Myelodysplastic SyndromesPhase 2NCT06304103
A Study of Efficacy and Safety of AND017 in Patients With Myelodysplastic Syndrome
A Phase 2 study testing AND017 in people with Myelodysplastic Syndromes, run by Kind Pharmaceuticals LLC at 1 site in China.
Trial summary
This is a Phase 2, multicenter, randomized, open-lable, dose ranging study to evaluate the efficacy and safety of AND017 for the treatment of anemia due to lower risk Myelodysplastic syndromes (MDS) in patients subjects who are Red blood cell (RBC) non-transfusion dependent (NTD) and low transfusion burden (LTB). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- AND017
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 1 siteChina
- Study dates
- 14 Mar 2025to May 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed of primary myelodysplastic syndrome with a PISS-R grading of very low, low or intermediate risk and a bone marrow primitive cell count < 5%, the time frame for this grading assessment should be at least 12 weeks prior to the first dose
- Non-5q(del)-associated myelodysplastic syndrome.
- Two non-transfused hemoglobin ≥ 6.0 g/dL and < 10.0 g/dL, averaged over the screening period, at least one week and more apart, and with no more than 1.3 g/dL difference between the two Hb.
- Non-transfused subjects (NTD cohort) defined as no red blood cell transfusion in the 16 weeks prior to randomization or low transfusion load subjects defined as 3-7 pRBC units transfused in the 16 weeks prior to randomization and at least two different time points (LTB-1 cohort) or 1-2 pRBC units transfused at one time point in the 16 weeks prior to randomization ( LTB-2 cohort) (except in the case of transfusion for treatment of other comorbidities such as blood loss, surgery, etc.);
- Baseline EPO level ≤ 500 mU/mL
- Platelets ≥ 30,000 /mm3 and absolute neutrophil count ≥ 800/mm3
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- Adequate liver function with:
- Total bilirubin <2 x upper limit of normal (ULN) (subjects with Gilbert's syndrome, i.e., unconjugated hyperbilirubinemia, have a total bilirubin <3 x ULN)
- Aspartate aminotransferase (AST) <3 x ULN
- Alanine aminotransferase (ALT) <3×ULN
Who cannot join
- Diagnosed of secondary myelodysplastic syndrome or concurrent anemia from a cause other than the primary myelodysplastic syndrome.
- Significant myelofibrosis (fibrosis ≥ 2+).
- Planned clearing chemotherapy or whole brain spinal cord radiotherapy during the study period.
- Previous diagnosis of MDS IPSS-R high or very high risk.
- Prior or planned hematopoietic stem cell transplant during the study period.
- Received granulocyte colony-stimulating factor (G-CSF), or thrombopoietin, or thrombopoietin receptor agonist therapy within 8 weeks prior to the first dose;
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- Treatment with antithymocyte globulin, azacitidine, decitabine, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to the first dose.
- The presence of active infection or inflammatory disease requiring systemic anti-infective therapy, including concomitant autoimmune disease with inflammatory symptoms (e.g., generalized erythema, ankylosing spondylitis, rheumatoid arthritis, psoriatic arthritis, dry syndrome, celiac disease, etc.)
- Concurrent retinal neovascularization requiring treatment (diabetic proliferative retinopathy, age-related exudative macular degeneration, retinal vein occlusion, macular edema, etc.)
- Inability to take oral medications, or a history of gastrectomy, concomitant gastroparesis, or other conditions that may have an impact on the absorption of gastrointestinal medications (excluding gastric polyps or colonic polypectomy)
- Clinically significant bleeding (including transfusions required to treat bleeding or bleeding resulting in a decrease in hemoglobin ≥ 2 g/dL) within 4 weeks prior to the first dose, or a bleeding constitutional or bleeding risk that has not been medically or surgically corrected.
