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Gene Therapy Trial Report

Summary

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A


NCTID NCT05265767 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name CD68-ET3-LV
Compound Description Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
Sponsor Christian Medical College, Vellore, India
Funder Type Other
Recruitment Status
Completed
Enrollment Count 6 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant F8
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Dose 1 5.0-6.1E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1
Submit Date 2022-02-16
Completion Date 2024-06-28
Last Update 2024-07-12

Participation Criteria


Eligible Age 18 Years - 45 Years
Standard Ages Adult
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: * Able to provide informed consent for the protocol approved by the Institutional Review Board. * Male subjects who are ≥18 years of age and \< 45 years of age. * Diagnosis of severe hemophilia A (\<1 IU/dl factor VIII activity). * Documented history of more than 100 exposures of factor VIII treatment. * Average of at least 3 bleeds requiring treatment per year over the prior three years, at least 3 bleeds per year during the 3 years preceding the initiation of prophylaxis, or evidence of joint damage (knee, elbow or ankle) on physical or radiographic examination thought to be related to hemophilia. * Performance status (Karnofsky score) of at least 70. * Willing and able to comply with the requirements of the protocol. Exclusion Criteria: * History of spontaneous central nervous system bleeding within the last 5 years. * Significant organ dysfunction which could interfere with outcome of therapy such as: - * Cardiac: There should be no evidence of significant cardiac dysfunction (resting left ventricular ejection fraction of \< 50%) and no cardiomegaly. There should not be uncontrollable hypertension. * Renal: Glomerular Filtration Rate (GFR) \< 60 ml/min/1.73m2 as calculated using the Cockcroft-Gault equation. * Hepatic: There should be no evidence of hepatic dysfunction which is defined as a serum bilirubin of \> 1.5 mg/dl and Aspartate Amino Transferase (AST) / Alanine Amino Transferase (ALT) \> 3X the upper limit of normal, * Hematologic: Absolute neutrophil counts (ANC) \< 1000/mm3 and platelets counts \< 150,000/μL. * Pulmonary function with a corrected Diffusing Capacity of lung for Carbon Monoxide (DLCO) of \< 50% predicted * History of a FVIII inhibitor (\>0.6 Bethesda Units/ml) including at least 2 measurements over the preceding 5 years or any single titer \>5 Bethesda Units (BU) /ml. * Previous stem cell transplant. * HIV positive. * Evidence of hepatitis B active infection or chronic carrier * Evidence of chronic hepatitis C infection. Absence of chronic infection will be documented with at least 2 negative viral loads at least 6 months apart. * Diagnosis of a bleeding disorder other than hemophilia A * Use of medication(s) that can affect hemostasis (e.g. aspirin and non- cyclooxygenase (COX-2) selective non-steroid anti-inflammatory drugs). * History of cancer or familial cancer syndromes * Any condition in the opinion of the principle investigator that will negatively impact the subject's ability to safely undergo an autologous stem cell transplant. * Any reason in the opinion of the principle investigator that will negatively impact the subject's ability to complete the clinical trial per the trial protocol.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations India

Regulatory Information


Has US IND True
FDA Designations
Recent Updates This vector belongs to Expression Therapeutics, LLC who is the sponsor of the US-based clinical trial (NCT04418414)

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