Hematologic MalignanciesPhase 2NCT01882374
Efficacy Study of a TXA127 to Reduce Acute Graft-vs-Host Disease in Subjects Undergoing Double Umbilical Cord Blood Transplantation
A Phase 2 study testing TXA127 in people with Hematologic Malignancies, run by Tarix Pharmaceuticals at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- TXA127
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to Dec 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provided written informed consent.
- ≥18 years of age.
- Meet institutional standard criteria for double UCB transplantation
- Myeloablative conditioning regimen
- Histologically confirmed diagnosis of a hematologic malignancy.
- Life expectancy of ≥4 months.
- Female subjects capable of reproduction (defined as a subject who has started menses) must agree to the following: 1) Use of an effective oral or IM contraceptive method during the course of the study and 2 months following the last administration of Investigational Product; and 2) must have a negative pregnancy test result within 7 days prior to first Investigational Product dose.
Who cannot join
- Uncontrolled infection at the time of transplant.
- Pregnant or breastfeeding.
- Known to be seropositive for HIV or HTLV-1.
- Active CNS disease at the time of study enrollment.
- Treatment with an investigational agent within 30 days of anticipated administration of the first dose of Investigational Product.
- Current alcohol use, illicit drug use or any other condition (e.g., psychiatric disorder) that, in the opinion of the Investigator, may interfere with the subject's ability to comply with the study requirements or visit schedule.
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- Any co-morbid condition which, in the view of the Principal Investigators, renders the subject at too high a risk from treatment complications and regimen-related morbidity/mortality.
- Prophylactic treatment with palifermin for mucositis.
- Subjects with a known sensitivity to any of the Investigational Product components.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Virginia Cancer Center
Charlottesville, Virginia, 22903
About this study
Official title: 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
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.
Read the detailed description
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.
- Conditions
- Hematologic Malignancies
- Being tested
- TXA127
- Keywords
- Double Umbilical Cord Blood Transplantation, Acute Graft versus Host Disease, Neutrophil Engraftment, Platelet Recovery, Immune Reconstitution, Mucositis
- Medical subject headings
- Hematologic Neoplasms, Mucositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TXA127Drug
Injection, 300mcg/kg/day for 28 days
| Study group | Role | What they receive |
|---|---|---|
| TXA127, blood draws, physical exams | Experimental | 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. Receives: Drug: TXA127 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Grade II-IV acute graft-vs-host disease (aGVHD)
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.
Measured: 100 days post-transplantation
Secondary outcomes (5)
Incidence, severity, and duration of mucositis
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.
Measured: 100 days post-transplantation
Neutrophil engraftment and platelet recovery
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.
Measured: 100 days post-transplantation
Platelet transfusion requirements
Platelet transfusion requirements are based on cumulative units of platelets transfused and cumulative days of platelet transfusions.
Measured: 100 days post-transplantation
Immune reconstitution
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).
Measured: 100 days post-transplantation
Duration of corticosteroid use
Duration of corticosteroid use for GVHD will be summarized by frequency (i.e., number of days).
Measured: 100 days post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2013 (estimated)
- Study startDec 2013
- Primary completionDec 2013
- Study completionDec 2013
Why it stopped: Change in drug product development strategy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignanciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tarix Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mary J Laughlin, MD, Principal investigator, University of Virginia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01882374
- Sponsor's protocol ID
- TXA127-2012-01
- Status last verified
- Aug 2016
- First submitted
- 17 Jun 2013
- Last updated
- 31 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01882374
- Acronym / sponsor ID
- TXA127-2012-01
- Conditions
- Hematologic Malignancies
- Keywords
- Double Umbilical Cord Blood Transplantation, Acute Graft versus Host Disease, Neutrophil Engraftment, Platelet Recovery, Immune Reconstitution, Mucositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Change in drug product development strategy
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Tarix PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2013
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 20 Jun 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TXA127, blood draws, physical exams | Experimental | Drug: TXA127Single-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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Provided written informed consent.
- ≥18 years of age.
- Meet institutional standard criteria for double UCB transplantation
- Myeloablative conditioning regimen
- Histologically confirmed diagnosis of a hematologic malignancy.
Key exclusion criteria (9 listed)
- Uncontrolled infection at the time of transplant.
- Pregnant or breastfeeding.
- Known to be seropositive for HIV or HTLV-1.
- Active CNS disease at the time of study enrollment.
- Treatment with an investigational agent within 30 days of anticipated administration of the first dose of Investigational Product.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TXA127Injection, 300mcg/kg/day for 28 days | Drug | TXA127, blood draws, physical exams |
Primary outcome
Incidence of Grade II-IV acute graft-vs-host disease (aGVHD)
Time frame: 100 days post-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.
5 secondary outcomes
Incidence, severity, and duration of mucositis
Time frame: 100 days post-transplantation
Neutrophil engraftment and platelet recovery
Time frame: 100 days post-transplantation
Platelet transfusion requirements
Time frame: 100 days post-transplantation
Immune reconstitution
Time frame: 100 days post-transplantation
Duration of corticosteroid use
Time frame: 100 days post-transplantation
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mary J Laughlin, MD principal investigator
University of Virginia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing TXA127 in people with Hematologic Malignancies, run by Tarix Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 31 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Grade II-IV acute graft-vs-host disease (aGVHD) (time frame: 100 days post-transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2016.