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Phase 1 Open-Label Dose-Escalating Study Evaluating the Safety and Preliminary Efficacy of TXA127 in Patients With Low/Intermediate-1 Risk Myelodysplastic Syndrome and Thrombocytopenia

Phase 1
18 Years
65 Years
Not Enrolling
Myelodysplastic Syndrome (MDS)

Thank you

Trial Information

Phase 1 Open-Label Dose-Escalating Study Evaluating the Safety and Preliminary Efficacy of TXA127 in Patients With Low/Intermediate-1 Risk Myelodysplastic Syndrome and Thrombocytopenia

The hematopoietic properties demonstrated in the preclinical and clinical studies support
the investigation of TXA127 to stimulate stem cell and progenitor cell proliferation. This
is an exploratory study in a limited population of low or intermediate-1 MDS subjects who
have platelet counts of ≤50 x 109/L to evaluate the effects of TXA127 on platelet response
and on granulocytic and erythroid response.

Platelet response will be defined as complete and major as below:

- Complete platelet response: increase of platelet count to >100 x 109/L

- Major platelet response: increase of absolute platelet count by >30 x 109/L Other
responses will be according to modified IWG MDS criteria (2006). Daily subcutaneous
dosing of TXA will be carried out both in the clinic at scheduled visits and at home
between clinic visits for a period fo 28 days.

Inclusion Criteria:

- Diagnosis of MDS using the World Health Organization classification and Low or
Intermediate-1 risk MDS using the IPSS

- The mean of two platelet counts taken during the 2-week Screening Period must be ≤50
x 109/L, with no individual non-transfused count >60 x 109/L. Platelet counts taken
prior to Informed Consent may be used as one of the two counts taken within 2 weeks
prior to study Day 1, but both counts must be obtained within 4 weeks of commencement
of treatment.

- Subjects must be ≥18 years of age at the time of obtaining informed consent

- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at the time of

- Adequate Liver Function, as evidenced by a serum bilirubin ≤2 times the laboratory
upper limit of normal (ULN) (except for patients with a confirmed diagnosis of
Gilbert's Disease), ALT and/or AST ≤3 times the laboratory ULN.

- A serum creatinine concentration ≤2 mg/dL

Exclusion Criteria:

- Currently receiving any treatment for MDS other than transfusions (last transfusion
must be at least 4 weeks prior to treatment).

- If granulocyte and/or erythropoetic growth factors are currently being received,
there should be a 4-week washout prior to treatment, and they may not be used during
the study period.

- Concurrent active malignancy (other than controlled prostate cancer, in situ cervical
cancer, or basal cell cancer of the skin)

- Prior history of bone marrow transplantation

- Unstable angina, uncontrolled congestive heart failure [NYHA > class II],
uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or
recent (within 1 year) myocardial infarction, or a QTc interval value >450ms.

- Received Anti-Thymocyte Globuline (ATG) within 6 months of screening

- Received hypomethylating agents, immunomodulating agents, histone deacetylase
inhibitors, cyclosporine, or mycophenolate within 4 weeks of start of treatment

- Received IL-11 (oprelvekin) within 4 weeks before screening

- Have ever previously received rTPO, PEG-rHuMGDF, eltrombopag, or romiplostim

- Less than 4 weeks since receipt of any investigational agent (not FDA approved, for
any indication)

- History of arterial thrombosis (e.g., stroke or transient ischemic attack) in the
past year

- History of venous thrombosis that currently requires anti-coagulation therapy

- Female subjects who are pregnant or breastfeeding. Women of childbearing potential
are required to have a positive HCG serum or urine pregnancy test performed 7 days
prior to first study drug dose

- Women of childbearing potential who are unwilling to use an adequate form of
contraception during the course of the study.

- Subjects with current alcohol abuse, illicit drug use, or any other condition (e.g.,
psychiatric disorder) that, in the opinion of the Investigator, may interfere with
the patient's ability to comply with the study requirements or visit schedule.

- Subjects with a known sensitivity to any of the study medication components.

- Subjects known to have active HIV or known to be seropositive for HTLV-I.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability (changes from baseline for safety parameters) of TXA127 in thrombocytopenic subjects with low or Intermediate-1 risk myelodysplastic syndrome (MDS).

Outcome Description:

Evaluations performed during the study include vital signs and physical exam at all visits (twice/week for 4 weeks of treatment and at follow-up visits occurring 2 & 4 weeks following last treatment), blood chemistry, CBC, and platelet counts (once per week for 4 weeks, and at follow-up visits), concomitant medication and adverse event evaluations throughout the study period (8 weeks). Safety and tolerability will be assessed by incidence, severity, and changes from baseline of all relevant parameters including adverse events (AEs), laboratory values, and vital signs

Outcome Time Frame:

Once or twice weekly during treatment and at follow-up visits. (up to 2 years)

Safety Issue:


Principal Investigator

Gere S diZerega, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Sponsor - US Biotest Inc,


United States: Food and Drug Administration

Study ID:




Start Date:

April 2011

Completion Date:

April 2012

Related Keywords:

  • Myelodysplastic Syndrome (MDS)
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thrombocytopenia



MD Anderson Leukemia DepartmentHouston, Texas  77230-1402