EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-29

Study Completion Date

2025-12-31

Brief Summary

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The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

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Detailed Description

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This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive

Conditions

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Transfusion Dependent Beta Thalassemia Hemoglobinopathies Thalassemia Major Thalassemia Intermedia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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EDIT-301

EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

Group Type EXPERIMENTAL

EDIT-301

Intervention Type GENETIC

Administered by intravenous infusion after myeloablative conditioning with busulfan.

Interventions

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EDIT-301

Administered by intravenous infusion after myeloablative conditioning with busulfan.

Intervention Type GENETIC

Other Intervention Names

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renizgamglogene autogedtemcel reni-cel

Eligibility Criteria

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Inclusion Criteria

Diagnosis of Transfusion Dependent B-Thalassemia as defined by:

* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and
* History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent
* Clinically stable and eligible to undergo autologous HSCT
* Karnofsky Performance Status ≥ 70

Exclusion Criteria

* Available 10/10 human leukocyte antigen (HLA)-matched related donor
* Prior HSCT or contraindications to autologous HSCT
* Participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records
* Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records
* Prior receipt of gene therapy
* Inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/L or a platelet count \< 100 x 10\^9/L (without hypersplenism), per investigator judgement
* Inadequate organ function
* Advanced liver disease
* Any prior or current malignancy, or immunodeficiency disorder,
* Immediate family member with a known or suspected Familial Cancer Syndrome
* Clinically significant and active bacterial, viral, fungal, or parasitic infection

Minimum Eligible Age

18 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No