Clinical trial NCT06363760: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Official title: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Sickle Cell Disease
Transfusion-dependent Beta-Thalassemia
Hemoglobinopathies
Keywords: Sickle Cell Disease
Sickle Cell Anemia
Sickling Disorder Due to Hemoglobin S
Beta-Thalassemia
Thalassemia Major
Thalassemia Intermedia
Hemoglobinopathies
CRISPR-Cas12a
Autologous CD34+
Genetic Diseases, Inborn
Hematologic Diseases
Anemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Lead sponsor: Editas Medicine, Inc.
Sponsor class: INDUSTRY
First posted: 2024-04-12
Enrollment: 54
Sex: ALL
Minimum age (years): 12
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)

Brief summary: The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.

Detailed description: This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.

Eligibility criteria: Inclusion Criteria:

* Participant must have received an EDIT-301 infusion as part of a clinical study.
* Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.

Exclusion Criteria:

* Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.

Primary outcomes:
measure: Adverse events of special interest (AESI)
timeFrame: up to 15 years post EDIT-301 infusion
measure: Adverse events of interest (AEI)
timeFrame: up to 15 years post EDIT-301 infusion
measure: All-cause mortality
timeFrame: up to 15 years post EDIT-301 infusion
measure: Adverse events (AEs) related to EDIT-301
timeFrame: up to 15 years post EDIT-301 infusion
measure: Serious adverse events (SAEs)
timeFrame: up to 15 years post EDIT-301 infusion
Secondary outcomes:
measure: Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion
timeFrame: up to 15 years post EDIT-301 infusion
measure: Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion
timeFrame: up to 15 years post EDIT-301 infusion
measure: Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: Proportion of TDT participants with sustained transfusion independence (TI) over time
timeFrame: up to 15 years post EDIT-301 infusion
measure: Proportion of TDT participants with sustained transfusion reduction (TR) over time
timeFrame: up to 15 years post EDIT-301 infusion
measure: Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time
timeFrame: up to 15 years post EDIT-301 infusion
measure: SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time
timeFrame: up to 15 years post EDIT-301 infusion
measure: SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time
timeFrame: up to 15 years post EDIT-301 infusion
measure: Average HbF (pg) per RBC over time for SCD participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: Complete Blood Count (CBC) red cell indices assay over time for SCD participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: Average HbF (pg) per F-cell over time for SCD participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: HbF and HbS concentration (g/dL) over time for SCD participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.
timeFrame: up to 15 years post EDIT-301 infusion
measure: Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.
timeFrame: up to 15 years post EDIT-301 infusion
measure: HbF concentration (g/dL) over time for TDT participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: Iron overload of TDT participants
timeFrame: up to 15 years post EDIT-301 infusion
measure: Proportion of TDT participants receiving iron chelation therapy over time.
timeFrame: up to 15 years post EDIT-301 infusion
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: New Haven
country: United States
facility: Smilow Cancer Hospital
state: Connecticut
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New York
country: United States
facility: Columbia University Medical Center - Department of Pediatrics
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Charlotte
country: United States
facility: Atrium Health
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers
state: Tennessee
city: Dallas
country: United States
facility: Texas Oncology - Baylor Charles A. Sammons Cancer Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's
state: Texas
city: Ottawa
country: Canada
facility: Ottawa Hospital Research Institute
state: Ontario
city: Toronto
country: Canada
facility: Princess Margaret Cancer Centre-University Health Network
state: Ontario
Study officials:
References: