Clinical Trials

The primary purpose of this dose-ranging study is to assess the safety and tolerability of 3 ascending doses of LX2006 gene therapy for the purpose of selecting the appropriate dose for further clinical development. In addition, assessments of biomarkers and preliminary efficacy are included in this study.

The primary purpose of the LTFU is to assess the long-term safety and tolerability of LX2006 up to 5 years post-treatment. Additionally, efficacy assessments will be evaluated quarterly during Year 2 and annually up to 5 years post-treatment.

The purpose of this study is to investigate the effectiveness and safety of two doses of RGX-314 administered as a single subretinal injection in the study eye of patients with nAMD, and to evaluate mean change from baseline in best-corrected visual acuity (BCVA) of RGX-314 relative to aflibercept at Week 54.

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 ...

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. 

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.

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.

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.

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).

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.

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. 

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.