Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

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

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-08-25

Study Completion Date

2025-01-06

Brief Summary

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This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

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Detailed Description

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CADPT03A12101 was a multicenter, multi-part, first-in-human, proof-of-concept, open label non-randomized, clinical study in Sickle Cell Disease (SCD) subjects. This study included apheresis of mobilized hematopoietic stem and progenitor cells (HSPCs), ex vivo CRISPR/Cas9-mediated genome editing and expansion, followed by myeloablative conditioning and autologous hematopoietic stem cell transplant (HSCT) with follow-up for a minimum of one year and up to two years.

The study was divided into the following parts:

* Part A - Adult subjects were dosed with OTQ923.
* Part B - Assessment of OTQ923 in pediatric patients, however Part B was not opened.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

The is an open-label study.

Study Groups

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OTQ923

Single intravenous infusion of OTQ923 Part A - Adults treated with OTQ923; Part B - Children age 2-17 treated with OTQ923 based on review of data from Part A by Health agency after a formal interim analysis.

Group Type EXPERIMENTAL

OTQ923

Intervention Type BIOLOGICAL

Single intravenous infusion of OTQ923 cell suspension

OTQ923

Intervention Type BIOLOGICAL

Single intravenous infusion of OTQ923, based on review of data from Part A by Health agencies after a formal interim analysis

Interventions

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OTQ923

Single intravenous infusion of OTQ923 cell suspension

Intervention Type BIOLOGICAL

OTQ923

Single intravenous infusion of OTQ923, based on review of data from Part A by Health agencies after a formal interim analysis

Intervention Type BIOLOGICAL

Other Intervention Names

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Adult Part A Children 2-17 years old - Part B

Eligibility Criteria

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Inclusion Criteria

1. Male or female subjects age 2-40 years inclusive
2. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others)
3. Performance status \>70% (Karnofsky for subjects \>16 years of age and Lansky for subjects \<16 years of age)
4. At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization
5. Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

Exclusion Criteria

1. Available matched related donor for HSCT
2. Clinically significant active infection
3. Seropositive for HIV or HTLV
4. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency
5. Prior HSCT or gene therapy
6. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
7. Protocol defined iron overload
8. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya
9. Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya

Minimum Eligible Age

2 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No