lovotibeglogene autotemcel for Sickle cell disease

Inclusion criteria

• {"criterion_text":"- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)"}
• {"criterion_text":"- Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study"}

Exclusion criteria

• {"criterion_text":"- There are no exclusion criteria for this study."}

Primary endpoints

• {"endpoint_text":"- The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)","definition_or_measurement_approach":"Count of subjects with immune-related adverse events as described (e.g., autoimmune disorders, graft‑versus‑host disease, opportunistic infections, HIV)."}
• {"endpoint_text":"- The number of subjects with new or worsening hematologic disorders","definition_or_measurement_approach":"Count of subjects experiencing new or worsening hematologic disorders."}
• {"endpoint_text":"- The number of subjects with new or worsening neurologic disorders","definition_or_measurement_approach":"Count of subjects experiencing new or worsening neurologic disorders."}
• {"endpoint_text":"- The number of subjects with malignancies","definition_or_measurement_approach":"Count of subjects diagnosed with malignancies."}

Secondary endpoints

• {"endpoint_text":"- Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15","definition_or_measurement_approach":"Proportion of subjects meeting complete resolution of severe VOEs over time through Year 15."}
• {"endpoint_text":"- The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15","definition_or_measurement_approach":"Proportion of subjects with complete resolution of VOEs over time through Year 15."}
• {"endpoint_text":"- Annualized number of severe VOEs over time through Year 15","definition_or_measurement_approach":"Annualized count of severe VOEs per subject over time through Year 15."}
• {"endpoint_text":"- Annualized number of VOEs over time through Year 15","definition_or_measurement_approach":"Annualized count of VOEs per subject over time through Year 15."}
• {"endpoint_text":"- Change from parent study baseline in annualized number of severe VOEs over time through Year 15","definition_or_measurement_approach":"Change from parent study baseline in the annualized number of severe VOEs, measured through Year 15."}
• {"endpoint_text":"- Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb","definition_or_measurement_approach":"Serial hematologic measurements post-infusion through Year 15: total hemoglobin, non-transfused total hemoglobin, %HbS in non-transfused Hb, %HbAT87Q in non-transfused Hb, % non-HbS in non-transfused Hb."}
• {"endpoint_text":"- Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase","definition_or_measurement_approach":"Change from parent study baseline in specified hemolysis laboratory markers through Year 15."}
• {"endpoint_text":"- Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content","definition_or_measurement_approach":"Change from parent study baseline in serum ferritin and liver iron content through Year 15."}

France

Earliest CTIS Part Ii Submission Date

24-04-2024

Latest Decision Or Authorization Date

25-06-2024

Processing Time Days

62

Number Of Sites

1

Number Of Participants

3

Primary sponsor

Full Name

Bluebird Bio Inc.

Organisation Type

Pharmaceutical company

Contract research organisations

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

Study Management

Name

Icon Clinical Research Limited

Name

Icon Laboratory Services Inc.

Name

Icon Development Solutions LLC

Third parties

• {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"Travel vendor","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"Study Management","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"Insertion site analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Edetek Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Voisin Consulting CH SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}