Anemia in Myelodysplastic SyndromesPhase 1/2NCT04623996
A Study of TP-0184 to Treat Anemia in Adults With IPSS-R Low or Intermediate Risk MDS
A Phase 1/2 study testing TP-0184 in people with Anemia in Myelodysplastic Syndromes, run by Sumitomo Pharma America, Inc. at 3 sites in United States.
Trial summary
This study will evaluate preliminary safety and efficacy of TP-0184 to treat anemia when administered to adult patients with Revised International Prognostic Scoring System (IPSS-R) low or intermediate risk MDS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anemia in Myelodysplastic Syndromes
- What is being tested?
- TP-0184
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 3 sitesUnited States
- Study dates
- 28 Dec 2020to 27 Apr 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- A documented diagnosis of lower risk MDS (IPSS-R Low, Intermediate) according to WHO 2016 classification, de novo or secondary..
- Bone marrow biopsy and/or aspirate performed pre-dose to assess disease status and available for review prior to full screening review. If the bone marrow biopsy and/or aspirate is nonproductive or nondiagnostic, the procedure must be repeated. Bone marrow biopsy/aspirate performed ≤ 12 weeks prior to baseline will not need to be repeated if results and a minimum of 6 slides are available.
- Relapse, refractory/resistant, intolerant, or inadequate response to ESA treatment, as defined by the following:
- Relapse according to IWG 2006
- Refractory/resistant - documented non-response or response that is no longer maintained to prior ESA-containing regimen, either as single agent or combination (e.g., with G-CSF); ESA regimen must have been either:
- Recombinant human erythropoietin (rHu EPO) ≥ 500 IU/wk for at least 8 doses or equivalent; OR
Show all 30Show fewer
- Darbepoetin alpha ≥ 300 μg Q3W for at least 4 doses or equivalent;
- Intolerant- documented discontinuation of prior ESA containing regimen, either as single agent or combination (e.g., with G-CSF), at any time after introduction due to intolerance or an adverse event
- Inadequate response - in the absence of transfusions support, patients under ESA treatment for at least 12 weeks that do not show a rise in hemoglobin of greater than equal to 1 g/dl.
- Patients with 5q deletions are allowed only if they have failed or are intolerant to lenalidomide treatment.
- o Failure or intolerance to lenalidomide defined as clinical and cytogenetic responses to according to the international working group 2006 MDS: (1) absence of response, or (2) bone marrow progression during treatment with or without prior response, or (3) secondary failure (loss of prior hematological response without bone marrow progression), or (4) intolerance (treatment discontinuation due to adverse events, with or without prior response) based on investigator judgement (Prebet 2017). 7
- Patients previously treated for anemia with or without RBC transfusion support:
- Low transfusion burden (LTb), defined as requiring less than 4 red blood cell units in the 8 weeks before treatment (and baseline hemoglobin < 9.0 g/dL),
- High transfusion burden (HTb), defined as requiring 4 or more red blood cell units in the 8 weeks before treatment
- At least 12 weeks of transfusion history immediately preceding the first dose of TP-0184. This transfusion data must include hemoglobin measured prior to transfusion (pre transfusion Hgb).
- Written, signed consent for trial participation must be obtained from the patient appropriately in accordance with applicable ICH guidelines and local and regulatory requirements prior to the performance of any study specific procedure.
- Must be ≥18 years of age.
- Patients with an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) score ≤ 2.
- Patients with a life expectancy of ≥3 months (90 days) per the treating investigator.
- Patients with adequate major organ functions meeting the following criteria on the basis of laboratory data within 28 days (4 weeks) before screening (if multiple data are available, most recent data during the period):
- Serum creatinine: ≤1.8 x the upper limit of the normal (ULN) range
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) except in patients with Gilbert's syndrome. Patients with Gilbert's syndrome may enroll if direct bilirubin ≤2.0 x ULN or if the elevated total bilirubin can be attributed to active red blood cell precursor destruction within the bone marrow (i.e., ineffective erythropoiesis). Elevated indirect bilirubin due to post-transfusion hemolysis is allowed.
