Treatment of β-thalassemia with AND017

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II clinical trial is a randomized, double-blinded, placebo-controlled study aimed at evaluating the safety and efficacy of AND017 in patients with β-thalassemia. Participants will receive either AND017 capsules or a placebo, along with optimal supportive care, which may include blood transfusions and iron removal as determined by their healthcare provider. The study will include both transfusion-dependent and non-transfusion dependent patients, with careful monitoring of their transfusion history and hemoglobin levels. The goal is to assess how well AND017 can improve patient outcomes compared to standard care.

Why it matters

Who can join: the full rules

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.

Where it's running

• Maoming People's Hospital
  Maoming, Guangdong, China Recruiting
• Liuzhou People's Hospital
  Liuzhou, Guangxi, China Recruiting
• Guangxi Medical University No.1 Affiliated Hospital
  Nanning, Guangxi, China Recruiting
• Hainan General Hospital
  Haikou, Hainan, China Recruiting
• Nanfang Hospital Southern Medical University
  Guangzhou, Guangdong, China Not yet recruiting

Study contacts

• Study coordinator: Yusha Zhu, MD, PhD
• Email: yushazhu@kindpharmaceutical.com
• Phone: 6467252552

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.