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

NCT ID: NCT06302491

Last Updated: 2024-06-28

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 64 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-05-27
• Study Completion Date: 2026-07-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

AND017 capsules 8 mg

Group Type: EXPERIMENTAL

AND017 capsules

Intervention Type: DRUG

Administer AND017 capsules once per day (QD)

AND017 capsules 12 mg

Group Type: EXPERIMENTAL

AND017 capsules

Intervention Type: DRUG

Administer AND017 capsules once per day (QD)

AND017 capsules 16 mg

Group Type: EXPERIMENTAL

AND017 capsules

Intervention Type: DRUG

Administer AND017 capsules once per day (QD)

AND017 Placebo capsules

Group Type: PLACEBO_COMPARATOR

AND017 Placebo

Intervention Type: DRUG

Administer AND017 matching placebo capsules once per day (QD)

Interventions

AND017 capsules

Administer AND017 capsules once per day (QD)

Intervention Type: DRUG

AND017 Placebo

Administer AND017 matching placebo capsules once per day (QD)

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kind Pharmaceuticals LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yusha Zhu, MD, PhD

Role: STUDY_DIRECTOR

Kind Pharmaceuticals LLC

Locations

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, China

Site Status: NOT_YET_RECRUITING

Maoming People's Hospital

Maoming, Guangdong, China

Site Status: RECRUITING

Liuzhou People's Hospital

Liuzhou, Guangxi, China

Site Status: RECRUITING

Guangxi Medical University No.1 Affiliated Hospital

Nanning, Guangxi, China

Site Status: RECRUITING

Hainan General Hospital

Haikou, Hainan, China

Site Status: RECRUITING

Countries

China

Central Contacts

Yusha Zhu, MD, PhD

Role: CONTACT

yushazhu@kindpharmaceutical.com

6467252552

Other Identifiers

AND017-BTH-205

Identifier Type: -

Identifier Source: org_study_id