Brief Summary

This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias

Conditions

• Hereditary Red Blood Cell Disorder (Disorder)

Intervention / Treatment

• Drug: Reblozyl

Primary Outcome Measures

• Evaluating Luspatercept in patients affected with rare inherited anemias - Transfusion dependent patients (Up to 52 weeks)
  the proportion of patients who achieve an erythroid response, defined as a reduction in the transfusion burden of at least 33% from baseline (the 12-week period before the first dose of luspatercept) during 12 weeks plus a reduction of at least 2 red cell units over this 12-week interval.
• Evaluating Luspatercept in patients affected with rare inherited anemias - Non Transfusion dependent patients (Up to 52 weeks)
  the proportion of patients with a mean hemoglobin concentration increase of 1.0 g/dL or higher from baseline over a continuous 12-week interval in the absence of red blood cell transfusions

Countries

France, Italy

Locations and Investigators

• GCS Groupement des Hôpitaux de l'Institut Catholique de Lille, Lille, France
  • Benjamin Carpentier, Phd
• Centre Hospitalier Universitaire de Montpellier, Montpellier, France
  • Patricia Aguilar Martinez, Phd
• Assistance Publique Hôpitaux de Paris - Hôpital Saint-Louis, Paris, France
  • Pierre Fenaux, MD/PhD
• Assistance Publique Hôpitaux de Paris - Hôpital Necker, Paris, France
  • Olivier Hermine, MD/Phd
• CHU Bordeaux-Hôpital Haut-Lévêque, Pessac, France
  • Chloé Dhunputh
• Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, Milan, Italy
  • Bruno Fattizzo

Study Officials

• Thierry Leblanc, Phd (Principal Investigator) — Assistance Publique Hôpitaux de Paris - Hôpital Robert-Debré