- Aspartate transaminase (AST) and alanine transaminase (ALT): ≤2.5 x ULN
- Left ventricular ejection fraction (LVEF) ≥ 45% by echocardiogram or multigated acquisition (MUGA) scan
- All previous therapy with ESAs, G-CSF and GM-CSF must be discontinued ≥ 14 days before Cycle 1 Day 1 dosing.
- Twenty-eight-day (4 weeks) washout period from prior treatment with cytotoxic chemotherapeutic agents, HMAs (hypomethylating agents), ImiDs (immunomodulatory imide drugs), luspatercept and / or investigational drugs before study dosing for the patient begins.
- Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test within 5 days prior to the first dose of TP-0184.
- Non-fertile or agree to use an adequate method of contraception while on study and for 7 months following the study and have a negative pregnancy test (if female of childbearing potential) and not currently nursing; males agree to use an adequate method of contraception while on study and for 4 months following the study.
- Patients must be able to comply with the requirements of the entire study and accessible for treatment and follow-up.
- Patient agrees not to participate in other interventional clinical studies during their participation in this trial, while on study treatment. Patients participating in surveys or observational studies are eligible to participate in this study.
Who cannot join
- IPSS-R high or very high risk MDS.
- Presence of concomitant severe cardiovascular disease; congestive heart failure (CHF), myocardial infarction, angina and/ or uncontrolled cardiac arrhythmia as determined by the investigator within 6 months (180 days) of study onset.
- Corrected QT interval (using Fridericia's correction formula) of > 465 msec in men and > 480 msec in women.
- History of stroke, deep venous thrombosis (DVT), pulmonary or arterial embolism within 6 months (180 days) prior to enrollment.
- Presence of clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
- Iron deficiency to be determined by a bone marrow aspirate stain for iron, calculated transferrin saturation (iron/total iron binding capacity) ≤ 20%, or serum ferritin ≤ 15 µg/L.
Show all 29Show fewer
- Iron-chelating agents, except for subjects on a stable or decreasing dose for at least 8 weeks prior to enrollment, are excluded
- Prior allogeneic or autologous stem cell transplant due to myeloid disease
- Known history of diagnosis of AML.
- Use of corticosteroid, except for subjects on a stable or decreasing (no greater than a 10 mg dose of prednisone or equivalent) for ≥ 2 weeks prior to enrollment for medical conditions other than MDS
- Patients are excluded if there is evidence of autoimmune hemolytic anemia manifested as a corrected reticulocyte count (reticulocyte index) of > 2% with either a positive Coombs' test or over 50% indirect bilirubin.
- Patients with a recent diagnosis of malignancy are excluded except for those with in situ malignancies treated with curative intent (eg, basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix). Patients with more advanced malignancies are allowed to enroll, provided they were treated with curative intent and have no evidence of active disease ≥ 2 years prior to Cycle 1 Day 1.
- Patients requiring systemic antibiotics or antifungals are not eligible until they have completed the prescribed course of antibiotics or antifungals and are clinically stable. Topical antibiotics or antifungals are permitted.
- Known HIV, active Hepatitis B, and/or active Hepatitis C infection.
- Patients with clinically active uncontrolled, bleeding in the past month.
- Thrombocytopenia (platelet count < 50,000/µL [50 x 109/L]).
- Neutropenia (absolute neutrophil count [ANC] <500 /µL [0.5 x 109/L])
- Women who are pregnant or breastfeeding.
- Male patients with partners of childbearing potential who are unwilling to use condoms in combination with a second effective method of contraception during the trial and for 7 months after the last administration of study treatment.
- Inability to undergo MRI imaging.
- Parenchymal iron overload by screening MRI.
- Patients who are unwilling or unable to comply with procedures required in this protocol.
- Have undergone recent surgery with potential to cause the impairment of gastrointestinal tract absorption or that could cause short bowel syndrome with diarrhea due to malabsorption.
