NCT06302491 A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
| NCT ID | NCT06302491 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Kind Pharmaceuticals LLC |
| Condition | β -Thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 64 participants |
| Start Date | 2024-05-27 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 64 participants in total. It began in 2024-05-27 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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.
Contact & Investigator
Yusha Zhu, MD, PhD
STUDY DIRECTOR
Kind Pharmaceuticals LLC
Frequently Asked Questions
Who can join the NCT06302491 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 65 Years, studying β -Thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06302491 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06302491 currently recruiting?
Yes, NCT06302491 is actively recruiting participants. Contact the research team at yushazhu@kindpharmaceutical.com for enrollment information.
Where is the NCT06302491 trial being conducted?
This trial is being conducted at Guangzhou, China, Maoming, China, Liuzhou, China, Nanning, China and 1 additional location.
Who is sponsoring the NCT06302491 clinical trial?
NCT06302491 is sponsored by Kind Pharmaceuticals LLC. The principal investigator is Yusha Zhu, MD, PhD at Kind Pharmaceuticals LLC. The trial plans to enroll 64 participants.