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Myelodysplastic SyndromesPhase 2NCT03900715

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

A Phase 2 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Celgene at 19 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. Read more about this study

Who is it for?
Ages 20 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Luspatercept
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
19 sitesJapan
Study dates
20 May 2019to 1 Mar 2023
Phase
Phase 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 20 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subjects must satisfy the following criteria to be enrolled in the study:
  • Subject is ≥ 20 years of age the time of signing the informed consent form (ICF)
  • Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
  • Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
  • Subject has a documented diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease, and:
  • < 5% blasts in bone marrow
Show all 17Show fewer
  • Subject has symptomatic anemia with mean Hgb concentration < 10.0 g/dL from 2 measurements (one performed within 1 day prior to W1D1 and the other performed 7 to 35 days prior to W1D1) that does not require RBC transfusion. If more than one measurement exists in the period of 7 to 35 days prior to W1D1, the most recent value will be used.
  • Subject must be TI, as documented by the following criteria:
  • No RBC transfusion administered within 16 weeks prior to W1D1 (except transfusions due to blood loss or infection that occurred between 16 and 8 weeks prior to W1D1)
  • Subject has Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
  • Females of childbearing potential (FCBP), defined as a sexually mature woman who:
  • 1) has achieved menarche at some point, 2) not undergone a hysterectomy or bilateral oophorectomy, or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy or amenorrhea due to other medical reasons does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months), must:
  • Have two negative pregnancy tests as verified by the investigator prior to starting study therapy (unless the screening pregnancy test was done within 72 hours of W1D1). She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence1 from heterosexual contact.
  • Either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, highly effective contraception without interruption, 5 weeks prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks after discontinuation of study therapy.
  • If breastfeeding, agree to stop breastfeeding prior to the participation in the study and not to resume breastfeeding after treatment discontinuation.
  • 9. Male subjects must:
  • Practice true abstinence2 (which must be reviewed prior to each IP administration or on a monthly basis [eg, in the event of dose delays]) or agree to use a condom (latex or non-latex, but not made out of natural [animal] membrane) during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 12 weeks following investigational product discontinuation, even if he has undergone a successful vasectomy.

