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CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2021-06-18

No results posted yet for this study

Summary

This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.

Conditions

  • Anemia, Sickle Cell

Interventions

BIOLOGICAL

Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734

* Cryopreserved formulated autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 in a bag for infusion * Plerixafor to mobilize hematopoietic stem cells prior to each apheresis * Single dose melphalan before administration of CSL200

Sponsors & Collaborators

  • CSL Behring

    lead INDUSTRY

Principal Investigators

  • Study Director · CSL Behring

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-02
Primary Completion
2021-05-05
Completion
2021-05-05
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04091737 on ClinicalTrials.gov