Thalassemia trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Thalassemia. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Upcoming readout windows
next 180 days · by monthNo Thalassemia study has a primary completion filed in the next 180 days. The registered pipeline below carries no window, so it still shows what is there.
Every registered study in Thalassemia
most recently updated first28 studies match.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| BMY | Bristol-Myers Squibb | $134.9B | A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle EastNCT07215975observationalChange in transfusion burden | — | Recruiting | Drug: Luspatercept | β-thalassemia | 200 | 2026-06-26 | 2030-02-28 | in 1,288 d | 2026-08-19 |
| NVO | Novo Nordisk A/S | — | A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaNCT06609226Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately | Phase 3 | Recruiting | Drug: Etavopivat A, Etavopivat B, Etavopivat C | Sickle Cell Disease, Thalassemia | 480 | 2025-01-10 | 2030-12-30 | in 1,593 d | 2026-08-13 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat StudyNCT05777993Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study | Phase 4 | Enrolling by invitation | Drug: Mitapivat | Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia | 59 | 2023-07-06 | Aug 2029 | ≤ 1,107 d | 2026-08-12 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | A Long-term Follow-up Study in Participants Who Received CTX001NCT04208529New malignancies | Phase 3 | Enrolling by invitation | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia | 160 | 2021-01-20 | 2039-09-30 | in 4,789 d | 2026-08-11 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)NCT07506863Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48 | Phase 3 | Recruiting | Drug: Mitapivat Matched Placebo, Mitapivat | Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | 54 | Sep 2026 | Jun 2029 | ≤ 1,045 d | 2026-08-03 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent ThalassemiaNCT07517133Percentage of Participants Who Achieved a Hemoglobin (Hb) Response | Phase 3 | Recruiting | Drug: Mitapivat, Placebo Matching Mitapivat | Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | 45 | Sep 2026 | Mar 2029 | ≤ 954 d | 2026-08-03 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)NCT04770753results2025-01-24Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline | Phase 3 | Active, not recruiting | Drug: Placebo Matching Mitapivat, Mitapivat | Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | 194 | 2021-12-20 | 2023-11-13actual | — | 2026-07-31 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)NCT04770779results2025-05-25Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) | Phase 3 | Active, not recruiting | Drug: Placebo Matching Mitapivat, Mitapivat | Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | 258 | 2021-11-30 | 2024-04-11actual | — | 2026-07-31 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemiaNCT07681440observationalParticipant age | — | Terminatedsponsor's stated reason: Insufficient enrollment | Drug: Luspatercept | Myelodysplastic Syndromes, Beta-thalassemia | 30 | 2023-11-16 | 2025-05-01actual | — | 2026-07-02 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)NCT05356195Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12) | Phase 3 | Active, not recruiting | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies | 16 | 2022-05-03 | 2027-11-14 | in 451 d | 2026-07-01 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent ThalassemiaNCT03692052results2021-11-15Percentage of Participants Achieving a Hemoglobin Response (HR) | Phase 2 | Active, not recruiting | Drug: AG-348 | Thalassemia | 20 | 2019-03-20 | 2020-08-20actual | — | 2026-03-30 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseNCT05477563Fetal Hemoglobin (HbF) Concentration Over Time | Phase 3 | Recruiting | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia | 26 | 2022-08-02 | 2027-06-09 | in 293 d | 2026-03-23 |
| NVO | Novo Nordisk A/S | — | A Study on the Effect of Etavopivat on Heart Rhythm in Healthy ParticipantsNCT07023029Part A: Number of treatment-emergent adverse events (AEs) | Phase 1 | Completed | Drug: Etavopivat, Moxifloxacin, Placebo | Healthy Volunteers, Sickle Cell Disease, Thalassemia | 33 | 2025-06-09 | 2025-09-12actual | — | 2026-01-02 |
| NVO | Novo Nordisk A/S | — | A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese ParticipantsNCT06581627AUC0-inf,etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose | Phase 1 | Completed | Drug: Etavopivat | Healthy Volunteers, Sickle Cell Disease, Thalassemia | 24 | 2024-08-26 | 2024-10-31actual | — | 2025-12-29 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-ThalassemiaNCT03655678Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) | Phase 2/3 | Completed | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies | 59 | 2018-09-14 | 2025-11-13actual | — | 2025-12-17 |
