Muscat

Diffuse large B-cell lymphoma DLBCL is the most common and aggressive subtype of non-Hodgkin lymphoma, with rapid tumor growth and symptoms varying by tumor location. The disease is classified using advanced methods like immunohistochemistry and molecular testing to guide prognosis and personalized treatment. Despite standard immunochemotherapy curing 60-70% of patients, many experience treatment failure or relapse, and options after second-line therapies remain limited. This observational study examines treatment patterns, demographic, and clinical characteristics of DLBCL patients in the Middle East and Africa. It includes two patient groups those newly diagnosed and eligible for treatment, and those with relapsed or refractory DLBCL who have failed previous therapies. The study aims to explore access to novel therapies and understand real-world treatment approaches in these regions. Participants will be observed over 22 months, during which researchers will document treatment regimens, patient risk profiles, and access to new treatments. Data collection includes patient demographics, disease characteristics, and treatment outcomes. The study does not involve experimental treatments but gathers detailed information to inform future care strategies for DLBCL patients.

Researchers are studying etavopivat, a new medicine, in children aged 12 to 16 years with sickle cell disease who have an increased risk of stroke. The trial focuses on patients with conditional or abnormal transcranial doppler TCD ultrasound results, assessing whether etavopivat is safe and helpful for these participants. The study is a Phase 2 open-label trial sponsored by Forma Therapeutics, Inc., aiming to understand the effect of etavopivat on blood flow velocities in brain arteries. Participants will be divided into two groups based on their TCD results and whether they are already taking the medication hydroxyurea. One group includes participants with abnormal or conditional TCD who are not on hydroxyurea, while the other group includes those with similar TCD results who are on a stable dose of hydroxyurea. All participants will take 400 mg of etavopivat orally once daily for 52 weeks, with the option to continue in a 48-week extension period to further monitor safety. Etavopivat is taken as two 200 mg tablets and may be taken with or without food. During the study, participants will visit the clinic frequently for monitoring. Assessments include measuring blood flow velocities in brain arteries using TCD at various time points, tracking changes in velocity categories, and monitoring safety. The study also includes evaluating blood counts and liver and kidney function. At the end of the treatment and extension periods, participants may be offered the chance to join another study to continue receiving etavopivat. Total participation can last up to about two years depending on extension and further studies.

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

Researchers are evaluating the effects of etavopivat in adolescents and adults with sickle cell disease. This study aims to confirm whether etavopivat reduces the number of painful vaso-occlusive crises caused by blood vessel blockages. It also examines if the medicine helps reduce organ damage, improve exercise tolerance, and decrease fatigue. The study is a phase 3, randomized, double-blind, placebo-controlled trial lasting about two years. Participants will be randomly assigned to receive either oral etavopivat or a placebo. The treatment is given by mouth, and neither the participants nor the researchers know which treatment is given during the study to ensure impartial results. The primary treatment period is 52 weeks, during which the number of vaso-occlusive crises with medical contact will be measured. Secondary assessments include changes in hemoglobin levels, fatigue scores, walking distance, and other blood markers related to sickle cell disease. During the trial, participants will undergo regular evaluations including blood tests, walking tests, and fatigue assessments from baseline to week 52. Researchers will monitor the safety and effectiveness of etavopivat throughout the study. The total participation time is about two years, with ongoing follow-ups to observe the treatment effects and any side effects. All procedures aim to provide detailed information on how etavopivat affects sickle cell disease symptoms and patient well-being.

Researchers are evaluating the effectiveness and safety of inavolisib combined with Phesgo compared to a placebo combined with Phesgo as maintenance treatment for participants with previously untreated HER2-positive advanced breast cancer that has a PIK3CA mutation. This Phase 3 study focuses on participants with locally advanced or metastatic breast cancer who have completed induction therapy. Participants first receive induction therapy with Phesgo plus taxane-based chemotherapy. Following this, they enter the maintenance phase where they are randomly assigned to receive either inavolisib tablets taken orally once daily for 21 days of each 21-day cycle along with Phesgo administered subcutaneously every 3 weeks, or a matching placebo tablet with Phesgo on the same schedule. Optional endocrine therapy may be given based on the investigators choice according to standard care. Throughout the study, participants undergo regular monitoring including tumor assessments, quality of life questionnaires, and safety evaluations lasting up to approximately 111 months. Key outcomes measured include progression-free survival assessed by investigators, overall survival, response rates, duration of response, and adverse event rates. Plasma concentrations of inavolisib are also measured at specific timepoints. Participants will be followed closely during and after treatment to assess these outcomes over an extended period.

