Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Condition(s) studied

Investigational drug(s) / intervention(s)

Luspatercept (ACE-536): Luspatercept (ACE-536)

Study summary

Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation.

The efficacy of luspatercept was demonstrated in the phase III clinical trial called "BELIEVE." More than 200 Italian patients with transfusion- dependent beta thalassemia aged ≥18 years who had no approved therapeutic alternatives to improve their clinical course were considered eligible for the 'compassionate' use program related to luspatercept and most of them received at least one dose of the drug before it was dispensed by the National Health System (NHS), after approval by the pharmaceutical company and the Ethics Committee of the Clinical Center in which they were being followed.

Eligibility

Primary outcome measure(s)

• To evaluate safety of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0 — 12-week interval period of luspatercept treatment.
  To evaluate safety of luspatercept in subjects with transfusion-dependent beta thalassemia who received at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.
• To evaluate tolerability of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0" — 12-week interval period of luspatercept treatment.
  To evaluate tolerability of luspatercept in subjects with transfusion-dependent beta thalassemia who received at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.
• To evaluate efficacy of luspatercept, Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment — 12-week interval period of luspatercept treatment.
  To evaluate efficacy of luspatercept in subjects with transfusion-dependent beta thalassemia who received at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.

Trial sites (1)

More Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia trials in Italy

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