thailand: Ministry of Public Health
Italy: Ministry of Health
Hong Kong: Department of Health
Israel: State of Israel Ministry of Health, Health Technology and Infrastructure Administration, Medical Devices Department
Poland: URZ.D REJESTRACJI PRODUKTÓW LECZNICZYCH, WYROBÓW MEDYCZNYCH I PRODUKTÓW BIOBÓJCZYCH,CEBK
Germany: Bundesinstitut für Arzneimittel und Medizinprodukte
Belgium: Federaal Agentschap voor Geneesmiddelen en Gezondheidsproducten
Russian Federation: Ministry of Health and social development of Russian Federation, Federal
Brazil: Agência Nacional de Vigilância Sanitária (ANVISA)
Peru: General Directorate of Pharmaceuticals, Devices, and Drugs
India: Drugs Controller General of India (DCGI)
Czech Republic: State Institute for Drug Control