Who cannot join

  • The presence of any of the following will exclude a subject from enrollment:
  • Subject with the any of the following prior treatments for underlying disease:
  • Disease modifying agents (eg, immune-modulatory drug [IMiDs such as lenalidomide])
  • Except if the subject received ≤ 1 week of treatment with a disease modifying agent ≥ 8 weeks from W1D1, at the investigator's discretion.
  • Hypomethylating agents
  • Subjects may be enrolled at the investigator's discretion contingent that the subject received no more than 2 injections of HMA. The last dose must be ≥ 8 weeks from the date of W1D1.
Show all 50Show fewer
  • Luspatercept (ACE-536) or sotatercept (ACE-011)
  • Allogeneic and/or autologous hematopoietic cell transplant
  • Subject with myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) according to WHO 2016 classification (ie, chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), BCR-ABL12, juvenile myelomonocytic leukemia (JMML), MDS/MPN unclassifiable.
  • Subject with secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
  • Subject with known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or hypothyroidism, or any type of known clinically significant bleeding or sequestration. Subject with drug induced anemia (eg, mycophenolate).
  • Iron deficiency to be determined by serum ferritin < 100 μg/L and additional testing if clinically indicated (eg, calculated transferrin saturation [iron/total iron binding capacity ≤ 20%] or bone marrow aspirate stain for iron).
  • Subject with known history of diagnosis of AML
  • Subject receiving any of the following treatment within 8 weeks prior to W1D1:
  • Anticancer cytotoxic chemotherapeutic agent or treatment
  • ESAs
  • Granulocyte colony-stimulating factor (G-CSF), granulocyte-macrophage colonystimulating factor (GM-CSF), unless given for treatment of febrile neutropenia
  • Immunosuppressive therapy for MDS
  • Systemic corticosteroid, except for subjects on a stable or decreasing dose for ≥ 1 week prior to W1D1 for medical conditions other than MDS
  • Other RBC hematopoietic growth factors (eg, Interleukin-3)
  • Androgens, unless to treat hypogonadism
  • Hydroxyurea, anagrelide
  • Oral retinoids
  • Arsenic trioxide
  • Interferon and interleukins
  • Investigational drug or device, or approved therapy for investigational use (if 5 times the half-life of the previous investigational drug exceeds 8 weeks, then the time of exclusion should be extended up to 5 times the half-life of the investigational drug)
  • Subject with uncontrolled hypertension, defined as repeated elevations of systolic blood pressure (SBP) of ≥ 150 mmHg and/or diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment, or with a history of hypertensive crisis or hypertensive encephalopathy.
  • Subject with any of the following laboratory abnormalities:
  • Absolute neutrophil count (ANC) < 500/μL (0.5 x 10^9/L)
  • Platelet count < 30,000/μL (30 x 10^9/L) (Exclude subjects that may be at risk of bleeding regardless of platelet counts. This includes [but is not limited to] subjects currently using aspirin or heparin, immediately after surgery, or easily causes bleeding such as nasal bleeding or subcutaneous bleeding or previous episode of major bleeding where the cause was not effectively treated.)
  • Estimated glomerular filtration rate (eGFR) < 40 mL/min/1.73 m^2 (via the 4-variable modification of diet in renal disease [MDRD] formula, Appendix G)
  • Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN)
  • Total bilirubin ≥ 2.0 x ULN. - Higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (ie, ineffective erythropoiesis) or in the presence of known history of Gilbert Syndrome.
  • Subject with prior history of malignancies, other than MDS, unless the subject has been free of the disease for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed if considered as curatively treated:
  • Basal or squamous cell carcinoma of the skin
  • Carcinoma in situ of the cervix
  • Carcinoma in situ of the breast
  • Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)
  • Subject with major surgery within 8 weeks prior to W1D1. Subjects must have completely recovered from any previous surgery prior to W1D1
  • Subject with history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (DVT; including proximal and distal), pulmonary or arterial embolism, arterial thrombosis or other venous thrombosis within 6 months prior to W1D1 Note: prior superficial thrombophlebitis is not an exclusion criterion.
  • Subject with the following cardiac conditions within 6 months prior to W1D1:
  • myocardial infarction, uncontrolled angina, acute decompensated cardiac failure or New York Heart Association (NYHA) Class III-IV heart failure, or uncontrolled cardiac arrhythmia as determined by the investigator. Subjects with a known ejection fraction ˂35%, confirmed by a local echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan performed within 6 months prior to W1D1.
  • Subject with uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
  • Subject with known human immunodeficiency virus (HIV), known evidence of active infectious hepatitis B, and/or known evidence of active Hepatitis C. Local laboratory testing confirming HIV, hepatitis B, and hepatitis C status should not have been performed beyond 4 weeks prior to the date of ICF signature.
  • Subject with history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in luspatercept (see current IB).
  • Pregnant or breastfeeding females.
  • Subject has any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations (eg, imprisoned or institutionalized) that would prevent the subject from participating in the study.
  • Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
  • Subject has any condition or receives concomitant medication that confounds the ability to interpret data from the study.
  • Subject has history of active SARS-CoV-2 infection within 4 weeks prior to screening, unless the subject has adequately recovered from COVID-19 symptoms and related complications as per investigator's discretion and following a discussion with the Medical Monitor. Use of a live COVID-19 vaccine is prohibited within 4 weeks prior to W1D1.

