Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

• Register: ClinicalTrials.gov
• Last refreshed on: 10 January 2022
• Main ID: NCT02912208
• Date of registration: 09/09/2016
• Prospective Registration: No
• Primary sponsor: Associazione Qol-one
• Public title: Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes
• Scientific title: Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes (EQol-MDS)
• Date of first enrolment: June 11, 2011
• Target sample size: 174
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02912208
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Single (Participant).
• Phase: Phase 2

Countries of recruitment

France, Germany, Italy, Slovenia

Contacts

• Name: Esther Natalie Oliva
• Affiliation: QOL-ONE Associazione Culturale e di Ricerca

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and
stable disease.

2. Subjects must have a platelet count taken within the 4 weeks prior to randomization
that is <30 Gi/L.

3. Subjects must be ineligible or relapsed or refractory to receive other treatment
options (such as azacitidine or lenalidomide) and must be ineligible to receive
intensive chemotherapy or autologous/allogeneic stem cell transplantation.

4. Subjects must have platelet count and platelet transfusion data available over a
period of 8 weeks prior to randomization.

5. During the 2 months prior to randomization, subjects must have a baseline Bone Marrow
examination which includes cytomorphology and cytogenetics. Histopathology should be
performed.

6. Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte
colonystimulating factor (G-CSF) in subjects with severe neutropenia and recurrent
infections are allowed during the study as per accepted standards. Subjects who enter
the study on ESAs or G-CSF should continue at the same dose schedule until the optimal
dose of study medication has been established.

7. ECOG (Eastern Cooperative Oncology Group) Performance Status 0-3

8. Subject is able to understand and comply with protocol requirements and instructions.

9. Subject has signed and dated informed consent.

10. Adequate baseline organ function defined by the criteria below:

total bilirubin (except for Gilbert's Syndrome) = 1.5 x Upper Limit Normal Alanine
aminotransferase and Aspartate aminotransferase = 3 x Upper Limit Normal creatinine =
2 x Upper Limit Normal albumin must not be below the lower limit of normal by more
than 20%.

11. Subject is practicing an acceptable method of contraception. Female subjects (or
female partners of male subjects) must either be of non-childbearing potential
(hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal >1
year), or of childbearing potential and use of an highly effective method of
contraception from 2 weeks prior to administration of study medication, throughout the
study, and 28 days after completion or premature discontinuation from the study.

Exclusion Criteria:

1. MDS with intermediate-2 or high IPSS risk.

2. History of treatment for cancer other than MDS with systemic chemotherapy and/or
radiotherapy within the last 2 years.

3. History of treatment with romiplostim or other Thrombopoietin receptor agonists.

4. Pre-existing cardiovascular disease (including congestive heart failure, New York
Heart Association Grade III/IV), or arrhythmia known to increase the risk of
thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc
>450 msec (QTc >480 msec for subjects with Bundle Branch Block).

5. BM fibrosis that leads to an inability to aspirate marrow for assessment.

6. Peripheral monocytosis > 1000/uL prior to Day 1 of study medication.

7. Leukocytosis >=25,000/uL prior to Day 1 of study medication.

8. Female subjects who are nursing or pregnant (positive serum or urine Beta-human
chorionic gonadotropin [B-hCG] pregnancy test) at screening or pre-dose on Day 1.

9. Current alcohol or drug abuse.

10. Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is
longer) preceding the first dose of study medication.

11. Active and uncontrolled infections.

12. Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV).



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Myelodysplastic Syndromes

Thrombocytopenia

Intervention(s)

Other: Placebo

Drug: Eltrombopag/Revolade

Primary Outcome(s)

long-term safety and tolerability (number adverse events in the long term) [Time Frame: five years]

Duration of platelet response [Time Frame: five years]

Response rate [Time Frame: Six months]

Safety and Tolerability (number of adverse events) [Time Frame: Six month]

Secondary Outcome(s)

incidence and severity of bleeding [Time Frame: six months]

duration of transfusion independence [Time Frame: six months]

overall survival [Time Frame: 2 and 5 years]

leukemia-free survival (LFS) [Time Frame: 2 and 5 years]

time to response [Time Frame: six months]

number of monthly platelet transfusions [Time Frame: six months]

Quality of life (QoL) score [Time Frame: six months]

Secondary ID(s)

Eqol_MDS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors