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Home › Conditions › Beta-thalassemia

Beta-thalassemia

0 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Beta-thalassemia

7 trials tracked for Beta-thalassemia: 3 in phase 3 or 4 and 3 with published results. The most-cited published study has 284 citations.

7Trials tracked
3Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 3 3 Phase 2 3 Other / NA 1
  1. Phase 3 A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype Completed · 284 cited
  2. Phase 3 An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia Completed · 119 cited
  3. Phase 3 A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia Completed · 51 cited
  4. Phase 2 Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia Completed
  5. Phase 2 Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment Completed
  6. Phase 2 Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload Completed
Show 1 more trials
  1. N/A Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada Completed

Showing the 7 most-cited and recently-updated of 7 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Beta-thalassemia: what the trials found

LentiGlobin BB305 Drug Product has demonstrated high efficacy in achieving transfusion independence (TI) for patients with beta-thalassemia. In two separate trials, 91.3% 1 and 88.9% 3 of participants achieved TI, with consistent results maintained at month 24 [1, 3]. Additionally, LentiGlobin BB305 showed high rates of transfusion reduction (TR) at 94.4% 3. Regarding specific reduction thresholds, 91.3% of participants achieved a 90% or 100% reduction in annualized pRBC transfusion volume 1.

Luspatercept demonstrated significant improvements in erythroid response and reduction of transfusion burden. Specifically, 21.0% of participants achieved an erythroid response from week 13 to 24 2. Furthermore, 19.6% of participants achieved a ≥ 33% reduction in transfusion burden from week 37 to 48 2, and 7.1% achieved a ≥ 50% reduction from week 13 to 24 2.

Recent results — preliminary, needs further review

  • VIT-2763 (once daily) is currently being evaluated in a Phase 2 trial 4.
  • Deferasirox was evaluated for its impact on liver iron concentration (LIC) and left ventricular ejection fraction (LVEF) over 101 weeks 5.
  • SPD602 (FBS0701, SSP-004184) was evaluated for changes in liver iron concentration at 96 weeks 6.

For the clinician treating this condition

  • LentiGlobin BB305 provides a high probability of achieving transfusion independence (TI) and significant reductions in transfusion volume [1, 3].
  • Luspatercept is associated with statistically significant improvements in erythroid response and reductions in transfusion burden 2.

AI synthesis of 6 cited trials, updated Sep 14, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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