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.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Safety and tolerability (changes from baseline for safety parameters) of TXA127 in thrombocytopenic subjects with low or Intermediate-1 risk myelodysplastic syndrome (MDS).
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
Once or twice weekly during treatment and at follow-up visits. (up to 2 years)
Gere S diZerega, MD
Sponsor - US Biotest Inc,
United States: Food and Drug Administration
|MD Anderson Leukemia Department||Houston, Texas 77230-1402|