About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.);

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

AND017
Drug
Administer AND017 once per day (QD)

Treatment groups

63 Participants

are divided into 3 treatment groups

Group A: AND017 capsules 4 mgExperimental treatment 1 intervention

Group B: AND017 capsules 8 mgExperimental treatment 1 intervention

Group C: AND017 capsules 12 mgExperimental treatment 1 intervention

Trial outcomes

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)

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

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

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

Time frame

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

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

About this trial

Eligibility criteria

Qualifiers

Disqualifiers

Trial design

Treatments tested in this trial

Treatment groups

Trial outcomes

Primary outcomes

Secondary outcomes

Other outcomes

Sponsors and contacts