Treatment of β-thalassemia with AND017

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

This study is testing a new drug called AND017 to see if it can help people with β-thalassemia feel better compared to standard treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 64 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Kind Pharmaceuticals LLC (Industry-sponsored)
• Locations: 5 sites (Guangzhou, Guangdong and 4 other locations)
• Trial ID: NCT06302491 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include patients diagnosed with β-thalassemia or hemoglobin E/β-thalassemia, either transfusion-dependent or non-transfusion dependent.

Not a fit: Patients with β-thalassemia who have not met the specific transfusion criteria or have certain genetic mutations may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of β-thalassemia, potentially reducing the need for blood transfusions.

How similar studies have performed: While there have been studies on treatments for β-thalassemia, the specific approach using AND017 is novel and has not been extensively tested in prior trials.

Eligibility criteria

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 this trial is running

Guangzhou, Guangdong and 4 other locations

• Nanfang Hospital Southern Medical University — Guangzhou, Guangdong, China (Not_yet_recruiting)
• 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)

Study contacts

• Study coordinator: Yusha Zhu, MD, PhD

Conditions β -Thalassemia