WithdrawnPhase 2

Evaluation of the Safety and Efficacy of TXA127 (Angiotensin 1-7) to Enhance Engraftment in Pediatric Patients Undergoing Single or Double Umbilical Cord Blood Transplantation

Stopped: Lack of enrollment

United States0Started 2012-03

Plain-language summary

Engraftment failure is a major obstacle to the success of cord blood transplantation in children with malignancies and inherited metabolic disorders, despite the fact that they receive relatively high doses of nucleated cells from UCB. TXA127 is pharmaceutically formulated Angiotensin 1-7 \[A(1-7)\], a non-hypertensive derivative of Angiotensin-II (which contains the 8th amino acid conferring receptor binding to blood pressure receptors). TXA127 has multilineage effects on hematopoietic progenitors in vitro and in vivo. Preclinical data show that TXA127 is a novel stimulator of early multilineage hematopoietic progenitors, increases engraftment of committed hematopoietic progenitors, and induces more rapid production of platelets and neutrophils in the peripheral circulation, especially in limited cell number transplants. Treatment with TXA127 following UCBT is expected to increase the numbers of hematopoietic progenitors and accelerate engraftment.

Who can participate

Age range6 Months – 20 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Subject, parent, or legal guardian provided written informed consent. * Subjects must be \>6 months and \<21 years of age. * Subjects must have one or two available 4, 5, or 6/6 antigen matching unrelated UCB unit(s) that will deliver a cell dose between 3.0-5.0 x 107cells/kg. * These units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the subject. * If two CB units will be used, the units must be HLA-matched at 3 of 6 HLA- A, B, and DRB1 loci with each other (using same resolution of molecular typing as indicated above). * For a single unit transplant, a minimum of 3 x 107cells/kg will be required. * For a double unit transplant, HLA-matched units must be available such that together both units deliver a combined pre-cryopreserved nucleated cell dose of at least 4.0 x 107 cells/kg with 1 unit of at least 2.5 x 107 cells/kg and the other at least 1.5 x 107 cells/kg. * Subjects must have histologically confirmed diagnosis of a hematologic malignancy or a laboratory confirmed inherited metabolic disease. * Subjects who have had a prior autologous or allogeneic transplant are allowed to participate provided it has been \>1 year since the transplant was completed. * Subjects must not have active CNS disease at the time of study enrollment. * Subjects must have a life expectancy of \>4 months. * Female subjects capable of reproduction (…

What they're measuring

Safety of TXA127 in subjects undergoing cord blood transplantation

Timeframe: Through Day 100 post transplant

Trial details

NCT IDNCT01554254

SponsorTarix Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2013-12