NCT05145062Active, not recruiting
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
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
Source: ClinicalTrials.gov · Last updated 12 Nov 2025
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 8 (actual)
- First submitted
- 22 Nov 2021
- Start date
- 21 Dec 2021
- Primary completion date
- 14 Jul 2038
- Lead sponsor
- Sangamo Therapeutics
- Diseases
- Sickle cell disease, Thalassaemia
- NCT number
- NCT05145062
What you commit to
- Study type: Observational
- Participants enrolled: 8
- From 21 Dec 2021 to the primary completion date, 14 Jul 2038
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study design.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 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.
Who the study is looking for
- 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)
Age in the registry: 18 years to 45 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Healthcare of Atlanta Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Henry Ford Health System Coordinator of this site? Confirm its status | Detroit | United States | Unknown status |
| Karmanos Cancer Institute Coordinator of this site? Confirm its status | Detroit | United States | Unknown status |
| University of Minnesota Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| UCSF Benioff Children s Hospital Coordinator of this site? Confirm its status | Oakland | United States | Unknown status |
| University of California Davis Health System Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.