β -ThalassemiaPhase 2NCT06302491
A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
A Phase 2 study testing AND017 capsules in people with β -Thalassemia, run by Kind Pharmaceuticals LLC at 5 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- β -Thalassemia
- What is being tested?
- AND017 capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 5 sitesChina
- Study dates
- 27 May 2024to Jul 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
China5 sites, 5 recruiting
- Not yet recruiting
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, 510515
Lin Jiang, MD
- Recruiting
Hainan General Hospital
Haikou, Hainan, 570203
Guyun Wang, MD
- Recruiting
Liuzhou People's Hospital
Liuzhou, Guangxi, 545006
Min Wei, MD
- Recruiting
Maoming People's Hospital
Maoming, Guangdong, 525000
Xiong Zhang, MD
- Recruiting
Guangxi Medical University No.1 Affiliated Hospital
Nanning, Guangxi, 530021
Yongrong Lai, MD
About this study
Official title: 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.
- Conditions
- β -Thalassemia
- Being tested
- AND017 capsules, AND017 Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AND017 capsulesDrug
Administer AND017 capsules once per day (QD)
AND017 PlaceboDrug
Administer AND017 matching placebo capsules once per day (QD)
| Study group | Role | What they receive |
|---|---|---|
| AND017 capsules 8 mg | Experimental | Not described Receives: Drug: AND017 capsules |
| AND017 capsules 12 mg | Experimental | Not described Receives: Drug: AND017 capsules |
| AND017 capsules 16 mg | Experimental | Not described Receives: Drug: AND017 capsules |
| AND017 Placebo capsules | Placebo comparator | Not described Receives: Drug: AND017 Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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).
For NTDT cohort, evaluation of the effect of AND017+BSC on Hb levels.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: From baseline to Week 24 or End of Treatment if discontinue early
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2024
- Study start27 May 2024
- Primary completionDec 2026 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β -ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kind Pharmaceuticals LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yusha Zhu, MD, PhD, Study director, Kind Pharmaceuticals LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06302491
- Sponsor's protocol ID
- AND017-BTH-205
- Status last verified
- Feb 2026
- First submitted
- 22 Mar 2023
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia
- ClinicalTrials.gov ID
- NCT06302491
- Acronym / sponsor ID
- AND017-BTH-205
- Conditions
- β -Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Kind Pharmaceuticals LLCIndustry (pharma, biotech, devices)
- Study start
- 27 May 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 8 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AND017 capsulesAdminister AND017 capsules once per day (QD) | Drug | AND017 capsules 12 mg, AND017 capsules 16 mg, AND017 capsules 8 mg |
| AND017 PlaceboAdminister AND017 matching placebo capsules once per day (QD) | Drug | AND017 Placebo capsules |
Primary outcome
Evaluate the safety and tolerability of different oral doses of AND017 in the treatment of β-thalassemia subjects
Time frame: From baseline to Week 24 or End of Treatment if discontinue early
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
17 secondary outcomes
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).
Time frame: Baseline, Week 8-12, Week 20-24
The level of Hb and the change from baseline at each visit throughout the treatment period.
Time frame: 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.
Time frame: Baseline, Week 8-12
Levels of and changes from baseline in red blood cell count throughout the treatment period
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: From baseline to Week 24 or End of Treatment if discontinue early
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yusha Zhu, MD, PhD study director
Kind Pharmaceuticals LLC
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AND017 capsules in people with β -Thalassemia, run by Kind Pharmaceuticals LLC at 5 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yusha Zhu, MD, PhD (6467252552, yushazhu@kindpharmaceutical.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety and tolerability of different oral doses of AND017 in the treatment of β-thalassemia subjects (time frame: From baseline to Week 24 or End of Treatment if discontinue early). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2026.