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

N/AActive Not RecruitingNCT05145062

Sangamo Therapeutics8 enrolled

Overview

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

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

Blood and Lymphatic Diseases

Interventions

BIVV003DRUG

Solution for intravenous administration

Eligibility

Sex: ALLMin age: 18 YearsMax age: 45 Years

Medical Language ↔ Plain English

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.

Locations (7)

UCSF Benioff Children's Hospital

Oakland, California, United States

University of California Davis Health System

Sacramento, California, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Karmanos Cancer Institute

Detroit, Michigan, United States

Henry Ford Health System

Detroit, Michigan, United States

University of Minnesota

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

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

Time frame: Up to 15 years

Overall Survival

Duration from first dose of study medication to death

Time frame: Up to 15 years

Secondary Outcomes

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)

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: Up to 15 years

Red blood cell transfusions

Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts

Time frame: Up to 15 years