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

Japan19 sites

  • Local Institution - 345

    Chiba, 260-0852

  • Local Institution - 334

    Fukuoka, 810-8563

  • Local Institution - 347

    Himeji, 670-8540

  • Local Institution - 337

    Hitachi, Ibaraki, 317-0077

  • Local Institution - 346

    Kamakura, 247-8533

  • Local Institution - 331

    Kamogawa, 296-8602

  • Local Institution - 348

    Kitakyushu, 806-8501

  • Local Institution - 349

    Kofu, 400-0027

  • Local Institution - 338

    Matsuyama, Ehime, 790-8524

  • Local Institution - 344

    Nagasaki, Nagasaki, 852-8511

  • Local Institution - 343

    Nagoya, 460-0001

  • Local Institution - 341

    Ōgaki, 503-8502

  • Local Institution - 335

    Okayama, 700-8557

  • Local Institution - 342

    Osaka, 545-8586

  • Local Institution - 333

    Sagamihara, 252-0375

  • Local Institution - 336

    Sayama, Osaka, 5898511

  • Local Institution - 339

    Sendai, 980-8574

  • Local Institution - 332

    Shibuya-ku, 150-8935

  • Local Institution - 330

    Shinagawa-ku, Tokyo, 141-8625

Other trials in Japan

About this study

Official title: A Phase 2, Multicenter, Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements.

This is a Phase 2, multicenter, single-arm study to evaluate the efficacy, safety and Pharmacokinetics (PK) of luspatercept (ACE-536) for the treatment of anemia due to International prognostic scoring system-Revised (IPSS-R) very low, low or intermediate risk Myelodysplastic syndromes (MDS)in Japanese subjects who are not requiring Red blood cell (RBC) transfusion.

The study is divided into the Screening Period, a Treatment Period and a Post-Treatment Follow up Period.

Read the detailed description

Anemia is considered to be one of the most prevalent cytopenias in patients who have myelodysplastic syndromes, an umbrella term used to describe disorders relating to the ineffective production of red blood cells, white blood cells, and/or platelets. Ranging in severity from mild (asymptomatic) to severe, anemia can result in patients requiring regular red blood cell (RBC) transfusions, which can lead to further complications from iron overload. The goal of this study is to assess the efficacy, safety and PK of luspatercept in anemic patients who are categorized as International Prognostic Scoring System-Revised (IPSS-R) very low, low, or intermediate risk Myelodysplastic syndrome (MDS), and not requiring RBC transfusion. Subjects deemed eligible for the study will be enrolled and treated with luspatercept. Best supportive care (BSC) may be used in combination with study treatment when clinically indicated per investigator. Best supportive care includes, but is not limited to, treatment with transfusions, antibiotic, antiviral and/or antifungal therapy, and nutritional support as needed. Best supportive care for this study excludes the use of ESAs. Patients should receive treatment up to a minimum of 24 weeks after which an MDS Disease assessment visit is scheduled to assess the response to treatment. Patients who are determined to be experiencing clinical benefit may continue treatment. Continued clinical benefit will be re-assessed every 24 weeks. Once patients are discontinued from study treatment, they will enter a post treatment follow-up period.

Being tested
Luspatercept
Keywords
Myelodysplastic Syndromes, MDS, ACE-536, Anemia
Medical subject headings
Myelodysplastic Syndromes, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LuspaterceptDrug

    Luspatercept

Study groups in NCT03900715
Study groupRoleWhat they receive
Luspatercept AdministrationExperimentalLuspatercept will be administered as a subcutaneous injection every 3 week (21 days; Q3W), at an initial dose level of 1.0 mg/kg. Doses may be titrated up starting at dosing visit Week 7 Day 1 (W7D1)
Receives: Drug: Luspatercept

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants Who Achieved Hematologic Improvement in Erythroid Response (HI-E) Per International Working Group (IWG) Through Week 24

    Hematologic improvement is defined as the percent of participants meeting HI-E criteria of >= 1.5 g/dL increase in hemoglobin (Hgb) sustained over any consecutive 56-day period in the absence of red blood cell (RBC) transfusions from Week 1 Day 1 through Week 24. Participants who discontinued from the Treatment Period without achieving HI-E are counted as non-responders.

