A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia

Phase 2RecruitingNCT06302491

Kind Pharmaceuticals LLC64 enrolled

Overview

This is a phase II, randomized, double-blinded, placebo-controlled study to treat patients with transfusion-dependent and non-transfusion dependent β -thalassemia with AND017 and optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

β -Thalassemia

Interventions

AND017 capsulesDRUG

Administer AND017 capsules once per day (QD)

AND017 PlaceboDRUG

Administer AND017 matching placebo capsules once per day (QD)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Documented diagnosis of β-thalassemia or hemoglobin E/β-thalassemia, HbS/ β-thalassemia (β-thalassemia with α-bead mutation and/or multiplication is not allowed). 2. TDT subjects: receive regular blood transfusions, defined as 6-20 RBC units (including threshold) in the 24 weeks prior to screening assessment, and no transfusion-free period of ≥ 5 weeks during this period. 3. NTDT cohort: having transfused \<6 RBC units in the 24 weeks prior to the screening assessment, no regular transfusion schedule, and no transfusion for 4 weeks prior to the screening assessment. 4. Subject transfusion records should be obtained within 24 weeks prior to the screening assessment, containing the date of transfusion, transfused RBC units, and pre-transfusion hemoglobin values. 5. ECOG score 0-1. 6. NTDT subjects with Hb ≤ 10.0 g/dL at screening test and one follow-up test (two tests more than one week apart) and difference in values between the two tests ≤ 1.0 g/dL. 7. Adequate liver function: Total bilirubin \< 1.5 x upper limit of normal (ULN) (subjects with Gilbert syndrome, i.e., unconjugated hyperbilirubinemia, have a total bilirubin \< 3 x ULN), aspartate aminotransferase Exclusion Criteria: 1. Other causes of anemia (e.g., hemolytic anemia, history of pure red blood cell aplastic anemia, myelodysplastic syndrome, or multiple myeloma) 2. Presence of active infection or inflammatory disease requiring systemic anti-infective therapy, including concomitant autoimmune diseases with inflammatory symptoms (e.g. generalized erythema, ankylosing spondylitis, rheumatoid arthritis, psoriatic arthritis, dry syndrome, etc.) 3. Complicated retinal neovascularization requiring treatment (diabetic proliferative retinopathy, age-related exudative macular degeneration, retinal vein occlusion, macular edema, etc.) 4. Inability to take oral medications, conditions with a history of gastrectomy/bowel resection that may have an effect on the absorption of gastrointestinal medications (excluding gastric polyps or colonic polypectomy), or gastroparesis that remains symptomatic on current therapy 5. Clinically significant bleeding (requiring emergency blood transfusion within 12 h or a decrease in hemoglobin ≥ 2 g/dL within one week) within 4 weeks prior to the first dose, or a tendency to bleed or risk of bleeding that has not been medically or surgically corrected 6. Uncontrolled hypertension, defined as a diastolic blood pressure value \>95 mmHg or a systolic blood pressure \>160 mmHg on 2 or more of 3 repeated blood pressure tests (each at least 5 minutes apart) during the screening period 7. Complicated congestive heart failure (New York Heart Association \[NYHA\] class III or higher). 8. history of stroke, transient ischemic attack (TIA), myocardial infarction, thromboembolic event (deep vein thrombosis, DVT), pulmonary embolism, or pulmonary infarction within 24 weeks prior to screening evaluation 9. history of significant coagulation abnormalities, or platelet count \>600 x 109/L or \<80 x 109/L 10. History of epilepsy or any past seizures.

Locations (5)

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, China

NOT_YET_RECRUITING

Maoming People's Hospital

Maoming, Guangdong, China

RECRUITING

Liuzhou People's Hospital

Liuchow, Guangxi, China

RECRUITING

Outcomes

Primary Outcomes

Evaluate the safety and tolerability of different oral doses of AND017 in the treatment of β-thalassemia subjects

Evaluate the safety and tolerability of different oral doses of AND017 in the treatment of β-thalassemia subjects by AE rate by CTCAE 5.0

Time frame: From baseline to Week 24 or End of Treatment if discontinue early

Secondary Outcomes

Change in mean Hb levels relative to baseline at weeks 8-12 and week 20-24 post-treatment compared to baseline (mean Hb values during the 4 weeks prior to the first dose).

For NTDT cohort, evaluation of the effect of AND017+BSC on Hb levels.

Time frame: Baseline, Week 8-12, Week 20-24

The level of Hb and the change from baseline at each visit throughout the treatment period.

For NTDT cohort, evaluation of the effect of AND017+BSC on Hb levels.

Time frame: From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24

Proportion of patients with mean Hb elevation ≥1.0 g/dL from baseline to weeks 8-12 after dosing.

For NTDT cohort, evaluation of the effect of AND017+BSC on Hb levels.

Time frame: Baseline, Week 8-12

Levels of and changes from baseline in red blood cell count throughout the treatment period

For NTDT cohort, Evaluation of the effect of AND017+BSC on RBC count

Time frame: From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24

Levels of and changes from baseline in reticulocyte count throughout the treatment period

For NTDT cohort, Evaluation of the effect of AND017+BSC on reticulocyte count

Time frame: From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24

Central Contacts

Yusha Zhu, MD, PhD

CONTACT

6467252552 yushazhu@kindpharmaceutical.com

Data from ClinicalTrials.gov

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