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A TRHEE-PART STUDY OF ELTROMBOPAG IN THROMBOCYTOPENIC SUBJECTS WITH MYELODYSPLASTIC SYNDROMES OR ACUTE MYELOID LEUKEMIA (PART 1: OPEN-LABEL, PART 2: RANDOMIZED, DOUBLE-BLIND, PART 3: EXTENSION).

Not Applicable
Registration Number
PER-018-12
Lead Sponsor
GlaxoSmithKline,
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Sex
Not specified
Target Recruitment
0
Inclusion Criteria

1. ADULTS SUBJECTS (18 YEARS OF AGE OR OLDER) WITH MDS OR AML (BONE MARROW BLASTS ≤ 50 %) WITH THROMBOCYTOPENIA DUE TO BONE MARROW INSUFFICIENCY FROM THE DISEASE OR PRIOR TREATMENT. SUBJECTS WITH TRANSIENT THROMBOCYTOPENIA DUE TO ACTIVE TREATMENT WITH DISEASE MODIFYING AGENTS OR CHEMOTHERAPY (EXCEPT FOR HYDROXYUREA) ARE EXCLUDED.
2. SUBJECTS MUST HAVE GRADE 4 THROMBOCYTOPENIA (PLATELET COUNTS < 25 Gi/L DUE TO PLATELET TRANSFUSION). IN ADDITION , SUBJECTS MUST HAVE HAD AT LEAST ONE OF THE FOLLOWING DURING THE 4 WEEK SCREENING PERIOD: PLATELET TRANSFUSION, OR SYMPTOMATIC BLEEDING OR PLATELET COUNT < 10 Gi/L. SUBJECTS WHOSE THROMBOCYTOPENIA BELOW 10 gi/L IS DUE TO CAUSES OTHER THAN BONE MARROW INSUFFICIENCY (E.G., FEVER, INFECTION, AUTOIMMUNE DISEASE) ARE NOT ELIGIBLE.
3. SUBJECTS MUST HAVE PLATELET COUNT, BLEEDING AND PLATELET TRANSFUSION DATA AVAILABLE OVER A PERIOD OF A LEAST 4 WEEKS PRIOR TO RANDOMIZATION.
4. PRIOR SYSTEMIC TREATMENT FOR MALIGNANCY, WITH THE EXCEPTION OF HYDROXYUREA (SEE SECTION 6.1.2), MUST HAVE BEEN DISCONTINUED PRIOR TO ENTRY INTO THE STUDY:
. AL LESAST 4 WEEKS BEFORE DAY 1 FOR THE FOLLOWING: CHEMOTHERAPY, DEMETHYLATING AGENTS

Exclusion Criteria

1. SUBJECTS WITH MMDS AND AN IPSS OF LOW OR INTERMEDIATE-1 RISK.
2. SUBJECTS WITH A DIAGNOSIS OF ACUTE PROMYELOCYTIC OR MEGAKARYOCYTIC LEUKEMIA OR AML SECONDARY TO A MYELOPROLIFERATIVE NEOPLASM.
3. HISTORY OF TREATMENT WITH ROMIPLOSTIM OR OTHER TPO-R AGONISTS.
4. SUBJECTS WITH A QTc > 480 msec (QTc > 510 msec FOR SUBJECTS WITH BUNDLE BRANCH BLOCK) AT BASELINE.
5. SUBJECTS WITH A PALPABLE SPLEE MUST HAVE A SPLENIC ULTRASOUND TO CONFIRM SPLEEN LENGT H IS ≤ 16 CM. SUBJECTS WITH PALPABLE SPLEEN 16 CM ARE NOT ELIGIBLE.
6. LEUKOCYTOSIS ≥25,000/Ul ON DAY 1 OF TREATMENT WITH STUDY MEDICATION.
7. SUBJECTS WITH KNOWN THROMBOPHILIC RISK FACTORS. EXCEPTION: SUBJECTS FOR WHOM THE POTENTIAL BENEFITS OF PARTICIPATING IN THE STUDY OUTWEIGH THA POTENCIAL RISKS OF THROMBOEMBOLIC EVENTS, AS DETERMINED BY THE INVESTIGATOR.
8. FEMALE SUBJECTS WHO ARE NURSING OR PREGNANT (POSITIVE SERUM OR URINE β-HUMAN CHORIONIC GONADOTROPIN [β-hCG] PREGNANCY TEST) AT SCREENING OR PRE-DOSE ON DAY 1.
9. CURRENT ALCOHOL OR DRUG ABUSE.
10. TREATMENT WITH AN INVESTIGATIONAL DRUG WITHIN 30 DAYS OR 5 HALF-LIVES (WHICHEVER IS LONGER) PRECEDING THE FIRST DOSE OF STUDY MEDICATION.
11. ACTIVE AND UNCONTROLLED INFECTIONS (E.G. SEPSIS).
12. SUBJECTS INFECTED WITH HEPATITIS B, C OR HUMAN IMMUNODEFICIENCY VIRUS (HIV).

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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