    Measured: Week 1 Day 1 through Week 24

Secondary outcomes (15)

  1. Percentage of Participants Who Achieved Modified Hematologic Improvement in Erythroid Response (mHI-E) Per International Working Group (IWG)

    Modified hematologic improvement is defined as the percent of participants meeting mHI-E criteria of >= 1.5 g/dL mean increase in hemoglobin (Hgb) compared to baseline sustained over any consecutive 56-day period in the absence of red blood cell (RBC) transfusions from Week 1 Day 1 through Week 24 and Week 48 of the Treatment Period. Participants who discontinued from the Treatment Period without achieving mHI-E are counted as non-responders. Baseline Hgb is defined as the mean of the two central laboratory values (one performed within 1 day prior to W1D1 and the other performed 7 to 35 days prior to W1D1).

    Measured: Week 1 Day 1 through Week 24 and Week 48

  2. Percentage of Participants Who Achieved Hematologic Improvement in Erythroid Response (HI-E) Per International Working Group (IWG) Through Week 48

    Hematologic improvement is defined as the percent of participants meeting HI-E criteria of >= 1.5 g/dL increase in hemoglobin (Hgb) sustained over any consecutive 56-day period in the absence of red blood cell (RBC) transfusions from Week 1 Day 1 through Week 48 of the Treatment Period. Participants who discontinued from the Treatment Period without achieving HI-E are counted as non-responders.

    Measured: Week 1 Day 1 through Week 48

  3. Time to Hematologic Improvement in Erythroid Response (HI-E)

    Defined as time from Week 1 Day 1 to first onset of achieving ≥ 1.5 g/dL increase in hemoglobin (Hgb) over any consecutive 56-day period in the absence of red blood cell (RBC) transfusions.

    Measured: Week 1 Day 1 through Week 24 and Week 48

  4. Time to Modified Hematologic Improvement in Erythroid Response (mHI-E)

    Defined as the time from Week 1 Day 1 to first onset of achieving ≥ 1.5 g/dL mean increase in hemoglobin (Hgb) compared to baseline over any consecutive 56-day period in the absence of red blood cell (RBC) transfusions. Baseline Hgb is defined as the mean of the two central laboratory values (one performed within 1 day prior to W1D1 and the other performed 7 to 35 days prior to W1D1.

    Measured: Week 1 Day 1 through Week 24 and Week 48

  5. Duration of Hematologic Improvement in Erythroid Response (HI-E)

    Defined as the maximum duration of achieving ≥ 1.5 g/dL increase in hemoglobin (Hgb) for participants who achieve Hgb increase ≥ 56 days in the absence of red blood cell (RBC) transfusions. Participants who maintain HI-E through the end of the Treatment Period or time of analysis will be censored at the date of treatment discontinuation/time of analysis or death, whichever occurs first.

    Measured: Week 1 Day 1 through end of treatment period (up to an average of 74 weeks and maximum of 178 weeks)

  6. Duration of Modified Hematologic Improvement in Erythroid Response (mHI-E)

    Defined as the maximum duration of achieving ≥ 1.5 g/dL increase in hemoglobin for participants who achieve mean hemoglobin (Hgb) increase compared to baseline ≥ 56 days in the absence of red blood cell (RBC) transfusions. Participants who maintain mHI-E through the end of the Treatment Period or time of analysis will be censored at the date of treatment discontinuation/time of analysis or death, whichever occurs first. Baseline Hgb is defined as the mean of the two central laboratory values (one performed within 1 day prior to W1D1 and the other performed 7 to 35 days prior to W1D1.

    Measured: Week 1 Day 1 through end of treatment period (up to an average of 74 weeks and maximum of 178 weeks)

  7. Red Blood Cell Transfusion Independence (RBC-TI)

    Defined as the percentage of participants not being given any red blood cell (RBC) transfusion during the treatment period.

    Measured: Week 1 Day 1 through Week 24, Week 48, and Week 72

  8. Progression to Acute Myeloid Leukemia (AML)

    Number of participants progressing to acute myeloid leukemia (AML). AML progression is defined per WHO classification of ≥ 20% blasts in peripheral blood or bone marrow. Subjects with diagnosis of AML will be considered to have had an event. Subjects who have not progressed to AML at the time of analysis will be censored at the last assessment date which does not indicate progression to AML.

