Myelodysplastic Syndrome (MDS)Phase 1NCT01362036
Phase I Study Evaluating TXA127 in Low/Intermediate-1 Risk Myelodysplastic Syndrome and Thrombocytopenia
A Phase 1 study testing TXA127 in people with Myelodysplastic Syndrome (MDS), run by Tarix Pharmaceuticals at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myelodysplastic Syndrome (MDS)
- 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
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2011to Apr 2012
- Phase
- Phase 1
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of MDS using the World Health Organization classification and Low or Intermediate-1 risk MDS using the IPSS
- The mean of two platelet counts taken during the 2-week Screening Period must be ≤50 x 109/L, with no individual non-transfused count >60 x 109/L. Platelet counts taken prior to Informed Consent may be used as one of the two counts taken within 2 weeks prior to study Day 1, but both counts must be obtained within 4 weeks of commencement of treatment.
- Subjects must be ≥18 years of age at the time of obtaining informed consent
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at the time of screening
- Adequate Liver Function, as evidenced by a serum bilirubin ≤2 times the laboratory upper limit of normal (ULN) (except for patients with a confirmed diagnosis of Gilbert's Disease), ALT and/or AST ≤3 times the laboratory ULN.
- A serum creatinine concentration ≤2 mg/dL
Who cannot join
- Currently receiving any treatment for MDS other than transfusions (last transfusion must be at least 4 weeks prior to treatment).
- If granulocyte and/or erythropoetic growth factors are currently being received, there should be a 4-week washout prior to treatment, and they may not be used during the study period.
- Concurrent active malignancy (other than controlled prostate cancer, in situ cervical cancer, or basal cell cancer of the skin)
- Prior history of bone marrow transplantation
- Unstable angina, uncontrolled congestive heart failure [NYHA > class II], uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction, or a QTc interval value >450ms.
- Received Anti-Thymocyte Globuline (ATG) within 6 months of screening
Show all 17Show fewer
- Received hypomethylating agents, immunomodulating agents, histone deacetylase inhibitors, cyclosporine, or mycophenolate within 4 weeks of start of treatment
- Received IL-11 (oprelvekin) within 4 weeks before screening
- Have ever previously received rTPO, PEG-rHuMGDF, eltrombopag, or romiplostim
- Less than 4 weeks since receipt of any investigational agent (not FDA approved, for any indication)
- History of arterial thrombosis (e.g., stroke or transient ischemic attack) in the past year
- History of venous thrombosis that currently requires anti-coagulation therapy
- Female subjects who are pregnant or breastfeeding. Women of childbearing potential are required to have a positive HCG serum or urine pregnancy test performed 7 days prior to first study drug dose
- Women of childbearing potential who are unwilling to use an adequate form of contraception during the course of the study.
- Subjects with current alcohol abuse, illicit drug use, or any other condition (e.g., psychiatric disorder) that, in the opinion of the Investigator, may interfere with the patient's ability to comply with the study requirements or visit schedule.
- Subjects with a known sensitivity to any of the study medication components.
- Subjects known to have active HIV or known to be seropositive for HTLV-I.
Trial locations and contacts
1 site in 1 country.
United States1 site
MD Anderson Leukemia Department
Houston, Texas, 77230-1402
About this study
Official title: 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
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.
Read the 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)
- Being tested
- TXA127
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TXA127Drug
Also called Angiotensin 1-7
Cohorts in this study will receive 300, 600, or 900 ug/kg daily by subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| TXA127 sc injectable | Experimental | All cohorts will recieve TXA127; Cohorts receive either 300, 600, or 900 ug/kg daily Receives: Drug: TXA127 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability (changes from baseline for safety parameters) of TXA127 in thrombocytopenic subjects with low or Intermediate-1 risk myelodysplastic syndrome (MDS).
Evaluations performed during the study include vital signs and physical exam at all visits (twice/week for 4 weeks of treatment and at follow-up visits occurring 2 \& 4 weeks following last treatment), blood chemistry, CBC, and platelet counts (once per week for 4 weeks, and at follow-up visits), concomitant medication and adverse event evaluations throughout the study period (8 weeks).
Safety and tolerability will be assessed by incidence, severity, and changes from baseline of all relevant parameters including adverse events (AEs), laboratory values, and vital signs
Measured: Once or twice weekly during treatment and at follow-up visits. (up to 2 years)
Secondary outcomes (2)
Evaluate the platelet response (change in platelet count from baseline) of thrombocytopenic subjects with low or intermediate-1 risk MDS receiving TXA127.
Platelet counts and response will be evaluated twice per week at clinic visits during the treatment period (4 weeks), and again at the follow-up visits (week 6 and week 8).
Measured: Twice Weekly during treatment and at follow-up visits (up to 2 years)
Assess the erythroid and granulocytic response (change from baseline) to TXA127 in patients with low or intermediate-1 risk MDS.
Erythroid and granulocytic responses will be evaluated once weekly during the treatment period (first visit of each treatment week), and again at the follow-up clinic visits.
