Oslo

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating a new treatment approach for meningiomas that continue to grow despite local therapies like surgery and radiotherapy. This trial focuses on the precision medicine concept of combining molecular imaging for patient selection with targeted treatment using a radioligand called 177Lu-DOTATATE. The study builds on evidence showing high expression of somatostatin receptors in meningiomas and prior success of this radioligand therapy in other tumors, aiming to explore its use in refractory meningiomas in a randomized Phase II study. Participants are randomly assigned to receive either the investigational treatment 177Lu-DOTATATE given by intravenous injection or the local standard of care, which may include treatment or observation as decided by their doctor. The trial involves baseline PET imaging to confirm somatostatin receptor positivity and follows patients after treatment. This design allows researchers to compare the effects of the new therapy against current practices in managing recurrent meningioma. During the study, participants undergo assessments including cranial MRI scans to measure tumor status and PET imaging for receptor evaluation. Blood tests are performed to monitor organ function and electrolytes before and during treatment. The main outcome measured is progression-free survival, tracking the time from randomization until disease progression or death, with follow-up for up to two years. Safety and treatment adherence are closely monitored throughout the study period.

The LuDO-N Trial is a phase II study focused on children with recurrent or relapsed high-risk neuroblastoma, a type of cancer. It aims to evaluate the response to treatment with 177Lu-DOTATATE, a radiolabeled drug, at 1 month and 4 months after treatment ends. The trial builds on prior experience, using an intensified dosing schedule to deliver two doses over two weeks, intending to maximize effects against this rapidly progressing disease. Researchers also want to study survival rates, treatment-related side effects, and relationships between tumor imaging and treatment response. Participants receive 177Lu-DOTATATE based on their weight, with the first dose set at 200 MBq per kg. The second dose is adjusted using scans to measure kidney radiation exposure, ensuring the total radiation remains within safe limits. The treatment plan includes careful monitoring of radiation doses to avoid kidney damage while aiming for an effective whole-body dose across two courses. During the study, children undergo various assessments including imaging scans such as 68Ga-DOTATATE PET/CT and 123I-mIBG scintigraphy, laboratory blood tests, and monitoring of kidney function. Researchers track treatment response using established neuroblastoma criteria one month after treatment completion. The study requires informed consent and readiness for stem cell transplantation. Treatment safety, tumor response, and survival outcomes are closely followed throughout the trial.

Researchers are studying whether calderasib alone or combined with cetuximab can treat advanced solid tumors in people who have the KRAS G12C mutation. This phase 2, open-label trial aims to find out how many participants respond to these treatments and to compare their safety and tolerability. Participants receive calderasib by mouth and cetuximab through intravenous infusion. The study includes people with locally advanced or metastatic solid tumors other than colorectal cancer, who have already undergone standard treatments. The trial monitors response and side effects over time as participants receive either calderasib alone or in combination with cetuximab. During the study, participants undergo regular assessments to measure tumor response and track any side effects or adverse events. Researchers record how many people experience treatment-related side effects and how many stop treatment due to these effects. The study follows participants for up to approximately 76 months to assess long-term outcomes and safety.

Researchers are evaluating a new treatment called ifinatamab deruxtecan (I-DXd) for men with metastatic castration-resistant prostate cancer (mCRPC). This study compares I-DXd to chemotherapy to see if it helps people live longer overall and live longer without their cancer worsening. It is a Phase 3, open-label trial focused on patients who have progressed on prior therapies and have evidence of metastatic disease. Participants receive either I-DXd through an intravenous infusion every 3 weeks or docetaxel chemotherapy administered every 3 weeks. Prednisone tablets are also given daily as part of the treatment plan. Before each I-DXd dose, premedication is provided to help prevent nausea and vomiting using a combination of drugs such as corticosteroids and anti-nausea medicines. Treatment continues until disease progression, unacceptable side effects, or other reasons to stop. During the study, researchers monitor overall survival and how long patients live without their cancer progressing, for up to about 36 months. Participants undergo tumor tissue collection, scans, and assessments to track disease status and side effects. Safety is closely watched throughout treatment. The study includes men aged 18 and older with confirmed prostate cancer and metastatic disease who have previously received certain hormone therapies but no prior taxane chemotherapy for mCRPC.

