Cord Blood TransplantPhase 2NCT01554254
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
A Phase 2 study testing TXA127 in people with Cord Blood Transplant and Hematologic Malignancy, run by Tarix Pharmaceuticals at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 20 years
- Conditions the study is for
- Cord Blood Transplant, Hematologic Malignancy, Inherited Metabolic Disease
- 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
- Mar 2012to 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 6 months to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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 (defined as a subject who has started menses) must agree to the following:
- Use of an effective oral or IM contraceptive method during the course of the study and 2 months following the last administration of study drug.
- Female subjects capable of reproduction must have a negative pregnancy test result within 3 days prior to first study drug dose.
- Subjects must have adequate function of other organ systems as measured by:
- Creatinine <2.0 mg/dL and creatinine clearance >50 mL/min.
- Hepatic transaminases (ALT/AST) <4 x ULN, bilirubin <2.0 mg/dL.
- Adequate cardiac function by echocardiogram or MUGA scan (ejection fraction or shortening fraction >80% of normal value for age).
- Pulmonary function tests demonstrating FVC and FEV1 of >60% of predicted. DLCO should be used for subjects >10 years old. Crying vital capacity of >60% may be substituted for subjects unable to complete PFTs.
Who cannot join
- Subjects with an uncontrolled infection at the time of cytoreduction.
- Subjects who are pregnant or breast feeding.
- Subjects who are known to be seropositive for HIV or HTLV-1.
- Subjects who have had an autologous or allogeneic transplant <1 year from the anticipated administration of the first dose of study drug.
- Subjects who have received treatment with an investigational agent within 30 days of anticipated administration of the first dose of study drug.
- Subjects with 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.
- Subjects must not have 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Pediatric Bone and Cord Blood, Duke Univ. Med. Center
Durham, North Carolina, 27710
About this study
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.
- Being tested
- TXA127
- Keywords
- Cord blood transplant, hematological malignancy, inherited metabolic disease, Mucositis, Neutrophil engraftment, Platelet recovery, Immune reconstitution
- 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
300mcg/kg/day, subcutaneous injection for up to 28 days
| Study group | Role | What they receive |
|---|---|---|
| 300mcg/kg/day for 28 days | Experimental | Not described Receives: Drug: TXA127 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of TXA127 in subjects undergoing cord blood transplantation
Measured: Through Day 100 post transplant
Secondary outcomes (5)
Effect of TXA127 on incidence, severity and duration of aGVHD
Measured: Through Day 100 post transplant
Effect of TXA127 on incidence, severity and duration of mucositis
WHO oral toxicity score
Measured: Through Day 100 post transplant
Effect of TXA127 on neutrophil engraftment
Measured: Through Day 100 post transplant
Effect of TXA127 on platelet recovery
Measured: Through Day 100 post transplant
Effect of TXA127 on immune reconstitution
Measured: Through Day 100 post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2012 (estimated)
- Study startMar 2012
- Primary completionDec 2013
Why it stopped: Lack of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cord Blood TransplantStudies 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
- Treatment
- 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
- Kristin Page, MD, Principal investigator, Duke UniversityJoanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01554254
- Sponsor's protocol ID
- TXA127-2010-002
- Status last verified
- Aug 2016
- First submitted
- 12 Mar 2012
- Last updated
- 31 Aug 2016
- Results posted
- 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
- 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
- ClinicalTrials.gov ID
- NCT01554254
- Acronym / sponsor ID
- TXA127-2010-002
- Keywords
- Cord blood transplant, hematological malignancy, inherited metabolic disease, Mucositis, Neutrophil engraftment, Platelet recovery, Immune reconstitution
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Tarix PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2012
- Primary completion
- Dec 2013 (actual)
- First posted
- 14 Mar 2012 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 300mcg/kg/day for 28 days | Experimental | Drug: TXA127 |
Who may qualify
- Ages
- 6 months to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- 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.
- 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.
Key exclusion criteria (7 listed)
- Subjects with an uncontrolled infection at the time of cytoreduction.
- Subjects who are pregnant or breast feeding.
- Subjects who are known to be seropositive for HIV or HTLV-1.
- Subjects who have had an autologous or allogeneic transplant <1 year from the anticipated administration of the first dose of study drug.
- Subjects who have received treatment with an investigational agent within 30 days of anticipated administration of the first dose of study drug.
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 |
|---|---|---|
| TXA127300mcg/kg/day, subcutaneous injection for up to 28 days | Drug | 300mcg/kg/day for 28 days |
Primary outcome
Safety of TXA127 in subjects undergoing cord blood transplantation
Time frame: Through Day 100 post transplant
5 secondary outcomes
Effect of TXA127 on incidence, severity and duration of aGVHD
Time frame: Through Day 100 post transplant
Effect of TXA127 on incidence, severity and duration of mucositis
Time frame: Through Day 100 post transplant
Effect of TXA127 on neutrophil engraftment
Time frame: Through Day 100 post transplant
Effect of TXA127 on platelet recovery
Time frame: Through Day 100 post transplant
Effect of TXA127 on immune reconstitution
Time frame: Through Day 100 post transplant
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.
Kristin Page, MD principal investigator
Duke University
Joanne Kurtzberg, MD principal investigator
Duke University
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 Cord Blood Transplant and Hematologic Malignancy, 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 19 inclusion and 7 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?
Safety of TXA127 in subjects undergoing cord blood transplantation (time frame: Through Day 100 post transplant). 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.