ltf 303 nct02633943 results

Summary

This study is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. 1 The study aims to assess the long-term safety and efficacy of the drug product in TDT subjects. 1

According to

1 clinicaltrials.gov

Summaries from the best pages on the web

Summary Brief Summary: This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.

Longterm Follow-up of Subjects With Transfusion-Dependent β-Thalassemia ...

clinicaltrials.gov

After the 2-year study period, the patients could participate in a long-term follow-up study ( LTF - 303 ; ClinicalTrials.gov number, NCT02633943 ) for 13 additional years.

Betibeglogene Autotemcel Gene Therapy for Non–β0

nejm.org

After the 24-month primary study period, patients in the two studies were asked to transition to a long-term follow-up study (ClinicalTrials.gov number, NCT02633943 ) for 13 years of additional...

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

nejm.org

HGB-205 is a phase 1/2, open-label, single-arm, non-randomized interventional study of 2-year duration at a single center, followed by observation in long-term follow-up studies LTF-303 ( NCT02633943) and LTF-307 (…

Long-term outcomes of lentiviral gene therapy for the β ...

nih.gov

Transduction in the phase 3 studies used a refined manufacturing process compared to the phase 1/2 studies. LTF-303 assessments include Hb, peripheral blood vector copy number (PB VCN), assessment of…

Author notes - American Society of Hematology

ashpublications.org

( a) Controls showing that the Pacific Blue (PB) labeled anti-HbA monoclonal antibody (Rockland) does not cross react with HbS in individualized RBCs by flow cytometry analysis. S/S RBC exhibit…

Long-term outcomes of lentiviral gene therapy for the β ... - Nature

nature.com

After gene therapy, the median follow-up was 49.1 months (range, 23.3-71.8 months). The following article features coverage from the American Society of Hematology 2020 meeting. Click here to read more…

Long-Term Efficacy and Safety Demonstrated With Betibeglogene ...

hematologyadvisor.com

After 2 y of follow-up, pts could enroll in a long-term follow-up study ( LTF-303 [NCT02633943 ]) for up to an additional 13 y. We report results from LTF - 303 in pts with…

Abstract - American Society of Hematology

ashpublications.org

History of Changes for Study: NCT02633943 History of Changes for Study: NCT02633943 Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Latest version (submitted February 4, 2022)…

History of Changes for Study: NCT02633943 - ClinicalTrials.gov

clinicaltrials.gov

This research study is evaluating the long-term effects of an investigational gene transfer approach to treat beta thalassemia major, also referred to as transfusion-dependent β–thalassemia, and severe sickle cell disease…

LTF-303 - Health Research Authority

hra.nhs.uk

Bluebird also is conducting a global, long-term safety and effectiveness follow-up study, called LTF-303 ( NCT02633943 ), for participants in LentiGlobin clinical studies in SCD and beta-thalassemia. More information is…

Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial

sicklecellanemianews.com