Uppsala

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are conducting a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of rilzabrutinib in adults with active Immunoglobulin G4-related disease IgG4-RD. The study aims to measure the time to the first adjudicated disease flare and assess other important outcomes such as flare-free rates, disease activity control, glucocorticoid use, and safety parameters including adverse events, laboratory tests, and electrocardiograms ECG. Participants will be assigned to one of two groups one receiving rilzabrutinib tablets and the other receiving placebo tablets, both administered orally. The treatment period lasts 52 weeks in a double-blind manner, preceded by a 4 to 6 week screening period. After treatment, there is a 2-week follow-up, with an optional open-label extension lasting up to 108 weeks. The study includes a total of 16 visits during the main period and up to 9 additional visits during the optional extension. During their participation, adults diagnosed with IgG4-RD will undergo repeated imaging procedures such as CT, MRI, PET, or ultrasound to assess disease status. Researchers will monitor disease flares, remission status, glucocorticoid dosage, clinical activity scores, laboratory values, vital signs, and ECG results. Safety monitoring continues up to week 160 to capture treatment-emergent adverse events. Overall, participation lasts up to 60 weeks, with possible extension for those continuing in the optional phase.

Researchers are studying metastatic castration-resistant prostate cancer mCRPC to find new treatment options. This trial evaluates if the study medicine ifinatamab deruxtecan I-DXd or MK-2400 helps people live longer overall and experience slower cancer growth or spread compared to chemotherapy. The study is a Phase 3 trial comparing I-DXd with standard chemotherapy for mCRPC patients. Participants are randomly assigned to receive either I-DXd at 12 mgkg every 3 weeks through intravenous infusion or docetaxel chemotherapy at 75 mgm2 every 3 weeks combined with daily prednisone pills. Treatment continues until the disease progresses, unacceptable side effects occur, or treatment is stopped for other reasons. Premedication is given before each dose of I-DXd to help prevent nausea and vomiting. During the study, participants will have regular visits for treatment and monitoring. Researchers will assess overall survival and radiographic progression-free survival for up to about 36 months. Additional measures include response rates, time to pain progression, PSA progression, and adverse events. The study tracks safety, treatment effects, and quality of life over a long follow-up period to better understand the potential benefits and risks of I-DXd compared to chemotherapy.

Researchers are investigating new treatments for high-risk, early-stage breast cancer, specifically targeting two types triple-negative breast cancer TNBC and hormone receptor-low positiveHER2-negative breast cancer. These cancers are characterized by low or no HER2 protein and low hormone receptor presence. The study aims to evaluate if adding sacituzumab tirumotecan sac-TMT to pembrolizumab and chemotherapy can better reduce cancer cells in tumors and lymph nodes and improve the length of time patients live without cancer progression compared to pembrolizumab with chemotherapy alone. Participants in this trial receive one of two treatment plans. One group gets sacituzumab tirumotecan intravenously every two weeks plus pembrolizumab every three weeks for 12 weeks, followed by pembrolizumab with carboplatin and paclitaxel for another 12 weeks. After 3 to 6 weeks, surgery and optional radiation therapy take place, followed by pembrolizumab for about 28 weeks. Participants with remaining disease may receive additional treatments chosen by their doctors, including olaparib, capecitabine, doxorubicin, epirubicin, or cyclophosphamide. The other group receives chemotherapy drugs carboplatin and paclitaxel with pembrolizumab initially, then pembrolizumab with cyclophosphamide and doxorubicin or epirubicin, followed by surgery, optional radiation, and pembrolizumab for about 28 weeks, with similar additional options for residual disease. During the study, participants undergo core needle biopsies, receive intravenous infusions of study drugs, and have surgery and possible radiation therapy. Researchers assess outcomes such as the percentage of participants with no detectable cancer cells at surgery pathological complete response, event-free survival up to about 92 months, and overall survival up to nearly 10 years. Quality of life and side effects are monitored through questionnaires and adverse event tracking. The study lasts several years, with various assessments throughout treatment and follow-up periods to gather comprehensive data on treatment effects and safety.

Researchers are evaluating the effectiveness and safety of an investigational drug called BNT323 also known as DB-1303 compared with standard chemotherapy in women with recurrent endometrial cancer. The study includes two groups based on the level of HER2 protein in the tumor Cohort 1 with HER2 levels 1 or 2 who have been previously treated with immune checkpoint inhibitors, and Cohort 2 with HER2 level 3. The study aims to understand how well BNT323 or chemotherapy controls cancer progression and how the drug affects patients immune response and quality of life. Participants in Cohort 1 will be randomly assigned to receive either BNT323 or chemotherapy drugs such as doxorubicin, paclitaxel, or docetaxel. In Cohort 2, participants will receive BNT323 alone. Treatments are given intravenously and continue until the cancer progresses, unacceptable side effects occur, or consent is withdrawn. The study includes screening, treatment, safety follow-up, efficacy follow-up, and a long-term survival follow-up lasting up to about 53 months. During the study, participants will undergo regular assessments including tumor evaluations, safety monitoring, and quality of life questionnaires. Researchers will measure progression-free survival in Cohort 1 and tumor response rate in Cohort 2. Safety is monitored by tracking adverse effects and drug levels in the body. Participants can expect to be followed for up to 53 months after treatment to assess long-term outcomes and survival.

