Blood and Lymphatic DiseasesNCT05145062
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
An observational study of Blood and Lymphatic Diseases, run by Sangamo Therapeutics at 7 sites in United States.
Trial summary
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the biological treatment effect of BIVV003 in SCD Long-term efficacy of the clinical treatment… Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Blood and Lymphatic Diseases
- What is being tested?
- BIVV003 and ST-400
- Type of treatment
- Injection or infusion
- How many people
- 8 participants
- Where
- 7 sitesUnited States
- Study dates
- 21 Dec 2021to 14 Jul 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 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Study population: All participants who received BIVV003 in one of the primary parent or future studies or ST-400 in the ST-400-01 parent study and agree to participate.
Who may be able to join
- Received treatment with BIVV003 or ST-400 in one of the parent studies (ACT16222, ST- 400-01) or any future studies with BIVV003
- Capable of giving signed informed consent (and if applicable assent)
Who cannot join
- Unable to comply with study visit schedule or study procedures
- Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the participant unsuitable for participation in the study The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Boston Children's Hospital
Boston, Massachusetts, 02115
Karmanos Cancer Institute
Detroit, Michigan, 48201
Henry Ford Health System
Detroit, Michigan, 48202
University of Minnesota
Minneapolis, Minnesota, 55455
UCSF Benioff Children's Hospital
Oakland, California, 94609
University of California Davis Health System
Sacramento, California, 95817
About this study
Official title: An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Participants With Severe Sickle Cell Disease (SCD) or With ST-400 in Participants With Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant
Primary Objectives:
Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT)
Secondary Objectives:
- Long-term efficacy of the biological treatment effect of BIVV003 in SCD
- Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events
- Long-term efficacy of the biological treatment effect of ST-400 in TDT
- Long-term efficacy of the clinical treatment effect of ST-400 in TDT
Read the detailed description
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
- Conditions
- Blood and Lymphatic Diseases
- Being tested
- BIVV003, ST-400
- Keywords
- Sickle Cell Disease (SCD), Beta-thalassemia (TDT)
- Medical subject headings
- Lymphatic Diseases, Anemia, Sickle Cell, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BIVV003Drug
Also called SAR445136
Solution for intravenous administration
ST-400Drug
Solution for intravenous administration
| Study group | Role | What they receive |
|---|---|---|
| BIVV003 Cohort | All participants treated in parent and future studies with BIVV003 Receives: Drug: BIVV003 | |
| ST-400 Cohort | All participants treated in parent studies with ST-400 Receives: Drug: ST-400 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of participants with serious adverse events and adverse events related to BIVV003 or ST-400, including new malignancy, new incidence or exacerbation of neurologic or rheumatologic or autoimmune or hematologic disorder, or new incidence of infection potentially related to BIVV003 or ST-400
Measured: Up to 15 years
Overall Survival
Duration from first dose of study medication to death
Measured: Up to 15 years
Secondary outcomes (5)
Change in hemoglobin levels
Long-term change in levels of hemoglobin F, hemoglobin S and total hemoglobin (BIVV003 cohort), long term change in levels of hemoglobin F and total hemoglobin (ST-400 cohort)
Measured: Up to 15 years
Change in hemolysis markers
Long-term change in markers of hemolysis, including reticulocyte count, lactate dehydrogenase, haptoglobin, and serum bilirubin, over time in the BIVV003 cohort
Measured: Up to 15 years
Frequency of severe vaso-occlusive crises
Percentage of participants with severe vaso-occlusive crises, including acute pain crisis, acute chest syndrome, priapism, and splenic sequestration, in the BIVV003 cohort
Measured: Up to 15 years
Frequency and severity of SCD-related clinical events
Percentage of participants with SCD-related clinical events (e.g., acute renal failure, acute stroke) in the BIVV003 cohort
Measured: Up to 15 years
Red blood cell transfusions
Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts
Measured: Up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2021
- Study start21 Dec 2021
- Primary completion14 Jul 2038 (estimated)
- Study completion14 Jul 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Blood and Lymphatic DiseasesStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Sangamo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05145062
- Sponsor's protocol ID
- LTS16653
- Status last verified
- Nov 2025
- First submitted
- 22 Nov 2021
- Last updated
- 12 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Participants With Severe Sickle Cell Disease (SCD) or With ST-400 in Participants With Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant
- ClinicalTrials.gov ID
- NCT05145062
- Acronym / sponsor ID
- LTS16653
- Conditions
- Blood and Lymphatic Diseases
- Keywords
- Sickle Cell Disease (SCD), Beta-thalassemia (TDT)
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 21 Dec 2021 (actual)
- Primary completion
- 14 Jul 2038 (estimated)
- Study completion
- 14 Jul 2038 (estimated)
- First posted
- 6 Dec 2021 (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.
Primary Objectives:
Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT)
Secondary Objectives:
- Long-term efficacy of the biological treatment effect of BIVV003 in SCD
- Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events
- Long-term efficacy of the biological treatment effect of ST-400 in TDT
- Long-term efficacy of the clinical treatment effect of ST-400 in TDT
Detailed description
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BIVV003 Cohort | – | Drug: BIVV003All participants treated in parent and future studies with BIVV003 |
| ST-400 Cohort | – | Drug: ST-400All participants treated in parent studies with ST-400 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- All participants who received BIVV003 in one of the primary parent or future studies or ST-400 in the ST-400-01 parent study and agree to participate.
Key inclusion criteria (2 listed)
- Received treatment with BIVV003 or ST-400 in one of the parent studies (ACT16222, ST- 400-01) or any future studies with BIVV003
- Capable of giving signed informed consent (and if applicable assent)
Key exclusion criteria (2 listed)
- Unable to comply with study visit schedule or study procedures
- Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the participant unsuitable for participation in the study The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
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 |
|---|---|---|
| BIVV003Also: SAR445136Solution for intravenous administration | Drug | BIVV003 Cohort |
| ST-400Solution for intravenous administration | Drug | ST-400 Cohort |
Primary outcome
Adverse Events
Time frame: Up to 15 years
Number of participants with serious adverse events and adverse events related to BIVV003 or ST-400, including new malignancy, new incidence or exacerbation of neurologic or rheumatologic or autoimmune or hematologic disorder, or new incidence of infection potentially related to BIVV003 or ST-400
Overall Survival
Time frame: Up to 15 years
Duration from first dose of study medication to death
5 secondary outcomes
Change in hemoglobin levels
Time frame: Up to 15 years
Change in hemolysis markers
Time frame: Up to 15 years
Frequency of severe vaso-occlusive crises
Time frame: Up to 15 years
Frequency and severity of SCD-related clinical events
Time frame: Up to 15 years
Red blood cell transfusions
Time frame: Up to 15 years
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 12 Nov 2025.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Medical Monitor study director
Sangamo Therapeutics
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Blood and Lymphatic Diseases, run by Sangamo Therapeutics at 7 sites in United States.
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 2 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: under way, not looking for participants, as of the sponsor's last update on 12 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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?
Adverse Events (time frame: Up to 15 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Nov 2025.