Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Digestive System Diseases

Mouth and Tooth Diseases

Rare Diseases

Hematologic Neoplasms

Graft vs Host Disease

Mucositis

Hematologic Diseases

Homologous Wasting Disease

Acute Graft Versus Host Disease

Antihypertensive Agents

Vasodilator Agents

All Drugs and Chemicals

Vasoconstrictor Agents

Angiotensin I (1-7)

Angiotensin II

Angiotensinogen

Single-arm safety/efficacy trial of TXA127 (Angiotensin 1-7) in subjects undergoing double cord blood transplantation for the treatment of hematologic malignancies. Treatment dose is 300 mcg/kg/day TXA127.

TXA127

Mary J Laughlin, MD

The purpose of this study is to evaluate the efficacy of TXA127 to reduce the incidence (Grade II-IV) of acute Graft-vs.-Host Disease (aGVHD) in adult subjects undergoing double umbilical cord blood transplantation (UCBT). The study will also evaluate the effects of TXA127 on incidence, severity and duration of mucositis; neutrophil engraftment and platelet recovery; platelet transfusion requirements; immune reconstitution; and duration of corticosteroid use. TXA127 has shown to be well tolerated by patients and appears to induce rapid production of neutrophils and platelets in the bloodstream, as well as increase the immune system components. TXA127 has also been shown reduce the severity of chemotherapy-induced mucositis.

Cord blood (CB) as a hematopoietic stem cell source has multiple advantages. Cord blood is normally discarded at birth and can easily be collected and stored. Availability of numerous CB banks has resulted in genetically diverse CB units including those from non-Caucasians. Once a suitable CB unit is located, confirmatory typing can be quickly performed and a donor unit can be shipped to the transplant center. Furthermore, because a CB graft results in a lower incidence of graft-versus-host disease (GVHD), one or two antigen-mismatches may be acceptable for transplantation. Despite these advantages, CB has a significant drawback: the number of hematopoietic stem cells obtained from a unit of CB is significantly lower than from a bone marrow (BM) or peripheral blood stem cell (PBSC) harvest. The number of stem cells can be increased by transplanting two cord blood units, however the incidence of GVHD increases in patients receiving two CB units compared to patients who receive one unit. Another issue in this population is mucositis, as a result of myeloablative conditioning given prior to the transplant, which can be debilitating to patients. TXA127 is pharmaceutically-formulated angiotensin 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. The hematopoietic properties demonstrated in preclinical and clinical studies support the investigation of TXA127 to reduce the incidence of acute GVHD (aGVHD) and mucositis in this patient population.

Efficacy Study of a TXA127 to Reduce Acute Graft-vs-Host Disease in Subjects Undergoing Double Umbilical Cord Blood Transplantation

Phase II Evaluation of the Efficacy of TXA127 (Angiotensin 1-7) to Reduce Acute Graft-vs-Host Disease in Adults Undergoing Double Umbilical Cord Blood Transplantation

Incidence of Grade II-IV acute graft-vs-host disease (aGVHD) will be assessed using clinical staging and grading criteria as defined in Przepiorka et al. (1995). Duration and severity of aGVHD will also be evaluated.

Incidence of mucositis is defined by the occurrence of least one adverse event with MedDRA preferred term that includes "mucositis" or "stomatitis". The severity grade will be determined by NCI-CTCAE.

Time to initial neutrophil engraftment is defined as the number of days from infusion of UCB units to the first of 3 consecutive days of an ANC ≥0.5 × 10\^9/L. Time to initial platelet recovery is defined as the number of days from infusion of UCB units to the first of 3 consecutive platelet count measurements tested on different days with a count ≥20 × 10\^9/L with no platelet transfusion in the prior 7 days.

Platelet transfusion requirements are based on cumulative units of platelets transfused and cumulative days of platelet transfusions.

Immune reconstitution will be assessed via the measurement of peripheral blood concentrations of CD3+, CD4+, CD8+, CD19+, and CD56+ cells (performed at Study Days 62 and 100).

Duration of corticosteroid use for GVHD will be summarized by frequency (i.e., number of days).

Tarix Pharmaceuticals

Symptoms and General Pathology

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Recurrence

Neuroblastoma

Sarcoma

Wilms Tumor

Disease Attributes

Neoplasms by Histologic Type

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Soft Tissue Sarcoma

Wilms' Tumor

Neuroepithelioma

Antineoplastic Agents

Fibrinolytic Agents

Anticoagulants

Carboplatin

Etoposide

Heparin

Ifosfamide

Etoposide phosphate

Calcium heparin

Isophosphamide mustard

Mesna

Patients will receive standard doses of ICE Chemotherapy:

* Ifosfamide 1800 mg/m2 mixed with Mesna 360 mg/m2 IV over 2 hours on days 1, 2, 3, 4, and 5
* Carboplatin 400 mg/m2 IV over 1 hour on days 1 and 2
* Etoposide 100 mg/m2 IV over 1 hour on days 1, 2, 3, 4, and 5

ODSH will be administered as a 4 mg/kg bolus 30 minutes after the first ifosfamide dose followed immediately by a continuous intravenous ODSH infusion of 0.25 mg/kg/hour for five consecutive days, on days 1-5, for a total of 120 hours of continuous ODSH infusion.

ODSH

Mitchell Cairo, MD

This study will be with pediatric patients who have refractory/recurrent solid tumors. They will receive standard chemotherapy (ICE) and we are investigating if the addition of a new drug, ODSH, will help to increase the time of their platelet recovery after ICE chemotherapy.The purpose of this study is to evaluate the safety and tolerability of ODSH in pediatric patients receiving "ICE" chemotherapy.

ODSH + ICE Chemotherapy in Pediatric Solid Tumors

A Pilot Study to Evaluate the Safety and Preliminary Evidence of an Effect of ODSH (2-O, 3-O Desulfated Heparin) in Accelerating Platelet Recovery in Pediatric Patients With a Recurrent Solid Tumor Receiving "ICE" Chemotherapy

All patients will be followed for 60 days after the last dose of ODSH in a treatment cycle in which ODSH is administered. All serious adverse events related to ODSH will be monitored.

Platelet counts will be monitored to determine when the platelet count remained \> 100,000 after nadir for two consecutive days in the absence of a prior platelet transfusion within the previous 48 hours in pediatric patients receiving "ICE" chemotherapy.

New York Medical College

Nutritional and Metabolic Diseases

Metabolic Diseases

300mcg/kg/day, subcutaneous injection for up to 28 days

Kristin Page, MD

Joanne Kurtzberg, MD

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.

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

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy Study of a TXA127 to Reduce Acute Graft-vs-Host Disease in Subjects Undergoing Double Umbilical Cord Blood Transplantation	NCT01882374	Hematologic Mal...	TXA127	18 Years -	Tarix Pharmaceuticals
ODSH + ICE Chemotherapy in Pediatric Solid Tumors	NCT02164097	Recurrent Solid... Refractory Soli... Sarcoma Wilms' Tumor Neuroblastoma	ODSH	1 Year - 21 Years	New York Medical College
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	NCT01554254	Cord Blood Tran... Hematologic Mal... Inherited Metab...	TXA127	6 Months - 20 Years	Tarix Pharmaceuticals
Efficacy Study of a TXA127 to Reduce Acute Graft-vs-Host Disease in Subjects Undergoing Double Umbilical Cord Blood Transplantation	NCT01882374	Hematologic Mal...	TXA127	18 Years -	Tarix Pharmaceuticals

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