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 to Evaluate PR006 in Patients with Frontotemporal Dementia with Progranulin Mutations (FTD-GRN)
The purpose of this study is to evaluate the safety and effect of intra-cisternal PR006 administration on progranulin protein (PGRN) levels in patients with frontotemporal dementia with progranulin mutations (FTD-GRN). Three escalating dose (low dose, medium dose and high dose) cohorts are planned.
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 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.
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.