A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
Conditionβ -Thalassemia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorKind Pharmaceuticals LLC
About this trial
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
This trial does not accept healthy volunteersQualifiers
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).
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.
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.
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.
Disqualifiers
Other causes of anemia (e.g., hemolytic anemia, history of pure red blood cell aplastic anemia, myelodysplastic syndrome, or multiple myeloma)
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.)
Complicated retinal neovascularization requiring treatment (diabetic proliferative retinopathy, age-related exudative macular degeneration, retinal vein occlusion, macular edema, etc.)
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
Trial design
Design model
Parallel
Treatments tested in this trial
AND017 capsules
DrugAdminister AND017 capsules once per day (QD)
AND017 Placebo
DrugAdminister AND017 matching placebo capsules once per day (QD)
Treatment groups
64 Participants
are divided into 4 treatment groupsGroup A: AND017 capsules 8 mgExperimental treatment 1 intervention
Group B: AND017 capsules 12 mgExperimental treatment 1 intervention
Group C: AND017 capsules 16 mgExperimental treatment 1 intervention
Group D: AND017 Placebo capsulesPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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 earlySecondary outcomes
1
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 20-242
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, 243
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 8-124
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, 24Sponsors and contacts
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Source ClinicalTrials.gov