- Have known hemochromatosis at baseline or a family history of hemochromatosis.
- Presence of any psychological, familial, sociological or geographical condition that, in the opinion of the investigator, could potentially hinder compliance with the study protocol and follow-up schedule.
- Patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
- Live vaccines within 14 days prior to first study drug administration. COVID-19 vaccines (non-live) approved by regional health authorities are allowed.
- Medications that are known strong to moderate CYP3A4 inducers must be discontinued at least 21 days prior to first dose of study drug. Medications that are known strong to moderate CYP3A4 inhibitors must be stopped 21 days (or 5 half-lives, whichever is shorter) prior to the first dose of study drug.
- Patients who have received medications with known or possible risk of prolonging the QT interval or inducing Torsades de Pointes within the previous 7 days.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
US Oncology - Austin Texas Oncology Midtown
Austin, Texas, 78222
BRCR Global
Plantation, Florida, 33322
Montefiore Medical Center
The Bronx, New York, 10467
About this study
Official title: A Phase 1/2, Open-Label Clinical Study To Evaluate Safety And Efficacy Of TP-0184 To Treat Anemia When Administered To Adult Patients With IPSS-R Low Or Intermediate Risk Myelodysplastic Syndromes
This study will evaluate preliminary safety and efficacy of TP-0184 to treat anemia when administered to adult patients with Revised International Prognostic Scoring System (IPSS-R) low or intermediate risk MDS. The recommended Phase 2 dose (RP2D) will be determined by the maximum tolerated dose (MTD) or maximum administered dose (MAD) in the Phase 1 portion of the study.
- Conditions
- Anemia in Myelodysplastic Syndromes
- Being tested
- TP-0184
- Keywords
- Ph 1/2, Hematologic disease, MDS, Anemia, IPSS-R low, IPSS-R intermediate
- Medical subject headings
- Hematologic Diseases, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TP-0184Drug
Oral dose
| Study group | Role | What they receive |
|---|---|---|
| Single Arm TP-0184 | Experimental | TP-0184 is administered orally once a day Receives: Drug: TP-0184 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Tolerability of TP-0184 as Evaluated by Incidence of Dose Limiting Toxicities (DLTs) as Observed in Cycle 1
DLTs are defined as follows: Any Gr 3 or greater nonhematologic toxicity; New onset cardiac failure or worsening symptomatic cardiac failure; Echocardiogram (ECHO) or multigated acquisition (MUGA) reduction of ejection fraction (EF) > 10%; Any Gr 4 neutropenia; Any Gr 3 thrombocytopenia associated with clinically significant bleeding, or Gr 4 thrombocytopenia in the absence of myelodysplasia-related marrow failure or transformation to acute leukemia; All other hematologic toxicity Grade 3 or higher other than defined above for ANC or platelets
Measured: Cycle 1 (28 days)
Phase 1: Number of Participants With Adverse Events and Serious Adverse Events
Assessment of safety of TP-0184 administered in participants by reporting of adverse events and serious adverse events
Measured: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, an average of 4 months.
Phase 2: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 8 Weeks
Response rate based on composite response criteria: Hemoglobin increase greater than or equal to 1.5 g/dL, maintained for a consecutive period of 8 weeks with no transfusions; OR reduction in units of greater than or equal to 4 RBC transfusions / 8 weeks (consecutive) compared with the pretreatment transfusion number in previous 8 weeks; OR patients who are RBC transfusion-free over any consecutive 8-week (56-day) period
Measured: 56 days
Secondary outcomes (15)
Phase 1: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 8 Weeks
Response rate based on composite response criteria: Hemoglobin increase greater than or equal to 1.5 g/dL, maintained for a consecutive period of 8 weeks with no transfusions; OR reduction in units of greater than or equal to 4 RBC transfusions / 8 weeks (consecutive) compared with the pretreatment transfusion number in previous 8 weeks; OR patients who are RBC transfusion-free over any consecutive 8-week (56-day) period
Measured: 56 days
Phase 1/2: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 12 Weeks
Response rate based on composite response criteria: Hemoglobin increase greater than or equal to 1.5 g/dL, maintained for a consecutive period of 12 weeks with no transfusions; OR reduction in units of greater than or equal to 4 RBC transfusions / 12 weeks (consecutive) compared with the pretreatment transfusion number in previous 12 weeks; OR patients who are RBC transfusion-free over any consecutive 12-week (84-day) period
Measured: 84 days
Phase 1/2: Determine the Time to RBC Transfusion-free Period
Time from first dose of TP-0184 to the first onset of a transfusion-free period for consecutive 8 weeks
Measured: 56 days
Phase 1/2: Determine the Median Duration of Hemoglobin Response
Duration of hemoglobin increase greater or equal to 1.5 g/dL maintained for a consecutive period of > 8 weeks with no transfusions.