Researchers are investigating new treatments for advanced ovarian cancer, specifically in patients who do not have homologous recombination deficiency (non-HRD positive). This Phase 3 study aims to assess whether maintenance treatment with sacituzumab tirumotecan (sac-TMT), alone or combined with bevacizumab, can improve progression-free survival compared to the current standard care after initial platinum-based chemotherapy and surgery. Participants receive sacituzumab tirumotecan through intravenous infusion at a dose of 4 mg/kg. Some also receive bevacizumab intravenously at 15 mg/kg as part of their maintenance treatment. Before sac-TMT infusion, participants are given prophylactic steroid mouthwash and recommended rescue medications including histamine-1 and histamine-2 receptor antagonists, acetaminophen or equivalent, and dexamethasone or equivalent. The study compares these treatments to standard care or observation following first-line chemotherapy. During the study, participants are monitored for progression-free survival for up to approximately 49 months. Researchers will assess how long participants live without their cancer getting worse. Throughout the trial, safety and response to treatment are evaluated. The study includes women aged 18 years and older who have completed surgery and first-line chemotherapy with specific responses and meet certain health criteria.

Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide (TransCon CNP) in infants diagnosed with achondroplasia, a genetic condition affecting growth. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial involves infants aged from birth up to but not including 2 years, with genetically confirmed heterozygous achondroplasia. The study aims to monitor the growth impact and safety of weekly doses of Navepegritide over one year. Participants receive either 100 micrograms per kilogram of Navepegritide or a placebo, both administered as subcutaneous injections once per week for 52 weeks. This treatment period is followed by an open-label extension phase where participants may continue receiving the study drug. The trial compares the effects of the investigational drug against placebo to assess its tolerability and growth outcomes. Throughout the 52 weeks, infants will undergo regular medical evaluations including physical examinations, vital signs monitoring, ECGs, imaging, and laboratory tests. Researchers will track adherence to weekly injections and daily vitamin D supplementation where applicable. The primary outcomes focus on safety and growth effects of Navepegritide, with continuous monitoring to ensure participant well-being during the trial period.

Researchers are evaluating a digital, personalized exercise program for adults recently diagnosed with axial spondyloarthritis (axSpA) to see if it can reduce disease activity. This 6-month randomized controlled trial includes a re-randomization after 3 months and measures disease activity primarily at the 3-month mark using the ankylosing spondylitis disease activity score (ASDAS). Participants have active disease, are biologic disease-modifying anti-rheumatic drug (bDMARD) naive, and meet specific classification criteria for axSpA. The exercise program includes high intensity interval training (HIIT), muscular strength exercises, and moderate aerobic activity, delivered remotely through the SPARK app. One group receives personalized coaching and remote monitoring to support exercise progression and adherence, while the other group follows a standard exercise program without direct coach contact. The exercise consists of five sessions per week on three days, including two HIIT sessions targeting 85-95% of maximal heart rate. Participants will complete their exercises at home or another chosen location while logging activity data via a sports watch. Researchers will assess disease activity, physical fitness including cardiorespiratory and muscular strength tests, and psychological wellbeing. Safety is monitored through physical exams, laboratory tests, adverse event recording, and MRI scans. The total study duration is 6 months with assessments at 3 months for the primary disease activity outcome.

Researchers are investigating the physical and psychological effects of hormonal treatment in transsexual persons. This European multicenter study, named 'Transparency,' addresses the lack of detailed descriptions of these effects by using a common standardized hormonal protocol. The study aims to provide prospective data on this topic, motivated by the low prevalence of transsexualism and the need for collaboration across European partners. Participants undergo various assessments including descriptive questionnaires such as SF-36 and physical activity questionnaires, blood sampling for a broad range of tests including liver function, hormones, and metabolic markers, and clinical evaluations measuring height, weight, blood pressure, and other physical parameters. Additional evaluations include dermatology assessments for hair and skin conditions, and bone density measurements using PQCT scanning. Throughout the study, participants are monitored annually to evaluate the physical and psychological impacts of the hormonal treatments. The study collects detailed data through questionnaires, laboratory tests, clinical exams, and imaging to measure the outcomes. This comprehensive approach aims to better understand the effects of hormone therapy in this population over time.