Measured: Weekly during treatment and at follow-up visits (up to 2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2011 (estimated)
- Study startApr 2011
- Primary completionApr 2012
- Study completionApr 2012
Why it stopped: Enrollment feasibility issues, new study in design
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic Syndrome (MDS)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tarix Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gere S diZerega, MD, Study chair, Sponsor - US Biotest Inc,
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01362036
- Sponsor's protocol ID
- TXA127-2010-003
- Status last verified
- Aug 2016
- First submitted
- 23 Apr 2011
- 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 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
- ClinicalTrials.gov ID
- NCT01362036
- Acronym / sponsor ID
- TXA127-2010-003
- Conditions
- Myelodysplastic Syndrome (MDS)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment feasibility issues, new study in design
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Tarix PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Apr 2011
- Primary completion
- Apr 2012 (actual)
- Study completion
- Apr 2012 (actual)
- First posted
- 27 May 2011 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TXA127 sc injectable | Experimental | Drug: TXA127All cohorts will recieve TXA127; Cohorts receive either 300, 600, or 900 ug/kg daily |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of MDS using the World Health Organization classification and Low or Intermediate-1 risk MDS using the IPSS
- The mean of two platelet counts taken during the 2-week Screening Period must be ≤50 x 109/L, with no individual non-transfused count >60 x 109/L. Platelet counts taken prior to Informed Consent may be used as one of the two counts taken within 2 weeks prior to study Day 1, but both counts must be obtained within 4 weeks of commencement of treatment.
- Subjects must be ≥18 years of age at the time of obtaining informed consent
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at the time of screening
- Adequate Liver Function, as evidenced by a serum bilirubin ≤2 times the laboratory upper limit of normal (ULN) (except for patients with a confirmed diagnosis of Gilbert's Disease), ALT and/or AST ≤3 times the laboratory ULN.
Key exclusion criteria (17 listed)
- Currently receiving any treatment for MDS other than transfusions (last transfusion must be at least 4 weeks prior to treatment).
- If granulocyte and/or erythropoetic growth factors are currently being received, there should be a 4-week washout prior to treatment, and they may not be used during the study period.
- Concurrent active malignancy (other than controlled prostate cancer, in situ cervical cancer, or basal cell cancer of the skin)
- Prior history of bone marrow transplantation
- Unstable angina, uncontrolled congestive heart failure [NYHA > class II], uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction, or a QTc interval value >450ms.
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 |
|---|---|---|
| TXA127Also: Angiotensin 1-7Cohorts in this study will receive 300, 600, or 900 ug/kg daily by subcutaneous injection | Drug | TXA127 sc injectable |
Primary outcome
Safety and tolerability (changes from baseline for safety parameters) of TXA127 in thrombocytopenic subjects with low or Intermediate-1 risk myelodysplastic syndrome (MDS).
Time frame: Once or twice weekly during treatment and at follow-up visits. (up to 2 years)
Evaluations performed during the study include vital signs and physical exam at all visits (twice/week for 4 weeks of treatment and at follow-up visits occurring 2 \& 4 weeks following last treatment), blood chemistry, CBC, and platelet counts (once per week for 4 weeks, and at follow-up visits), concomitant medication and adverse event evaluations throughout the study period (8 weeks). Safety and tolerability will be assessed by incidence, severity, and changes from baseline of all relevant parameters including adverse events (AEs), laboratory values, and vital signs
2 secondary outcomes
Evaluate the platelet response (change in platelet count from baseline) of thrombocytopenic subjects with low or intermediate-1 risk MDS receiving TXA127.
Time frame: Twice Weekly during treatment and at follow-up visits (up to 2 years)
Assess the erythroid and granulocytic response (change from baseline) to TXA127 in patients with low or intermediate-1 risk MDS.
Time frame: Weekly during treatment and at follow-up visits (up to 2 years)
Results and safety
The study was stopped early (reason given: Enrollment feasibility issues, new study in design). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2016.
Related trials for Myelodysplastic Syndrome (MDS)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
RecruitingPhase 1
Study of REM-422 in Patients With AML or Higher Risk MDS
Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes
RecruitingObservational study
Natural History of Trisomy 8-Associated Autoinflammatory Disease (TRIAD) and Related Disorders
Trisomy 8 Mosaicism, Trisomy 8 Associated Autoinflammatory Disease
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
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.
Gere S diZerega, MD study chair
Sponsor - US Biotest Inc,
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TXA127 in people with Myelodysplastic Syndrome (MDS), 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 6 inclusion and 17 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: stopped early, 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 stopped early (reason given: Enrollment feasibility issues, new study in design). Results are not available in the source record.
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 and tolerability (changes from baseline for safety parameters) of TXA127 in thrombocytopenic subjects with low or Intermediate-1 risk myelodysplastic syndrome (MDS). (time frame: Once or twice weekly during treatment and at follow-up visits. (up to 2 years)). 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.