A Phase 1 interventional study of Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 in Anemia, Sickle Cell, sponsored by CSL Behring. Terminated at 1 site in United States. Open to participants aged 18 Years to 45 Years. Per ClinicalTrials.gov, last updated 2021-06-18.
Sponsored by CSL Behring · Phase 1, Interventional, and Treatment
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.
1,104 studies on the registry are indexed under Anemia, Sickle Cell; 236 are open to participants now.
This study's enrollment of 1 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →CSL Behring is the lead sponsor of 142 studies on the registry; 18 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Severe sickle cell disease symptomatology, defined as any one or more of the following:
Exclusion Criteria:
Inadequate bone marrow function, defined as at least 1 of the following:
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
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
Also known as: CSL200
Number of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs) associated with the administration of CSL200
Adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, or is medically significant. Adverse event of special interest (AESI) is defined in this study as any of the following: acute immune reactions, autoimmunity to CSL200; malignancy; predominant integration site in presence of malignancy or other abnormality.
Time frame: Up to 48 weeks
Number of subjects experiencing AEs, SAEs, and AESIs associated with the administration of CSL200
Time frame: Up to 48 weeks
Number of AEs, SAEs, and AESIs associated with the collection of CD34+ HSPCs by apheresis after mobilization with plerixafor
Time frame: Up to 6 weeks
Number of subjects experiencing AEs, SAEs, and AESIs associated with the collection of CD34+ HSPCs by apheresis after mobilization with plerixafor
Time frame: Up to 6 weeks
Number of AEs, SAEs, and AESIs associated with reduced intensity conditioning with melphalan
Time frame: Up to 3 weeks
Number of subjects experiencing AEs, SAEs, and AESIs associated with reduced intensity conditioning with melphalan
Time frame: Up to 3 weeks
Total by-subject number of CD34+ HSPCs collected in total and in each apheresis session
Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by CD34+ HSPCs collected
Time frame: Up to 2 days
Number of subjects receiving plerixafor and number of plerixafor doses administered by subject
Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by plerixafor administrations
Time frame: Up to 2 days
Number of subjects undergoing apheresis and number of apheresis sessions by subject
Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by apheresis sessions
Time frame: Up to 2 days
The number of subjects undergoing reduced intensity conditioning with melphalan and able to receive CSL200
Reduced intensity conditioning assessed by subjects receiving melphalan
Time frame: 2 days
Number of subjects receiving CSL200
Time frame: 1 day
By-subject number of separate CSL200 drug products administered
Time frame: 1 day
Number of CSL200 CD34+ HSPCs/kg administered by subject and by CSL200 drug product
Time frame: 1 day
By-subject total number and percentage of CD34+ HSPCs transduced with CAL-H
Time frame: Up to 48 weeks
Vector copy number (VCN)
VCN will be determined by using the average number of CAL-H vector genomes per cell
Time frame: Up to 48 weeks
Plan to share: Yes — CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
Supporting information: Study protocol, Sap
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This study is terminated, as verified in Jun 2021. You cannot join it, but the record below documents what was studied.
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