A Study to Evaluate Gene Therapy to Treat Neovascular Age-related Macular Degeneration
The primary objective of this study is to evaluate mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to ranibizumab at Week 38.
Second objectives of this study are to evaluate the safety and tolerability of RGX-314 through Week 102, to evaluate the effect of RGX-314 relative to ranibizumab on BCVA, to evaluate the effect of RGX-314 relative to ranibizumab on central retinal thickness (CRT) as measured by spectral domain-ocular coherence tomography (SD-OCT), to evaluate the effect of RGX-314 relative to ranibizumab on center point thickness (CPT) as measured by SD-OCT, to assess the need for supplemental anti-vascular endothelial growth factor ...
Attitudes and Beliefs About Gene Transfer Therapy in Adults with Cocaine Use Disorder
The purpose of this study is to investigate the facilitators and barriers of adults with a history of cocaine use disorder (CocUD) in sustained remission to participating in a Phase I gene transfer study aimed at reducing the rate of cocaine relapse.
Gene Therapy and Radioactive Iodine in Treating Patients with Locally Recurrent Prostate Cancer that Did Not Respond to External-Beam Radiation Therapy
RATIONALE: Radioactive drugs, such as radioactive iodine, may carry radiation directly to tumor cells and not harm normal cells. Placing a gene called Ad5CMV-NIS in prostate cancer cells may help the prostate cells take in more radioactive iodine and thus kill the cancer cells. Drugs, such as liothyronine sodium, may protect the thyroid from the side effects of radioactive iodine.
PURPOSE: This phase I trial is studying the side effects and best dose of gene therapy given together with radioactive iodine in treating patients with locally recurrent prostate cancer that did not respond to external-beam radiation therapy.
A pivotal study of derazantinib in patients with inoperable or advanced intrahepatic cholangiocarcinoma and FGFR2 gene fusions or FGFR2 gene mutations or amplifications
Scottsdale/Phoenix, AZ; Jacksonville, FL; Rochester, MN
This pivotal, open-label, single-arm study will evaluate the anti-cancer activity of ARQ 087 by Objective Response Rate (ORR) by central radiology review as per RECIST v1.1 in subjects with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) whose tumors harbor FGFR2 gene fusions (by FISH performed by the central laboratory) and who received at least one prior regimen of systemic therapy. Subjects will be dosed orally once per day at 300 mg of ARQ 087 capsules.
A Study to Assess Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (The EXACT Trial)
The primary purpose of this trial is to determine the safety of XC001 (AdVEGFXC1) in patients who suffer from angina caused by coronary artery disease and have no other treatment options. Subjects in this study will receive one of four intramyocardial doses of XC001 that expresses human vascular endothelial growth factor (VEGF) which induces therapeutic angiogenesis (revascularization).
A Study of the Safety of Joint Injected Sc-rAAV2.5IL-1Ra (a Virus Carried Gene Therapy) in Patients with Moderate Knee Osteoarthritis
The purpose of this study is to determine the safety of sc-rAAV2.5IL-1Ra, a gene therapy carried by a virus and delivered by injection into the knee joint of patients with moderate osteoarthritis of the knee.
A Study to Evaluate the Safety and Effectiveness of Idecabtagene Vicleucel to Treat Multiple Myeloma
Jacksonville, FL; Rochester, MN
The purpose of this study is to evaluate the safety and effectiveness of nonconforming idecabtagene vicleucel (ide-cel) in subjects with multiple myeloma per the approved prescribing information.
Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells
Rochester, MN; Scottsdale/Phoenix, AZ; Jacksonville, FL
This is a prospective study for the long-term follow-up (LTFU) of safety and efficacy for all pediatric and adult subjects exposed to Gene-modified (GM) T cell therapy participating in a previous Celgene sponsored or Celgene alliance partner sponsored study. Subjects who received at least one GM T cell infusion, will be asked to roll-over to this LTFU protocol upon either premature discontinuation from, or completion of the prior parent treatment protocol.
A Study to Evaluate ADP-A2M4 SPEAR™ T Cells in Subjects with Advanced Synovial Sarcoma or Myxoid/Round Cell Liposarcoma
The purpose of this study is to evaluate genetically-engineered ADP-A2M4 in HLA-A*02 subjects with metastatic or inoperable (advanced) Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (MRCLS) who have received prior chemotherapy and whose tumor expresses the MAGE-A4 tumor antigen.