Measured: 56 days
Phase 1/2: Determine the Median Duration of Reduction in RBC Transfusions
Duration of reduction in units of greater or equal to 4 RBC transfusions / 8 weeks (consecutive)
Measured: 56 days
Phase 1/2: Determine the Median Duration of RBC-transfusion-free Period Greater or Equal to 8 Weeks
Duration of RBC transfusion-free period
Measured: 56 days
Phase 1/2: Determine the Cardiac Safety of TP-0184 by Assessing the Presence of Cardiac Symptoms
Assessment of cardiac safety of TP-0184 administered in participants by reporting of cardiac symptoms including congestive heart failure (CHF). Assessment of CHF will be based based on NYHA criteria, 12-Lead ECG abnormalities, quantification of cardiac iron by MRI, ECHO or MUGA scans, and peripheral blood cardiac markers
Measured: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, an average of 4 months.
Phase 1/2: Determine the Proportion of Patients Progressing to AML
Proportion of patients progressing to AML
Measured: 4 months
Phase 1/2: Determine Overall Survival
The effect of TP-0184 on the overall survival
Measured: From the first dose of TP-0184 until the time of death due to any cause, up to 4 months off treatment.
Phase 1: Determination of the Maximum Observed Concentration (Cmax) and Area Under the Plasma Concentration vs. Time Curve (AUClast)
To determine the maximum concentration of TP-0184 and the area under the plasma concentration vs. time curve of TP-0184
Measured: Phase 1: Blood samples drawn on days 1, 2, 4, 8, 15, and 22 of the first study cycle; days 1, 8, 15, and 16 of the 2nd cycle. Phase 2: Blood samples drawn on Cycle 1, day 22; cycle 2, days 1, 8 and 15; cycle 3, day 1.
Phase 2: Determine the Changes in Neutrophil Counts
Proportion of patients achieving hematologic improvement in neutrophil count (HI-N) over any consecutive 8-week (56-day) period and / or decrease in neutrophil count
Measured: 56 days
Phase 2: Determine the Changes in Platelet Counts
Proportion of patients achieving hematologic improvement in platelets (HI-P) over any consecutive 8-week (56-day) period and / or decrease in platelet count
Measured: 56 days
Phase 2: Number of Participants With Adverse Events (AEs) & Serious Adverse Events (SAEs)
Assessment of safety and tolerability of TP-0184 administered in participants by reporting of AEs and SAEs
Measured: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, up to 6 months.
Phase 2: Determine Steady-state Trough PK
Characterize TP-0184 plasma trough concentration data at various timepoints.