Researchers are evaluating the safety, tolerability, and distribution of a drug called 177LuLu-ABY-271 in people with HER2-positive metastatic breast cancer. This early phase 1 trial aims to understand how the drug behaves in tumors and key organs, helping to guide future treatments. The study is open-label and randomized, involving female participants aged 18 and older with advanced breast cancer confirmed by specific tests. The trial has two parts. Part A will enroll up to 6 participants to study how the drug is absorbed by tumors and critical organs after a single infusion. Part B will include 15 participants divided into three groups, each receiving a different protein mass dose with varying radioactivity levels, to determine the best dose for future studies. Treatments are given as a single infusion of 177LuLu-ABY-271. Participants will be closely monitored through assessments of side effects and dose-limiting toxicities up to 29 days for Part A and 43 days for Part B. Researchers will also measure radiation absorbed by organs and tumors within 8 days after treatment. The study includes regular evaluations of tumor size, health status, and overall safety. The total participation time varies depending on the study part and dosing schedule.

Researchers are conducting a first-in-human phase 1 trial to study OX118, a human monoclonal antibody targeting OX40 ligand, in healthy adults aged 18 to 60 years. The trial aims to evaluate the safety, tolerability, and how the body processes OX118 when given as a single intravenous dose. This study involves 32 healthy volunteers with a body mass index between 18.5 and 30.0. Participants are divided into five groups receiving different single doses of OX118 or a placebo by intravenous infusion. The doses range from 0.1 mgkg to 10 mgkg, with participants randomized to receive either the drug or placebo based on a pre-arranged schedule. The trial includes sentinel dosing for initial participants in each group, and all participants stay at the clinic for at least 24 hours after dosing for monitoring. Each participant will attend 7 clinic visits over about 91 days, including a 28-day screening period. Visits include treatment administration, safety assessments such as vital signs, ECGs, physical exams, laboratory tests, and monitoring for adverse events. Safety data is reviewed by an internal committee before moving to the next dosing group. Researchers will measure adverse events, vital signs, lab results, ECG changes, and physical exam findings from Day 1 to Day 63, along with drug concentration and immune response data.

Researchers are studying people with idiopathic pulmonary fibrosis IPF or progressive pulmonary fibrosis PPF who have previously taken nerandomilast in another study. The aim is to assess how well patients tolerate long-term treatment with nerandomilast and to evaluate whether it improves lung function and delays worsening symptoms, hospital visits, or death. This open-label extension trial is sponsored by Boehringer Ingelheim and focuses on treatment over an extended period. Participants take nerandomilast tablets for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study involves a single treatment group receiving the drug, and no placebo or comparison groups. Regular visits with doctors help monitor health and collect data during this extended treatment phase. Throughout the study, participants undergo regular lung function tests and health assessments to track any adverse events and changes in lung capacity. The main outcome measured is the occurrence of any adverse events for up to about 99 weeks. Secondary outcomes include changes in forced vital capacity and time to worsening of disease symptoms or hospitalization. The study includes ongoing safety monitoring with a total participation time of up to nearly two years.

Researchers are studying Severe and Enduring Eating Disorders SEED, which are long-lasting eating disorders affecting adolescents and adults. The study aims to understand the clinical, psychological, and biological factors that contribute to the development and persistence of SEED. It follows two groups of patients, one aged 14-17 and one aged 18 and older, to observe changes and impacts over time. Participants will be followed at multiple points at the start baseline, after treatment, two years later, and then at five, 10, and 20 years after baseline. During these times, they will undergo physical exams, provide blood samples, participate in interviews, and complete questionnaires. Caregivers of minors will also complete questionnaires. The study collects extensive data on psychiatric symptoms, personality, emotion regulation, loneliness, and inflammatory markers. Throughout the study, participants will be assessed with various interviews and questionnaires to measure eating disorder symptoms, psychosocial functioning, quality of life, and biological markers. Weekly weight checks occur during treatment and follow-up. Researchers will analyze how different factors influence the course of the disorder and treatment outcomes. The study may last up to 20 years for some participants and includes regular digital questionnaire responses and registry data reviews.

Researchers are evaluating the binding of a new PET tracer called 68GaGa-DOTA-Cys-ATH001 in the liver and gastrointestinal tract. The study compares healthy volunteers with patients who have metabolically caused steatohepatitis MASH, fibrostenotic Crohns Disease CD, and primary sclerosing cholangitis PSC. This first-in-human, phase 0, multi-center trial aims to understand tracer uptake patterns and assess its safety and distribution in the body. Participants receive a single intravenous injection of up to 100 micrograms of the tracer, followed by PET and MRI scans to visualize tracer binding and body function. Some participants in specific cohorts undergo a second injection and imaging visit within six weeks to test tracer reliability. Blood samples are collected from certain groups to measure tracer levels over time. Safety assessments and remote follow-up calls are also part of the study protocol. During the study, participants will have health checks and eligibility screening before dosing. Imaging includes whole-body PETMRI scans and specific examinations like FibroScan for some groups. Safety is monitored through vital signs, ECG, lab tests, and injection site observations. Follow-up calls check on any side effects or medication use. The trial involves 2 to 3 visits depending on the participants cohort and includes detailed assessments of tracer uptake and pharmacokinetics.