- Uncontrolled hypertension (more than one-third of identifiable diastolic blood pressure values ≥ 100 mmHg and/or systolic blood pressure ≥ 160 mmHg at 16 weeks prior to and including screening testing)
- Comorbid heart failure (New York Heart Association [NYHA] class III or higher)
- Medical history of significant liver disease or active liver disease at screening assessment
- Have been treated with any other hypoxia-inducing factor-prolyl hydroxylase inhibitor (HIF-PHI) in the 8 weeks prior to the first dose
- Have been treated with an erythropoietic ESA within 8 weeks prior to the first dose
- Have been treated with an androgenic anabolic steroid, testosterone enanthate or methandrostenolone within 8 weeks prior to the first dose
- Have been treated with an iron chelator within 8 weeks prior to the first dose
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 30003
Huili Zhou, 0571-87236685, yixuelunli123@163.com
About this study
Official title: An Efficacy and Safety Study of AND017 for the Treatment of Anemia Due to Lower Risk Myelodysplastic Syndromes (MDS)
This is a Phase 2, multicenter, randomized, open-lable, dose ranging study to evaluate the efficacy and safety of AND017 for the treatment of anemia due to lower risk Myelodysplastic syndromes (MDS) in patients subjects who are Red blood cell (RBC) non-transfusion dependent (NTD) and low transfusion burden (LTB).
- Conditions
- Myelodysplastic Syndromes
- Being tested
- AND017
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AND017Drug
Administer AND017 once per day (QD)
| Study group | Role | What they receive |
|---|---|---|
| AND017 capsules 4 mg | Experimental | Not described Receives: Drug: AND017 |
| AND017 capsules 8 mg | Experimental | Not described Receives: Drug: AND017 |
| AND017 capsules 12 mg | Experimental | Not described Receives: Drug: AND017 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of HI-E/RBC-TI responding subjects
- NTD cohort: non-transfused Hb levels ≥1.5 g/dL relative to baseline* during any 8-week period from baseline to 24 weeks post-dose.
- LTB-1 cohort: No transfusion during any 8-week period from baseline to 24 weeks post-dose.
- LTB-2 cohort: No transfusions during any 8-week period from baseline to 24 weeks post-dose and untransfused Hb levels ≥1.5 g/dL.
- Hb baseline is the average of the two lowest Hb over the 16 weeks prior to the first dose
Measured: From baseline to up to Week 25
Secondary outcomes (10)
For changes in mean transfusion units throughout the treatment period compared to baseline (mean transfusion units 16 weeks prior to first dose)
For changes in mean transfusion units throughout the treatment period compared to baseline (mean transfusion units 16 weeks prior to first dose)
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
For LTB cohorts, the average time required to reach first transfusion independence throughout the treatment period
For LTB cohorts, the average time required to reach first transfusion independence throughout the treatment period
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of reticulocyte count at each visit and change from baseline
Levels of reticulocyte count at each visit and change from baseline
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of hematocrit at each visit and change from baseline
Levels of hematocrit at each visit and change from baseline
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of mean corpuscular volume at each visit and change from baseline
Levels of mean corpuscular volume at each visit and change from baseline
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of mean corpuscular hemoglobin at each visit and change from baseline
Levels of mean corpuscular hemoglobin at each visit and change from baseline
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean level and change from baseline in non-transfused Hb at each visit throughout the treatment period and
Mean level and change from baseline in non-transfused Hb at each visit throughout the treatment period
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean level and change from baseline in non-transfused Hb throughout the first 8 weeks of treatment
Mean level and change from baseline in non-transfused Hb throughout the first 8 weeks of treatment
Measured: Baseline, Week 3, 5, 7, and 9
Percentage of visits in which non-transfused Hb was maintained in this range after reaching two consecutive increases of ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Percentage of visits in which non-transfused Hb was maintained in this range after reaching two consecutive increases of ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean time required after two consecutive increases in non-transfused Hb ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Mean time required after two consecutive increases in non-transfused Hb ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2024
- Study start14 Mar 2025
- Primary completionDec 2026 (estimated)
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 63 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kind Pharmaceuticals LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yusha Zhu, MD, PhD, Study director, Kind Pharmaceuticals LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06304103
- Sponsor's protocol ID
- AND017-MDS-206
- Status last verified
- Feb 2026
- First submitted
- 22 Mar 2023
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Efficacy and Safety Study of AND017 for the Treatment of Anemia Due to Lower Risk Myelodysplastic Syndromes (MDS)
- ClinicalTrials.gov ID
- NCT06304103
- Acronym / sponsor ID
- AND017-MDS-206
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 63 participants (estimated)
- Lead sponsor
- Kind Pharmaceuticals LLCIndustry (pharma, biotech, devices)
- Study start
- 14 Mar 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- May 2027 (estimated)
- First posted
- 12 Mar 2024 (actual)
- 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.