Measured: Cycle 1 Day 1 of Week 4 and Cycle 2 Day 1 of each Week 5, 6, 7, and 9
Phase 2: Determine the Level of Fatigue Based Off of the Brief Fatigue Inventory (BFI)
The BFI measures the severity of fatigue based on the worst fatigue experienced during the past 24-hours
Measured: From the first dose of TP-0184 up to 4 months or study closure
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2020
- Study start28 Dec 2020
- Primary completion27 Apr 2021
- Study completion27 Apr 2021
Why it stopped: Due to limitations imposed by the COVID pandemic and the competitive landscape for myelodysplastic syndrome, this study was unable to enroll a sufficient patient population.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Anemia in Myelodysplastic SyndromesStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sumitomo Pharma America, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04623996
- Sponsor's protocol ID
- BBI-TP-0184-102
- Status last verified
- Nov 2023
- First submitted
- 20 Oct 2020
- Last updated
- 9 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label Clinical Study To Evaluate Safety And Efficacy Of TP-0184 To Treat Anemia When Administered To Adult Patients With IPSS-R Low Or Intermediate Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT04623996
- Acronym / sponsor ID
- BBI-TP-0184-102
- Conditions
- Anemia in Myelodysplastic Syndromes
- Keywords
- Ph 1/2, Hematologic disease, MDS, Anemia, IPSS-R low, IPSS-R intermediate
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to limitations imposed by the COVID pandemic and the competitive landscape for myelodysplastic syndrome, this study was unable to enroll a sufficient patient population.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Sumitomo Pharma America, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Dec 2020 (actual)
- Primary completion
- 27 Apr 2021 (actual)
- Study completion
- 27 Apr 2021 (actual)
- First posted
- 10 Nov 2020 (actual)
- Results
- Posted 30 Mar 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate preliminary safety and efficacy of TP-0184 to treat anemia when administered to adult patients with Revised International Prognostic Scoring System (IPSS-R) low or intermediate risk MDS. The recommended Phase 2 dose (RP2D) will be determined by the maximum tolerated dose (MTD) or maximum administered dose (MAD) in the Phase 1 portion of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm TP-0184 | Experimental | Drug: TP-0184TP-0184 is administered orally once a day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (30 listed)
- A documented diagnosis of lower risk MDS (IPSS-R Low, Intermediate) according to WHO 2016 classification, de novo or secondary..
- Bone marrow biopsy and/or aspirate performed pre-dose to assess disease status and available for review prior to full screening review. If the bone marrow biopsy and/or aspirate is nonproductive or nondiagnostic, the procedure must be repeated. Bone marrow biopsy/aspirate performed ≤ 12 weeks prior to baseline will not need to be repeated if results and a minimum of 6 slides are available.
- Relapse, refractory/resistant, intolerant, or inadequate response to ESA treatment, as defined by the following:
- Inadequate response - in the absence of transfusions support, patients under ESA treatment for at least 12 weeks that do not show a rise in hemoglobin of greater than equal to 1 g/dl.
- Patients with 5q deletions are allowed only if they have failed or are intolerant to lenalidomide treatment.
Key exclusion criteria (29 listed)
- IPSS-R high or very high risk MDS.
- Presence of concomitant severe cardiovascular disease; congestive heart failure (CHF), myocardial infarction, angina and/ or uncontrolled cardiac arrhythmia as determined by the investigator within 6 months (180 days) of study onset.
- Corrected QT interval (using Fridericia's correction formula) of > 465 msec in men and > 480 msec in women.
- History of stroke, deep venous thrombosis (DVT), pulmonary or arterial embolism within 6 months (180 days) prior to enrollment.
- Presence of clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
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 |
|---|---|---|
| TP-0184Oral dose | Drug | Single Arm TP-0184 |
Primary outcome
Phase 1: Tolerability of TP-0184 as Evaluated by Incidence of Dose Limiting Toxicities (DLTs) as Observed in Cycle 1
Time frame: Cycle 1 (28 days)
DLTs are defined as follows: Any Gr 3 or greater nonhematologic toxicity; New onset cardiac failure or worsening symptomatic cardiac failure; Echocardiogram (ECHO) or multigated acquisition (MUGA) reduction of ejection fraction (EF) > 10%; Any Gr 4 neutropenia; Any Gr 3 thrombocytopenia associated with clinically significant bleeding, or Gr 4 thrombocytopenia in the absence of myelodysplasia-related marrow failure or transformation to acute leukemia; All other hematologic toxicity Grade 3 or higher other than defined above for ANC or platelets
Phase 1: Number of Participants With Adverse Events and Serious Adverse Events
Time frame: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, an average of 4 months.
