nctId: NCT06302491 id: AND017-BTH-205 briefTitle: A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia overallStatus: NOT_YET_RECRUITING date: 2024-05 date: 2025-12 date: 2026-07 date: 2024-03-08 date: 2024-03-08 name: Kind Pharmaceuticals LLC class: INDUSTRY briefSummary: 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. conditions: β -Thalassemia studyType: INTERVENTIONAL phases: PHASE2 allocation: RANDOMIZED interventionModel: PARALLEL primaryPurpose: TREATMENT masking: NONE count: 64 type: ESTIMATED name: AND017 capsules name: AND017 Placebo measure: Evaluate the safety and tolerability of different oral doses of AND017 in the treatment of β-thalassemia subjects measure: 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). measure: The level of Hb and the change from baseline at each visit throughout the treatment period. measure: Proportion of patients with mean Hb elevation ≥1.0 g/dL from baseline to weeks 8-12 after dosing. measure: Levels of and changes from baseline in red blood cell count throughout the treatment period measure: Levels of and changes from baseline in reticulocyte count throughout the treatment period measure: Levels of and changes from baseline in mean corpuscular volume (MCV) throughout the treatment period measure: Levels of and changes from baseline in mean corpuscular hemoglobin (MCH) throughout the treatment period measure: Levels of and changes from baseline in mean corpuscular hemoglobin concentration (MCHC) throughout the treatment period measure: Throughout the treatment period, changes in the levels and relative baseline of transferrin will be assessed. measure: Throughout the treatment period, changes in the levels and relative baseline of transferrin saturation (TSAT) will be assessed. measure: Throughout the treatment period, changes in the levels and relative baseline of ferritin will be assessed. measure: Throughout the treatment period, changes in the levels and relative baseline of serum iron level will be assessed. measure: Throughout the treatment period, changes in the levels and relative baseline of total iron binding capacity (TIBC) will be assessed. measure: Change in transfusion load (units transfused) at 12-24 weeks post-dose compared to baseline (12 weeks to W0 before first dose). measure: Change in number of transfusions at 12-24 weeks post-dose compared to baseline (12 weeks to W0 before first dose). measure: Proportion of subjects with ≥33% reduction in transfusion load (transfusion units) relative to baseline (12 weeks prior to first dose to W0) from baseline to any consecutive 12-week period after dosing. measure: Duration (days) of maintenance below this transfusion dose after a 33% reduction in transfusion load from baseline has been achieved. sex: ALL minimumAge: 18 Years maximumAge: 65 Years stdAges: ADULT stdAges: OLDER_ADULT hasResults: False