A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia

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

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.

Study Overview

• Status: Recruiting
• Conditions: β -Thalassemia
• Intervention / Treatment: Drug: AND017 capsules; Drug: AND017 Placebo

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Yusha Zhu, MD, PhD; Phone Number: 6467252552; Email: yushazhu@kindpharmaceutical.com

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Not yet recruiting
      • Nanfang Hospital Southern Medical University
      • Principal Investigator: Lin Jiang, MD
    • Maoming, Guangdong, China, 525000
      • Recruiting
      • Maoming People's Hospital
      • Principal Investigator: Xiong Zhang, MD
  • Guangxi
    • Liuchow, Guangxi, China, 545006
      • Recruiting
      • Liuzhou People's Hospital
      • Principal Investigator: Min Wei, MD
    • Nanning, Guangxi, China, 530021
      • Recruiting
      • Guangxi Medical University No.1 Affiliated Hospital
      • Principal Investigator: Yongrong Lai, MD
  • Hainan
    • Haikou, Hainan, China, 570203
      • Recruiting
      • Hainan General Hospital
      • Principal Investigator: Guyun Wang, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AND017 capsules 8 mg	Drug: AND017 capsules; Administer AND017 capsules once per day (QD)
Experimental: AND017 capsules 12 mg	Drug: AND017 capsules; Administer AND017 capsules once per day (QD)
Experimental: AND017 capsules 16 mg	Drug: AND017 capsules; Administer AND017 capsules once per day (QD)
Placebo Comparator: AND017 Placebo capsules	Drug: AND017 Placebo; Administer AND017 matching placebo capsules once per day (QD)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	From baseline to Week 24 or End of Treatment if discontinue early

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline, Week 8-12, Week 20-24
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.	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
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.	Baseline, Week 8-12
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	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
Levels of and changes from baseline in reticulocyte count throughout the treatment period	For NTDT cohort, Evaluation of the effect of AND017+BSC on reticulocyte count	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
Levels of and changes from baseline in mean corpuscular volume (MCV) throughout the treatment period	For NTDT cohort, Evaluation of the effect of AND017+BSC on the MCV	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
Levels of and changes from baseline in mean corpuscular hemoglobin (MCH) throughout the treatment period	For NTDT cohort, Evaluation of the effect of AND017+BSC on MCH	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
Levels of and changes from baseline in mean corpuscular hemoglobin concentration (MCHC) throughout the treatment period	For NTDT cohort, Evaluation of the effect of AND017+BSC on MCHC	From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24
Throughout the treatment period, changes in the levels and relative baseline of transferrin will be assessed.	For NTDT cohort, evaluation of the effect of AND017+BSC on transferrin level	From baseline to Week 4, 8, 12, 16, 20, 24
Throughout the treatment period, changes in the levels and relative baseline of transferrin saturation (TSAT) will be assessed.	For NTDT cohort, evaluation of the effect of AND017+BSC on TSAT.	From baseline to Week 4, 8, 12, 16, 20, 24
Throughout the treatment period, changes in the levels and relative baseline of ferritin will be assessed.	For NTDT cohort, evaluation of the effect of AND017+BSC on ferritin	From baseline to Week 4, 8, 12, 16, 20, 24
Throughout the treatment period, changes in the levels and relative baseline of serum iron level will be assessed.	For NTDT cohort, evaluation of the effect of AND017+BSC on serum iron level	From baseline to Week 4, 8, 12, 16, 20, 24
Throughout the treatment period, changes in the levels and relative baseline of total iron binding capacity (TIBC) will be assessed.	For NTDT cohort, evaluation of the effect of AND017+BSC on TIBC	From baseline to Week 4, 8, 12, 16, 20, 24
Change in transfusion load (units transfused) at 12-24 weeks post-dose compared to baseline (12 weeks to W0 before first dose).	For TDT cohort, evaluate changes in mean transfusion load by AND017+BSC at 12 to 24 weeks after dosing	Baseline, Week 20-24, or End of Treatment if discontinue early
Change in number of transfusions at 12-24 weeks post-dose compared to baseline (12 weeks to W0 before first dose).	For TDT cohort, evaluate changes in mean transfusion load by AND017+BSC at 12 to 24 weeks after dosing	Baseline and Week 20-24
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.	For TDT cohort, 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.	Baseline, Week 0-12, 2-14, 4-16, 6-18, 8-20, 10-22, and 12-24
Duration (days) of maintenance below this transfusion dose after a 33% reduction in transfusion load from baseline has been achieved.	For TDT cohort, duration (days) of maintenance below this transfusion dose after a 33% reduction in transfusion load from baseline has been achieved.	From baseline to Week 24 or End of Treatment if discontinue early

Collaborators and Investigators

• Sponsor: Kind Pharmaceuticals LLC
• Investigators: Study Director: Yusha Zhu, MD, PhD, Kind Pharmaceuticals LLC

Study record dates

Study Major Dates

• Study Start (Actual): May 27, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027

Study Registration Dates

• First Submitted: March 22, 2023
• First Submitted That Met QC Criteria: March 4, 2024
• First Posted (Actual): March 8, 2024

Study Record Updates

• Last Update Posted (Actual): February 27, 2026
• Last Update Submitted That Met QC Criteria: February 25, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: AND017-BTH-205

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No