Assessment of safety of TP-0184 administered in participants by reporting of adverse events and serious adverse events
Phase 2: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 8 Weeks
Time frame: 56 days
Response rate based on composite response criteria: Hemoglobin increase greater than or equal to 1.5 g/dL, maintained for a consecutive period of 8 weeks with no transfusions; OR reduction in units of greater than or equal to 4 RBC transfusions / 8 weeks (consecutive) compared with the pretreatment transfusion number in previous 8 weeks; OR patients who are RBC transfusion-free over any consecutive 8-week (56-day) period
15 secondary outcomes
Phase 1: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 8 Weeks
Time frame: 56 days
Phase 1/2: The Effect of TP-0184 on the Treatment and the Hematologic Improvement of Anemia in Terms of Hemoglobin Increase, Reduction in RBC Transfusions and Transfusion-free Equal or Greater Than 12 Weeks
Time frame: 84 days
Phase 1/2: Determine the Time to RBC Transfusion-free Period
Time frame: 56 days
Phase 1/2: Determine the Median Duration of Hemoglobin Response
Time frame: 56 days
Phase 1/2: Determine the Median Duration of Reduction in RBC Transfusions
Time frame: 56 days
Phase 1/2: Determine the Median Duration of RBC-transfusion-free Period Greater or Equal to 8 Weeks
Time frame: 56 days
Phase 1/2: Determine the Cardiac Safety of TP-0184 by Assessing the Presence of Cardiac Symptoms
Time frame: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, an average of 4 months.
Phase 1/2: Determine the Proportion of Patients Progressing to AML
Time frame: 4 months
Phase 1/2: Determine Overall Survival
Time frame: From the first dose of TP-0184 until the time of death due to any cause, up to 4 months off treatment.
Phase 1: Determination of the Maximum Observed Concentration (Cmax) and Area Under the Plasma Concentration vs. Time Curve (AUClast)
Time frame: Phase 1: Blood samples drawn on days 1, 2, 4, 8, 15, and 22 of the first study cycle; days 1, 8, 15, and 16 of the 2nd cycle. Phase 2: Blood samples drawn on Cycle 1, day 22; cycle 2, days 1, 8 and 15; cycle 3, day 1.
Phase 2: Determine the Changes in Neutrophil Counts
Time frame: 56 days
Phase 2: Determine the Changes in Platelet Counts
Time frame: 56 days
Phase 2: Number of Participants With Adverse Events (AEs) & Serious Adverse Events (SAEs)
Time frame: The time from the date of first treatment, while the patient is taking TP-0184, and for 30 days after stopping therapy, up to 6 months.
Phase 2: Determine Steady-state Trough PK
Time frame: Cycle 1 Day 1 of Week 4 and Cycle 2 Day 1 of each Week 5, 6, 7, and 9
Phase 2: Determine the Level of Fatigue Based Off of the Brief Fatigue Inventory (BFI)
Time frame: From the first dose of TP-0184 up to 4 months or study closure
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Anemia in Myelodysplastic Syndromes
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 3
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Myelodysplastic Syndromes
RecruitingPhase 3
A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Lower Risk Myelodysplastic Syndromes (LR-MDS) Who Need Regular Blood Transfusions
Myelodysplastic Syndrome, Anemia
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TP-0184 in people with Anemia in Myelodysplastic Syndromes, run by Sumitomo Pharma America, Inc. at 3 sites 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 30 inclusion and 29 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 9 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 combined 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.
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?
Yes. Results are posted in the registry (first posted 30 Mar 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Phase 1: Tolerability of TP-0184 as Evaluated by Incidence of Dose Limiting Toxicities (DLTs) as Observed in Cycle 1 (time frame: Cycle 1 (28 days)). The study lists 3 primary outcomes in all. 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 9 Nov 2023.