    Measured: From Week 1 Day 1 up to approximately 44 months

  9. Time to Acute Myeloid Leukemia (AML) Progression

    Defined as the time between W1D1 and first diagnosis of AML as per WHO classification of ≥ 20% blasts in peripheral blood or bone marrow. Participants with diagnosis of AML will be considered to have had an event. Participants who have not progressed to AML at the time of analysis will be censored at the last assessment date which does not indicate progression to AML.

    Measured: Week 1 Day 1 to first diagnosis of AML (up to approximately 44 months)

  10. Overall Survival

    Overall Survival is defined as time from Week 1 Day 1 to a participants death date or last known alive date. Participants who die, regardless of the cause of death, will be considered to have had an event. Participants who are alive at the time of analysis will be censored at the last assessment date at which the participant was known to be alive. All participants who were lost to follow-up will also be censored at the time of last contact.

    Measured: Week 1 Day 1 to to a participants death date or last known alive date (up to approximately 44 months)

  11. Number of Participants With Adverse Events (AEs)

    Treatment-emergent adverse events include adverse events that started on or after the first dose until 42 days after the last dose, as well as those SAEs made known to the investigator at any time thereafter that are suspected of being related to study drug. An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study.

    Grade 3 = Severe Grade 4 = Life-threatening Grade 5 = Death

    Measured: From first dose to 42 days after last dose (up to approximately 42.5 months)

  12. Maximum Plasma Concentration (Cmax)

    Measured: W1D1 (pre-dose), W1D3, W2D1, W2D3, W3D1, W4D1, W10D1, W13D1, W16D1, W17D1, W18D1, W19D1, W22D1, 24-Week MDS Disease Assessment, and every 12 weeks from the 24-Week MDS Disease Assessment Visit for up to 1 year from the first dose.

  13. Time to Maximum Plasma Concentration (Tmax)

    Measured: W1D1 (pre-dose), W1D3, W2D1, W2D3, W3D1, W4D1, W10D1, W13D1, W16D1, W17D1, W18D1, W19D1, W22D1, 24-Week MDS Disease Assessment, and every 12 weeks from the 24-Week MDS Disease Assessment Visit for up to 1 year from the first dose.

  14. Area Under the Concentration-Time Curve (AUC21d)

    Measured: W1D1 (pre-dose), W1D3, W2D1, W2D3 , W3D1, W4D1 (21 days after dose administration)

  15. Number of Participants With a Positive Anti-Drug Antibody (ADA) Test

    Number of participants under each ADA positive category. A participant is counted as 'Treatment-Emergent' if there is a positive post-baseline sample while the baseline sample is ADA negative, or there is a positive post-baseline sample with a titer >= 4-fold of the baseline titer while the baseline sample is ADA positive. A participant is counted as 'Preexisting' if the baseline sample is ADA positive and the participant is not qualified for 'Treatment-Emergent'. Positive to preexisting ADA is considered baseline sample is positive and all post-baseline samples are negative, or both baseline and post-baseline samples are positive, but all positive post-baseline sample have a titer < 4-fold of the baseline titer.

    Measured: W1D1 (must be collected before the first dose), W4D1, W10D1, W16D1, W22D1, 24-Week MDS Disease Assessment Visit and every 12 weeks (± 14 days) from the 24-Week MDS Disease Assessment Visit for up to 1 year from the first dose..

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2019
  2. Study start20 May 2019
  3. Primary completion1 Jul 2022
  4. Study completion1 Mar 2023

Resources

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
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03900715
Sponsor's protocol ID
ACE-536-MDS-003
Other IDs
U1111-1224-6268
Status last verified
Oct 2024
First submitted
14 Mar 2019
Last updated
17 Oct 2024
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Celgene at 19 sites in Japan.

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 17 inclusion and 50 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: finished, as of the sponsor's last update on 17 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in Japan. 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?

Yes. Results are posted in the registry (first posted 17 Oct 2024). 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?

Percentage of Participants Who Achieved Hematologic Improvement in Erythroid Response (HI-E) Per International Working Group (IWG) Through Week 24 (time frame: Week 1 Day 1 through Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03900715.

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 17 Oct 2024.

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