NCT01362036

Phase I Study Evaluating TXA127 in Low/Intermediate-1 Risk Myelodysplastic Syndrome and Thrombocytopenia

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Tarix Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Phase 1, single-center, open-label, sequential cohort dose escalation study. This is a 3 + 3 design study involving at least 3 subjects in ascending dose cohorts, with subjects participating up to 10 weeks. The overall study objectives are to evaluate the safety and tolerability of TXA127 in thrombocytopenic subjects with low or intermediate-1 risk MDS. Evaluation of the platelet response and the erythroid and granulocytic responses to TXA127 will provide preliminary efficacy data.

Detailed description

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.

Conditions

• Myelodysplastic Syndrome (MDS)

Interventions

Type	Name	Description
DRUG	TXA127	Cohorts in this study will receive 300, 600, or 900 ug/kg daily by subcutaneous injection

Timeline

• Start date: 2011-04-01
• Primary completion: 2012-04-01
• Completion: 2012-04-01
• First posted: 2011-05-27
• Last updated: 2016-08-31

Locations

1 site across 1 country: United States