This research investigates the effects of an Aescin-based herbal extract called Reparil compared with Ibuprofen in managing symptoms after surgical removal of impacted lower wisdom teeth. The study focuses on people aged 18 to 40 who undergo this common oral surgery, aiming to find out if the herbal treatment offers pain relief and reduces swelling and limited mouth opening with fewer side effects than traditional nonsteroidal anti-inflammatory drugs NSAIDs. Participants will be randomly assigned to receive either Reparil tablets containing 20 mg Aescin or 400 mg Ibuprofen tablets, each taken orally three times a day for five days after surgery. Both groups follow the same schedule, and a rescue pain medication is available if needed. Assessments will occur before surgery and on days 2 and 7 afterward to measure pain, facial swelling using 3D scanning, and mouth opening using a digital caliper. During the study, participants will have their pain levels, facial swelling, and jaw movement tracked at specific intervals. Researchers will record any adverse effects and analyze data to compare the treatments impacts. The total involvement includes the surgery and follow-up visits on days 2 and 7, helping to evaluate the herbal extract as a potential alternative to Ibuprofen for managing post-surgical symptoms.

Researchers are studying real-world treatment patterns, patient characteristics, survival outcomes, and safety of Trastuzumab Deruxtecan T-DXd in women with hormone receptor-positive metastatic breast cancer mBC that is HER2-low or HER2-ultralow. This study focuses on patients previously treated with endocrine therapy and aims to provide data from Gulf Cooperation Council countries to improve treatment strategies and clinical guidelines for mBC in the region. This observational study monitors patients who have initiated T-DXd treatment as part of routine care, including those who started the drug up to 30 days before consenting to participate. It includes women aged 18 years or older with confirmed HR-positive, HER2-low or ultralow metastatic breast cancer who have not received chemotherapy in the metastatic setting. The study collects data over 18 months on treatment patterns, safety, and progression-free survival. Participants are observed while receiving their usual medical care with T-DXd, without additional intervention by the study team. Researchers collect information on patient characteristics, treatment details, safety events, and time to next treatment. The main outcome measured is real-world progression-free survival at 18 months, along with safety profiles and treatment patterns. The study is expected to last until March 2029, supporting improved outcomes through real-world evidence.

Researchers are evaluating the effect of two different doses of dexamethasone, 4mg and 8mg, given as a submucosal injection to reduce pain after dental implant surgery. The study is a randomized, double-blind, controlled clinical trial that also includes a placebo group receiving normal saline. The goal is to find the best dose of dexamethasone for managing postoperative pain while considering the influence of preoperative anxiety on pain levels. Participants will be randomly assigned to one of three groups receiving 8mg dexamethasone, 4mg dexamethasone mixed with saline, or a placebo injection of saline alone. Each injection is given near the surgical site in the buccal vestibule. The injections are concealed to ensure blinding. The study is planned to start in March 2026 and will follow participants for one week after surgery. During the study, postoperative pain will be measured using a Visual Analog Scale at 6 hours after surgery and daily for the next six days. The number of pain-relief tablets taken will also be recorded during this time. Preoperative anxiety levels will be assessed on the day of surgery using the Generalized Anxiety Disorder-7 questionnaire. The study will monitor these outcomes to determine the effectiveness of the dexamethasone doses in controlling pain after dental implant surgery.

Researchers are conducting the INTERSTELLAR study to gather important real-world evidence about adding anifrolumab to the usual treatment for systemic lupus erythematosus SLE. This observational study is taking place across multiple countries, including regions like GCC, Mexico, Central America, Colombia, Argentina, Taiwan, and Egypt. It aims to provide data useful for doctors, payers, and patients by using consistent criteria and measures for SLE and skin symptoms related to lupus. Participants will be observed for one year before starting anifrolumab and followed for one year after beginning treatment. The study starts after anifrolumabs commercial launch in each country and includes patients from the time of their first anifrolumab prescription and infusion. Data collection continues even if participants stop taking anifrolumab, unless they withdraw their consent. During the study, clinical assessments and patient-reported outcomes will be collected at baseline and at months 1, 3, 6, and 12. These include disease activity scores PGA, SLEDAI-2K, skin involvement measures CLASI, fatigue assessments FACIT-Fatigue, and quality of life questionnaires LupusQoL, EQ-5D-5L. The study monitors participants until death, loss to follow-up, withdrawal, or study end, aiming to reflect real-world use and outcomes of anifrolumab in routine care.

Researchers are evaluating the long-term safety, tolerability, and effectiveness of marstacimab as a preventive treatment in males aged 1 to 74 years with severe hemophilia A or moderately severe to severe hemophilia B, including those with or without inhibitors. This open-label extension study follows participants who completed prior Phase 3 studies without early termination, aiming to gather extended data on marstacimabs impact over several years. Participants receive marstacimab as a subcutaneous injection using a prefilled pen or syringe. Those aged 12 years and older start with a 300 mg loading dose followed by 150 mg weekly, with possible dose escalation to 300 mg weekly. Children aged 6 to under 12 years receive a 150 mg loading dose followed by 75 mg weekly, with the option to increase to 150 mg weekly. The study provides devices for administration and allows some flexibility based on participant needs and country-specific availability. During the study, participants undergo regular assessments including monitoring of adverse events, laboratory tests, coagulation factor consumption, bleeding rates, joint health, and quality of life questionnaires over a period of up to seven years. Researchers will track safety outcomes such as thrombotic events, injection site reactions, and immune responses to the drug. This extended follow-up helps understand marstacimabs long-term effects and supports dosing decisions in younger age groups.