Clinical trial NCT05145062: Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Blood and Lymphatic Diseases
Keywords: Sickle Cell Disease (SCD), Beta-thalassemia (TDT)
Lead sponsor: Sangamo Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-12-06
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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

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

Eligibility 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.

Primary outcomes:
measure: Adverse Events
timeFrame: Up to 15 years
measure: Overall Survival
timeFrame: Up to 15 years
Secondary outcomes:
measure: Change in hemoglobin levels
timeFrame: Up to 15 years
measure: Change in hemolysis markers
timeFrame: Up to 15 years
measure: Frequency of severe vaso-occlusive crises
timeFrame: Up to 15 years
measure: Frequency and severity of SCD-related clinical events
timeFrame: Up to 15 years
measure: Red blood cell transfusions
timeFrame: Up to 15 years
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Sacramento
country: United States
facility: University of California Davis Health System
state: California
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
Study officials:
affiliation: Sangamo Therapeutics
name: Medical Monitor
role: STUDY_DIRECTOR
References: