Sickle Cell DiseaseNCT04628585
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
An observational study of Sickle Cell Disease, run by Genetix Biotherapeutics Inc. at 16 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 53 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Safety and efficacy assessments
- How many people
- 85 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 21 Oct 2020to Jan 2038
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 53 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subjects with sickle cell disease treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
Who may be able to join
- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s)
- Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study
Who cannot join
- There are no exclusion criteria for this study
Trial locations and contacts
16 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
16 sites in 2 countries
United States15 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, 20892
University of Alabama
Birmingham, Alabama, 35294
The University of North Carolina
Chapel Hill, North Carolina, 27514
Medical University of South Carolina
Charleston, South Carolina, 29425
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2991
Duke University Medical Center
Durham, North Carolina, 27705
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Baylor College of Medicine
Houston, Texas, 77030
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Cohen Children's Medical Center
New Hyde Park, New York, 11040
Columbia University Medical Center
New York, New York, 10032
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Virginia Commonwealth University
Richmond, Virginia, 23298
About this study
Official title: 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 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Safety and efficacy assessments
- Keywords
- Hematopoietic Stem Cells
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy assessmentsOther
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Other: Safety and efficacy assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
Measured: Through 15 years post-drug product infusion
Number of subjects with new or worsening hematologic disorders
Measured: Through 15 years post-drug product infusion
Number of subjects with new or worsening neurologic disorders
Measured: Through 15 years post-drug product infusion
Number of subjects with malignancies
Measured: Through 15 years post-drug product infusion
Secondary outcomes (12)
Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
Measured: Through 15 years post-drug product infusion
Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
Measured: Through 15 years post-drug product infusion
Annualized number of severe VOEs over time through Year 15
Measured: Through 15 years post-drug product infusion
Annualized number of VOEs over time through Year 15
Measured: Through 15 years post-drug product infusion
Change from parent study baseline in annualized number of severe VOEs over time through Year 15
Measured: Through 15 years post-drug product infusion
Assessment of total Hb over time post-drug product infusion through Year 15
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 15 years post-drug product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2020
- Study start21 Oct 2020
- Primary completionJan 2038 (estimated)
- Study completionJan 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anjulika Chawla, MD, Study director, bluebird bio, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04628585
- Sponsor's protocol ID
- LTF-307
- Other IDs
- 2019-004266-18, 2024-513901-30-00, 2019
- Status last verified
- Mar 2025
- First submitted
- 5 Nov 2020
- Last updated
- 20 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Publications
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.
PubMed 35075288
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04628585
- Acronym / sponsor ID
- LTF-307
- Conditions
- Sickle Cell Disease
- Keywords
- Hematopoietic Stem Cells
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 85 participants (estimated)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Oct 2020 (actual)
- Primary completion
- Jan 2038 (estimated)
- Study completion
- Jan 2038 (estimated)
- First posted
- 13 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects with sickle-cell disease | – | Other: Safety and efficacy assessmentsSubjects 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 |
Who may qualify
- Ages
- 2 to 53 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects with sickle cell disease treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
Key inclusion criteria (2 listed)
- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s)
- Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Safety and efficacy assessmentsSafety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant | Other | Subjects with sickle-cell disease |
Primary outcome
Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
Time frame: Through 15 years post-drug product infusion
Number of subjects with new or worsening hematologic disorders
Time frame: Through 15 years post-drug product infusion
Number of subjects with new or worsening neurologic disorders
Time frame: Through 15 years post-drug product infusion
Number of subjects with malignancies
Time frame: Through 15 years post-drug product infusion
12 secondary outcomes
Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
Time frame: Through 15 years post-drug product infusion
Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
Time frame: Through 15 years post-drug product infusion
Annualized number of severe VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Annualized number of VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline in annualized number of severe VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Assessment of total Hb over time post-drug product infusion through Year 15
Time frame: Through 15 years post-drug product infusion
Assessment of non-transfused total Hb over time post-drug product infusion through Year 15
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline through Year 15 in hemolysis markers
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline through Year 15 in markers of iron stores
Time frame: 15 years post-drug product infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2025.
- Derived from this study: PubMed 35075288Magrin 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.
- HGB-205 Clinicaltrials.govLink provided by the sponsor.
- HGB-206 Clinicaltrials.govLink provided by the sponsor.
- HGB-210 Clinicaltrials.govLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Anjulika Chawla, MD study director
bluebird bio, Inc.
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Genetix Biotherapeutics Inc. at 16 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 20 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) (time frame: Through 15 years post-drug product infusion). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04628585, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2025.