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RecruitingNCT06304103Updated Feb 27, 2026

A Study of Efficacy and Safety of AND017 in Patients With Myelodysplastic Syndrome

A Phase 2 interventional study of AND017 in Myelodysplastic Syndromes, sponsored by Kind Pharmaceuticals LLC. Recruiting at 1 site in China. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-02-27.

Sponsored by Kind Pharmaceuticals LLC · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Mar 2025; still recruiting 1 year 6 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
63
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
All
01

Study 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).

02

Conditions studied

  • Myelodysplastic Syndromes
03

In context

Myelodysplastic Syndromes

2,124 studies on the registry are indexed under Myelodysplastic Syndromes; 320 are open to participants now.

This study's planned enrollment of 63 is above the median of 39 across 1,740 interventional studies indexed under Myelodysplastic Syndromes.

Browse Myelodysplastic Syndromes studies →

Lead sponsor

Kind Pharmaceuticals LLC is the lead sponsor of 11 studies on the registry; 7 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. 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
  2. Non-5q(del)-associated myelodysplastic syndrome.
  3. 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.
  4. 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.);
  5. Baseline EPO level ≤ 500 mU/mL
  6. Platelets ≥ 30,000 /mm3 and absolute neutrophil count ≥ 800/mm3

  7. 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

Exclusion criteria

Exclusion Criteria:

  1. Diagnosed of secondary myelodysplastic syndrome or concurrent anemia from a cause other than the primary myelodysplastic syndrome.
  2. Significant myelofibrosis (fibrosis ≥ 2+).
  3. Planned clearing chemotherapy or whole brain spinal cord radiotherapy during the study period.
  4. Previous diagnosis of MDS IPSS-R high or very high risk.
  5. Prior or planned hematopoietic stem cell transplant during the study period.
  6. Received granulocyte colony-stimulating factor (G-CSF), or thrombopoietin, or thrombopoietin receptor agonist therapy within 8 weeks prior to the first dose;
  7. Treatment with antithymocyte globulin, azacitidine, decitabine, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to the first dose.
  8. 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.)
  9. Concurrent retinal neovascularization requiring treatment (diabetic proliferative retinopathy, age-related exudative macular degeneration, retinal vein occlusion, macular edema, etc.)
  10. 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)
  11. 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.
  12. 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)
  13. Comorbid heart failure (New York Heart Association [NYHA] class III or higher)
  14. Medical history of significant liver disease or active liver disease at screening assessment
  15. Have been treated with any other hypoxia-inducing factor-prolyl hydroxylase inhibitor (HIF-PHI) in the 8 weeks prior to the first dose
  16. Have been treated with an erythropoietic ESA within 8 weeks prior to the first dose
  17. Have been treated with an androgenic anabolic steroid, testosterone enanthate or methandrostenolone within 8 weeks prior to the first dose
  18. Have been treated with an iron chelator within 8 weeks prior to the first dose
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
63 participants (estimated)

Study arms

  • Experimental
    AND017 capsules 4 mg

    Drug: AND017

  • Experimental
    AND017 capsules 8 mg

    Drug: AND017

  • Experimental
    AND017 capsules 12 mg

    Drug: AND017

Interventions

  • DrugAND017

    Administer AND017 once per day (QD)

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: From baseline to up to Week 25

Secondary outcomes

  1. 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)

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25

  2. 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

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25

  3. Levels of reticulocyte count at each visit and change from baseline

    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

  4. Levels of hematocrit at each visit and change from baseline

    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

  5. Levels of mean corpuscular volume at each visit and change from baseline

    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

  6. Levels of mean corpuscular hemoglobin at each visit and change from baseline

    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

  7. 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

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25

  8. 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

    Time frame: Baseline, Week 3, 5, 7, and 9

  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

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25

  10. 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

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, and 25

07

Study locations

1 of 1 sites recruiting
  • First Affiliated Hospital of Zhejiang University School of Medicine
    Hangzhou, Zhejiang 30003, China
    Recruiting
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 27, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06304103
Lead sponsor
Kind Pharmaceuticals LLC
Responsible party
Sponsor
First posted
Mar 12, 2024
Start date
Mar 14, 2025
Primary completion
Dec 2026 (estimated)
Completion
May 2027 (estimated)
Last update
Feb 27, 2026

Study contacts

Yusha Zhu, MD, PhD
Contact
yushazhu@kindpharmaceutical.com
6467252552
Yusha Zhu, MD, PhD
study director · Kind Pharmaceuticals LLC

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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