This study is accepting new patients by invitation only

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

a study on Blood and Lymphatic Diseases Beta Thalassemia Sickle Cell Anemia Bone Marrow Transplant Hematopoietic Cell Transplantation Stem Cell Transplant Transplants Anemia

Summary

Eligibility
for people ages 18-45 (full criteria)
Location
at UC Davis UCSF
Dates
study started
completion around

Description

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 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

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

Details

The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.

Keywords

Blood and Lymphatic Diseases, Sickle Cell Disease (SCD), Beta-thalassemia (TDT), Sickle Cell Anemia, Thalassemia, beta-Thalassemia, Lymphatic Diseases, BIVV003

Eligibility

You can join if…

Open to people ages 18-45

  • 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)

You CAN'T join if...

  • 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.

Locations

  • UCSF Benioff Children's Hospital
    Oakland California 94609 United States
  • University of California Davis Health System
    Sacramento California 95817 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Sangamo Therapeutics
ID
NCT05145062
Study Type
Observational
Participants
Expecting 12 study participants
Last Updated