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

NCT06302491 | Recruiting Phase 2

• 1 other identifier: AND017-BTH-205
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 5

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.

Conditions

β -Thalassemia

Outcome Measures

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

  From baseline to Week 24 or End of Treatment if discontinue early

Secondary Outcomes (17)

• 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).

  Baseline, Week 8-12, Week 20-24

• The level of Hb and the change from baseline at each visit throughout the treatment period.

  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.

  Baseline, Week 8-12

• Levels of and changes from baseline in red blood cell count throughout the treatment period

  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

  From baseline to Week1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24

Study Arms (4)

AND017 capsules 8 mg

EXPERIMENTAL

Drug: AND017 capsules

AND017 capsules 12 mg

EXPERIMENTAL

Drug: AND017 capsules

AND017 capsules 16 mg

EXPERIMENTAL

Drug: AND017 capsules

AND017 Placebo capsules

PLACEBO COMPARATOR

Drug: AND017 Placebo

Interventions

AND017 capsules DRUG

Administer AND017 capsules once per day (QD)

AND017 capsules 12 mg; AND017 capsules 16 mg; AND017 capsules 8 mg

AND017 Placebo DRUG

Administer AND017 matching placebo capsules once per day (QD)

AND017 Placebo capsules

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• ECOG score 0-1.
• 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.
• 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

You may not qualify if:

• 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
• 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
• 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
• Complicated congestive heart failure (New York Heart Association \[NYHA\] class III or higher).
• 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
• history of significant coagulation abnormalities, or platelet count \>600 x 109/L or \<80 x 109/L
• History of epilepsy or any past seizures.

Sponsors & Collaborators

• Kind Pharmaceuticals LLC: lead

Study Sites (5)

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, 510515, China

NOT YET RECRUITING

Maoming People's Hospital

Maoming, Guangdong, 525000, China

RECRUITING

Liuzhou People's Hospital

Liuchow, Guangxi, 545006, China

RECRUITING

Guangxi Medical University No.1 Affiliated Hospital

Nanning, Guangxi, 530021, China

RECRUITING

Hainan General Hospital

Haikou, Hainan, 570203, China

RECRUITING

Study Officials

• Yusha Zhu, MD, PhD

  Kind Pharmaceuticals LLC

  STUDY DIRECTOR

Central Study Contacts

Yusha Zhu, MD, PhD

CONTACT

6467252552; yushazhu@kindpharmaceutical.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 22, 2023
• First Posted: March 8, 2024
• Study Start: May 27, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: February 27, 2026

Record last verified: 2026-02

Locations

CN (5)