Protocol Details
Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
This study is NOT currently recruiting participants.
Summary
Number | 20-H-0141 |
Sponsoring Institute | National Heart, Lung and Blood Institute (NHLBI) |
Recruitment Detail | Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 18 Years
Max Age: 99 Years |
Referral Letter Required | Yes |
Population Exclusion(s) | Adults who are or may become unable to consent;
Children |
Keywords | Longterm Follow-up;
Natural History |
Recruitment Keyword(s) | None |
Condition(s) | Sickle Cell Disease |
Investigational Drug(s) | bb1111
|
Investigational Device(s) | None |
Intervention(s) | None |
Supporting Site | National Heart, Lung, and Blood Institute |
Background:
In a previous study, people with sickle cell disease (SCD) were given LentiGlobin BB305. It is a gene transfer treatment. Researchers hoped that it could reduce or stop the symptoms of SCD. Researchers want to maintain long-term contact with the people from the study to follow their progress.
Objective:
To monitor the long-term effects of the LentiGlobin(TM) BB305 treatment.
Eligibility:
People with SCD who took part in the bluebird bio, Inc. protocol HGB-206, where LentiGlobin(TM) BB305 was used to treat SCD.
Design:
Participants will have a medical history and medicine review.
Participants will have periodic visits to NIH for 13 years.
Participants will have a physical exam. They will give blood and urine samples. They will be asked about their pain.
Participants will have magnetic resonance imaging (MRI) or magnetic resonance angiogram (MRA) of the brain. Those who are not able to have an MRI/MRA will have a CT scan. Participants age 16 and younger will have transcranial Doppler. These tests monitor and take pictures of the brain without going inside the body.
Participants will have an echocardiogram. It uses ultrasound to see the heart.
Participants will have an MRI or other image taken of their liver. It will be used to measure the level of iron in the liver and see if the iron has caused any damage to the liver.
Participants will complete surveys about how their SCD affects their quality of life, their cognitive function, and how they are feeling.
Participants may have a bone marrow biopsy.
Eligibility
INCLUSION CRITERIA:
Subjects meeting the following criteria are eligible for study participation:
1. Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)
2. Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study
EXCLUSION CRITERIA:
There are no exclusion criteria for this study.
Citations:
Hoban MD, Orkin SH, Bauer DE. Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease. Blood. 2016 Feb 18;127(7):839-48. doi: 10.1182/blood-2015-09-618587. Epub 2016 Jan 12. PMID: 26758916; PMCID: PMC4760089. de Montalembert M, Ribeil JA, Brousse V, Guerci-Bresler A, Stamatoullas A, Vannier JP, Dumesnil C, Lahary A, Touati M, Bouabdallah K, Cavazzana M, Chauzit E, Baptiste A, Lefebvre T, Puy H, Elie C, Karim Z, Ernst O, Rose C. Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome. PLoS One. 2017 Mar 3;12(3):e0172147. doi: 10.1371/journal.pone.0172147. PMID: 28257476; PMCID: PMC5336214. Madigan C, Malik P. Pathophysiology and therapy for haemoglobinopathies. Part I: sickle cell disease. Expert Rev Mol Med. 2006 Apr 28;8(9):1-23. doi: 10.1017/S1462399406010659. PMID: 16690007.
Contacts:
Clinical Trials Number:
NCT02140554