Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2019-004266-18 | EudraCT Number | ||
| 2024-513901-30-00 | EU Trial (CTIS) Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Subjects with sickle-cell disease | Subjects treated with ex vivo gene therapy drug product for sickle cell disease in a bluebird bio-sponsored study who agree to participate in this long-term follow-up study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Safety and efficacy assessments | Other | Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) | Through 15 years post-drug product infusion | |
| Number of subjects with new or worsening hematologic disorders | Through 15 years post-drug product infusion | |
| Number of subjects with new or worsening neurologic disorders | Through 15 years post-drug product infusion | |
| Number of subjects with malignancies | Through 15 years post-drug product infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 | Through 15 years post-drug product infusion | |
| Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 | Through 15 years post-drug product infusion |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Subjects with sickle cell disease treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Anjulika Chawla, MD | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35294 | United States | ||
| UCSF Benioff Children's Hospital Oakland |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35075288 | Derived | Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24. |
| Label | URL |
|---|---|
| HGB-205 Clinicaltrials.gov | View source |
Not provided
Bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted) following study completion and once all applicable regulatory submissions based on this study have been performed. Sharing of individual patient data will be done according to criteria established by bluebird bio and/or industry best practices to protect confidential information and maintain the privacy of study participants. For inquiries, please contact us at datasharing@bluebirdbio.com.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Annualized number of severe VOEs over time through Year 15 | Through 15 years post-drug product infusion |
| Annualized number of VOEs over time through Year 15 | Through 15 years post-drug product infusion |
| Change from parent study baseline in annualized number of severe VOEs over time through Year 15 | Through 15 years post-drug product infusion |
| Assessment of total Hb over time post-drug product infusion through Year 15 | Through 15 years post-drug product infusion |
| Assessment of non-transfused total Hb over time post-drug product infusion through Year 15 | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q | Through 15 years post-drug product infusion |
| Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q | Through 15 years post-drug product infusion |
| Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15 | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q | Through 15 years post-drug product infusion |
| Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q. Non-HbS is the total g/dL of HbF + HbA2 + HbAT87Q | Through 15 years post-drug product infusion |
| Change from parent study baseline through Year 15 in hemolysis markers | Change from parent study baseline through Year 15 in absolute reticulocyte count, percent reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase hemolysis markers | Through 15 years post-drug product infusion |
| Change from parent study baseline through Year 15 in markers of iron stores | Change from parent study baseline through Year 15 in serum ferritin and liver iron content markers of iron stores | 15 years post-drug product infusion |
| Oakland |
| California |
| 94609 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2991 | United States |
| Warren Grant Magnuson Clinical Center | Bethesda | Maryland | 20892 | United States |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55454 | United States |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | United States |
| Cohen Children's Medical Center | New Hyde Park | New York | 11040 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| The University of North Carolina | Chapel Hill | North Carolina | 27514 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Hospital Necker | Paris | 75015 | France |
| HGB-206 Clinicaltrials.gov | View source |
| HGB-210 Clinicaltrials.gov | View source |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D012449 | Safety |
| ID | Term |
|---|---|
| D000056 | Accident Prevention |
| D000059 | Accidents |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
Not provided
Not provided