| NVO | Novo Nordisk A/S | — | A Research Study of the Effect of Food on Etavopivat in Healthy ParticipantsNCT06433661AUC0-inf, etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose | Phase 1 | Completed | Drug: Etavopivat | Healthy Volunteers Sickle Cell Disease, Thalassemia | 16 | 2024-05-28 | 2024-07-06actual | — | 2025-10-10 |
| NVO | Novo Nordisk A/S | — | A Research Study of the Effect of Etavopivat on Other Drugs in Healthy ParticipantsNCT06813924Cmax, digoxin, SD: Maximum observed digoxin plasma concentration with and without etavopivat at steady state | Phase 1 | Completed | Drug: Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin | Healthy Volunteers Sickle Cell Disease, Thalassemia | 37 | 2025-02-06 | 2025-05-27actual | — | 2025-09-25 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.NCT05567458Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks | Phase 2 | Active, not recruiting | Drug: Luspatercept, Placebo | Beta-thalassemia | 94 | 2022-10-17 | 2025-08-01actual | — | 2025-08-12 |
| EDIT | Editas Medicine, Inc. | $442M | EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)NCT05444894Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days) | Phase 1/2 | Active, not recruiting | Genetic: EDIT-301 | Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia | 9 | 2022-04-29 | Sep 2025 | — | 2025-04-02 |
| EDIT | Editas Medicine, Inc. | $442M | A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301NCT06363760observationalAdverse events of special interest (AESI) | — | Enrolling by invitation | Other: Safety and efficacy assessments | Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies | 54 | 2024-06-17 | Aug 2040 | ≤ 5,125 d | 2025-04-02 |
| SLN | Silence Therapeutics plc | $2.0B | A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic SyndromeNCT04718844Incidence of treatment-emergent adverse events | Phase 1 | Completed | Drug: SLN124, Placebo | Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome, Very-Low Risk Myelodysplastic Syndrome | 44 | 2021-04-14 | 2023-05-23actual | — | 2024-01-03 |
| PTGX | Protagonist Therapeutics, Inc. | $10.2B | Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic AnemiaNCT03802201NTD: Proportion of responders at each dose | Phase 2 | Completed | Drug: PTG-300 | β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia | 63 | 2018-12-19 | 2020-07-31actual | — | 2021-07-16 |
| PTGX | Protagonist Therapeutics, Inc. | $10.2B | Safety Study for Beta Thalassemia Subjects on PTG-300NCT04054921Proportion of subjects with side effects and severity of side effects will be tabulated | Phase 2 | Completed | Drug: PTG-300 | β-thalassemia, Ineffective Erythropoiesis | 34 | 2019-05-10 | 2020-07-31actual | — | 2021-07-16 |
| NVS | Novartis | — | Cardiac T2* in Beta-thalassemia Patients on Deferasirox TreatmentNCT00447694results2021-06-07Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2* | Phase 2 | Completed | Drug: Deferasirox | Beta-thalassemia, Iron Overload | 30 | Feb 2006 | Nov 2009actual | — | 2021-06-14 |
| SLN | Silence Therapeutics plc | $2.0B | Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124NCT04176653# of participants with all AEs as assessed by CTCAE V4.0 included injection site reaction, will be measured from baseline to post dose follow up | Phase 1 | Withdrawnsponsor's stated reason: Study stopped prior to subject administered first dose, due to COVID-19 | Drug: SLN124 is a GalNAc conjugated double stranded fully modified siRNA. Sodium chloride 0.9% w/v is used as Placebo | Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome | 0 | 2019-08-20 | 2021-09-30 | — | 2020-04-27 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersNCT03165864Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx | Phase 1 | Completed | Drug: IONIS TMPRSS6-Lrx · Other: Placebo | Thalassemia | 36 | 2017-05-09 | 2018-09-25actual | — | 2019-01-22 |
| CERS | Cerus Corporation | $556M | Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC TransfusionNCT01740531Primary Efficacy Endpoint - Hemoglobin consumption | Phase 3 | Completed | Biological: S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells | Thalassemia Major | 86 | Dec 2012 | 2017-12-21actual | — | 2018-07-18 |
| NVS | Novartis | — | A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional HemosiderosisNCT00171171Liver Iron Concentration (as measured by biopsy) | Phase 3 | Completed | Drug: deferasirox | Beta-Thalassemia, Hemosiderosis | 252 | May 2004 | Nov 2006actual | — | 2017-03-01 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
How Thalassemia is matched — 10 exact registry spellings
A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.
- Thalassemia
- Beta-Thalassemia
- Transfusion-dependent Beta-Thalassemia
- β-thalassemia
- Healthy Volunteers Sickle Cell Disease, Thalassemia
- Non-Transfusion-dependent Alpha-Thalassemia
- Non-Transfusion-dependent Beta-Thalassemia
- Non-transfusion-dependent Thalassemia
- Thalassemia Major
- Transfusion-dependent Alpha-Thalassemia
Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “Thalassemia” (a text search, wider than this page)