This is a Phase 2, multicenter, randomized, open-lable, dose ranging study to evaluate the efficacy and safety of AND017 for the treatment of anemia due to lower risk Myelodysplastic syndromes (MDS) in patients subjects who are Red blood cell (RBC) non-transfusion dependent (NTD) and low transfusion burden (LTB).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AND017 capsules 4 mg | Experimental | Drug: AND017 |
| AND017 capsules 8 mg | Experimental | Drug: AND017 |
| AND017 capsules 12 mg | Experimental | Drug: AND017 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosed of primary myelodysplastic syndrome with a PISS-R grading of very low, low or intermediate risk and a bone marrow primitive cell count < 5%, the time frame for this grading assessment should be at least 12 weeks prior to the first dose
- Non-5q(del)-associated myelodysplastic syndrome.
- Two non-transfused hemoglobin ≥ 6.0 g/dL and < 10.0 g/dL, averaged over the screening period, at least one week and more apart, and with no more than 1.3 g/dL difference between the two Hb.
- Non-transfused subjects (NTD cohort) defined as no red blood cell transfusion in the 16 weeks prior to randomization or low transfusion load subjects defined as 3-7 pRBC units transfused in the 16 weeks prior to randomization and at least two different time points (LTB-1 cohort) or 1-2 pRBC units transfused at one time point in the 16 weeks prior to randomization ( LTB-2 cohort) (except in the case of transfusion for treatment of other comorbidities such as blood loss, surgery, etc.);
- Baseline EPO level ≤ 500 mU/mL
Key exclusion criteria (18 listed)
- Diagnosed of secondary myelodysplastic syndrome or concurrent anemia from a cause other than the primary myelodysplastic syndrome.
- Significant myelofibrosis (fibrosis ≥ 2+).
- Planned clearing chemotherapy or whole brain spinal cord radiotherapy during the study period.
- Previous diagnosis of MDS IPSS-R high or very high risk.
- Prior or planned hematopoietic stem cell transplant during the study period.
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 |
|---|---|---|
| AND017Administer AND017 once per day (QD) | Drug | AND017 capsules 12 mg, AND017 capsules 4 mg, AND017 capsules 8 mg |
Primary outcome
Percentage of HI-E/RBC-TI responding subjects
Time frame: From baseline to up to Week 25
* NTD cohort: non-transfused Hb levels ≥1.5 g/dL relative to baseline* during any 8-week period from baseline to 24 weeks post-dose. * LTB-1 cohort: No transfusion during any 8-week period from baseline to 24 weeks post-dose. * LTB-2 cohort: No transfusions during any 8-week period from baseline to 24 weeks post-dose and untransfused Hb levels ≥1.5 g/dL. * Hb baseline is the average of the two lowest Hb over the 16 weeks prior to the first dose
10 secondary outcomes
For changes in mean transfusion units throughout the treatment period compared to baseline (mean transfusion units 16 weeks prior to first dose)
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
For LTB cohorts, the average time required to reach first transfusion independence throughout the treatment period
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of reticulocyte count at each visit and change from baseline
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of hematocrit at each visit and change from baseline
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of mean corpuscular volume at each visit and change from baseline
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Levels of mean corpuscular hemoglobin at each visit and change from baseline
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean level and change from baseline in non-transfused Hb at each visit throughout the treatment period and
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean level and change from baseline in non-transfused Hb throughout the first 8 weeks of treatment
Time frame: Baseline, Week 3, 5, 7, and 9
Percentage of visits in which non-transfused Hb was maintained in this range after reaching two consecutive increases of ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Mean time required after two consecutive increases in non-transfused Hb ≥1.5 g/dL and ≥1.0 g/dL from baseline, respectively, throughout the treatment period
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
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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.
Yusha Zhu, MD, PhD study director
Kind Pharmaceuticals LLC
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 AND017 in people with Myelodysplastic Syndromes, run by Kind Pharmaceuticals LLC at 1 site in China.
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 10 inclusion and 18 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: looking for participants now, as of the sponsor's last update on 27 Feb 2026. 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 China. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yusha Zhu, MD, PhD (6467252552, yushazhu@kindpharmaceutical.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of HI-E/RBC-TI responding subjects (time frame: From baseline to up to Week 25). 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 27 Feb 2026.