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NCT05145062 | Enrolling by invitation | Blood and Lymphatic Diseases

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

Sangamo Therapeutics

Brief 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

Condition or disease

Blood and Lymphatic Diseases

Intervention/treatment

BIVV003

ST-400

Detailed Description:

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

Study Type : Observational
Estimated Enrollment : 12 participants
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
Actual Study Start Date : December 21, 2021
Estimated Primary Completion Date : August 10, 2037
Estimated Study Completion Date : August 10, 2037

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years to 45 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria
  • 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)
Exclusion Criteria
  • 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.
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Location Details

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

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, California

UCSF Benioff Children's Hospital

Oakland, California, United States, 94609

Not yet recruiting

United States, Georgia

Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30329

Not yet recruiting

United States, Massachusetts

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

Not yet recruiting

United States, road cancer

Karmanos Cancer Institute

Detroit, road cancer, United States, 48201

Not yet recruiting

United States, road cancer

Henry Ford Health System

Detroit, road cancer, United States, 48202

Not yet recruiting

United States, Minnesota

University of Minnesota

Minneapolis, Minnesota, United States, 55455