Glashã¼tten, Germany

Pemigatinib After Curative Local Therapy in Advanced iCCA With FGFR2 Fusion/Rearrangements

This is a prospective, exploratory, single-arm, non-randomized, open-label phase II study to investigate whether pemigatinib is clinically efficacious after curative local treatment including surgery/ SBRT or ablation in iCCA patients with FGFR2 fusion/rearrangements. Patients will receive pemigatinib 13.5 mg oral once daily (21-day cycle; two weeks on, one week off) until disease recurrence, unacceptable toxicity, withdrawal of consent, or investigator decision, but no longer than 12 months (max. 18 cycles). The primary objective is to assess the efficacy of pemigatinib administered after curative local therapy in treatment-naïve patients with resectable intrahepatic biliary tract cancer (recurrence free survival rate at 12 months, RFS@12). Secondary objectives are to assess the efficacy by overall survival (OS) and recurrence free survival (RFS); to assess safety of the treatment (AEs, impact on liver function, use of subsequent therapies); to assess quality of life (QoL). In addition, tissue samples will be analyzed for biomarkers predictive for RFS and OS. 20 patients are to be enrolled in this trial.

Treatment of Non-resectable Bile Duct Cancer with Radiofrequency Ablation or Photodynamic Therapy

Klatskin tumours are a form of bile duct cancer. They are generally not diagnosed until quite late and a curative operation is rarely a possibility. Their anatomic location usually results in bile duct obstruction and the aim of therapy is thus to keep the ducts open. This is accomplished through endoscopic retrograde cholangiopancreatography (ERCP) by implanting stents. Stent therapy combined with photodynamic therapy (PDT) extends life expectancy. PDT requires an injection of photosensitizer that is absorbed primarily by the cancer cells. Light of a particular wavelength is then applied with ERCP to kill the cancer cells. Drawbacks include not only high costs and poor availability, but foremost that patients have to avoid direct sunlight for a period of weeks. Radio frequency ablation (RFA) together with stent implantation constitutes an alternative by which the cancer cells are killed through heat applied during ERCP. The RFA technology is more widely available and easier to deploy. However, it has not been studied extensively and no randomized trials exist comparing the two methods. This trial will compare survival in patients with Klatskin tumours depending on whether they receive PDT or RFA. Moreover, data will be collected on side-effects and quality of life.

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

Evaluation of a Smartphone Application for Self-help for Social Anxiety

The study will test the effectiveness of a newly developed mobile app in treating patients with social anxiety. Participants will be randomly assigned to one of the following groups: app use only, therapist-guided app use, and a wait-list control group. Over a 12-week period, the study will examine whether mobile app use leads to significant reductions in symptoms associated with social anxiety, as well as improvements in secondary outcomes such as quality of life, depression, or psychological impairment.

MED-EL HEARING SOLUTIONS (MEHS): AN OBSERVATIONAL STUDY

Strategies and Treatments for Respiratory Infections & Viral Emergencies (STRIVE): Shionogi Protease Inhibitor (Ensitrelvir)

START: Safety and Anti-Tumor Activity of PeptiCRAd-1 in Treatment of Cancer

This is a Phase I, open-label, non-randomized, first-in-human study. All patients will be pre-treated with a low dose of intravenous (i.v.) Cyclophosphamide (CPO) followed by monotherapy doses of PeptiCRAd-1. PeptiCRAd-1 will be administered by intratumoral (i.t.) injection (Cohort 1) and by i.t. and subcutaneous (s.c.) injection (Cohort 2) followed by combination therapy with PeptiCRAd-1 and i.v. CPI (pembrolizumab).

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Safety and Efficacy of Intra-arterial Tenecteplase for Noncomplete Reperfusion of Intracranial Occlusions