Clinical trialCLINICALTRIALSApr 6
Researchers are testing a new drug called zipalertinib to see if it helps people with early-stage lung cancer that has specific genetic mutations. Patients who had surgery to remove their tumors will receive either the new drug or a placebo (fake medicine) along with standard chemotherapy. The study wants to find out if zipalertinib can prevent the cancer from coming back better than chemotherapy alone.
WHY IT MATTERSIf you have early-stage NSCLC with uncommon EGFR mutations (like exon 20 insertions) and recently had surgery, this trial offers access to a targeted therapy specifically designed for your mutation type before it becomes widely available.
ResearchPUBMEDApr 5
Researchers looked at studies from 2014 to 2024 about the cost and value of special medicines for rare kidney diseases in poorer countries. They found that there isn't much research on whether these expensive medicines are worth the money in these areas. The study shows that doctors and governments need better information to decide if patients should have access to these treatments.
WHY IT MATTERSIf you have a rare kidney disease in a low- or middle-income country, this research highlights why your access to orphan drugs is limited — there's a major gap in evidence about whether these treatments are affordable and effective in your region.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called ManNAc to treat primary focal segmental glomerulosclerosis (FSGS), a rare kidney disease that causes scarring and can lead to kidney failure. This is a Phase 2 trial, meaning the drug has already been tested for safety in a small group and now researchers want to see if it actually works to help patients. The trial is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers patients with primary FSGS access to an investigational treatment that may slow or stop kidney damage, with enrollment now open at sites participating in the National Human Genome Research Institute study.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called CYT107 to help people with HIV who have Kaposi Sarcoma (a type of cancer) and whose immune systems aren't responding well to treatment. CYT107 is designed to boost the immune system to help fight the cancer. This is a Phase 2 trial, meaning it's testing whether the drug works and is safe in a larger group of people. The trial is now accepting patients and will start in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for people with HIV-related Kaposi Sarcoma who haven't responded to standard immune-boosting treatments, addressing a significant gap in care for this vulnerable population.
Clinical trialUNITERAREApr 5
Researchers are testing a drug called baricitinib to see if it is safe and works well for patients with Job syndrome who also have lupus-like disease and/or atopic dermatitis (severe itchy skin). Job syndrome is a rare immune system disorder that makes it hard for the body to fight infections and causes skin problems. This is an early-stage study that will check for side effects and how well the drug works.
WHY IT MATTERSThis Phase 1 trial is now actively recruiting patients with Job syndrome complicated by lupus-like disease and/or atopic dermatitis — a rare combination that has limited treatment options and is being studied by the National Institute of Allergy and Infectious Diseases.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms — rare blood disorders where the bone marrow doesn't make enough healthy blood cells. The drug works by blocking several proteins that may be causing these diseases. This is an early-stage study sponsored by the National Cancer Institute that will start recruiting patients in April 2026.
WHY IT MATTERSThis trial offers patients with myelodysplastic syndromes access to a novel multi-targeted kinase inhibitor that may address disease mechanisms not covered by current standard treatments, with enrollment beginning in April 2026.
Clinical trialUNITERAREApr 4
Researchers are testing a new drug called JL15003 to see if it can safely treat glioblastoma, a serious brain cancer that has come back after previous treatment. This is an early-stage study that will check both how safe the drug is and whether it helps patients live longer or feel better. The study is now accepting patients and will run starting in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with recurrent glioblastoma, a condition with very limited treatment choices after initial therapy fails.
🔴 BreakingDrug approvalOPENFDAApr 4
The FDA has approved a new drug called FOUNDAYO (orforglipron), made by Eli Lilly. This approval means the medication is now available for patients to use. The drug was officially approved on April 1, 2026.
WHY IT MATTERSWithout information about which rare disease FOUNDAYO treats, we cannot specify the patient impact — the article lacks details about the condition it addresses or which patients are eligible.
Clinical trialUNITERAREApr 3
Researchers are testing a new treatment approach for elderly patients with a type of blood cancer (MDS or AML) that has come back or stopped responding to previous treatments. The study uses a combination of chemotherapy drugs followed by a stem cell transplant from a donor to try to help patients recover. This trial is now actively looking for patients to participate.
WHY IT MATTERSThis trial offers elderly patients with relapsed or refractory MDS/AML a potentially less toxic conditioning regimen before stem cell transplant — cladribine-bridged LABU may improve tolerability compared to standard high-dose approaches in this vulnerable population.
Clinical trialCLINICALTRIALSApr 3
Researchers are looking for 100 people with rare types of joint inflammation to better understand how these conditions develop and damage joints. Some of these inflammatory arthritides occur alongside rare autoimmune diseases like systemic sclerosis, while others develop as side effects from cancer immunotherapy drugs. By studying tissue samples and immune cells from patients, scientists hope to discover why these conditions happen and potentially develop better treatments.
WHY IT MATTERSIf you have rare inflammatory arthritis, systemic sclerosis with joint involvement, or developed joint inflammation after cancer immunotherapy, this trial could help researchers understand your condition better and may lead to more targeted treatments in the future.
Clinical trialUNITERAREApr 3
Researchers at St. Jude Children's Research Hospital are testing a drug called entrectinib to treat very young children (under 3 years old) who have brain or spinal cord tumors caused by changes in specific genes called NTRK1, NTRK2, NTRK3, or ROS1. This is a Phase 2 trial, meaning they've already tested the drug in a small group and now want to see if it works well in more patients. The trial is now accepting new patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers a targeted treatment option specifically designed for infants and toddlers with NTRK- or ROS1-fused CNS tumors, a group that has historically had very limited treatment options and poor outcomes.
Clinical trialUNITERAREApr 3
Researchers are testing a new experimental drug called KST-6051 in patients with advanced solid tumors (cancers that form in organs or tissues) that have a specific genetic mutation called KRAS. This is an early-stage study (Phase 1) that will test different doses of the drug to find the safest and most effective amount. The trial is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers patients with KRAS-mutated solid tumors access to a novel targeted therapy before it becomes widely available, potentially providing a treatment option for cancers that are often difficult to treat.
Clinical trialUNITERAREApr 3
Researchers are testing a new experimental drug called EPI-326 in people with two types of cancer: non-small cell lung cancer and head and neck cancer, both caused by mutations in the EGFR gene. This is an early-stage study (Phase 1) to see if the drug is safe and how much patients can tolerate. The study is currently accepting new participants and is expected to start in April 2026.
WHY IT MATTERSThis trial offers access to a novel EGFR-targeted therapy for patients with EGFR-mutant lung or head and neck cancers who may have limited treatment options or resistance to current therapies.
Clinical trialUNITERAREApr 3
Researchers are looking for patients with a specific type of brain cancer called glioblastoma (GBM) to test a new drug combination. The study will test a drug called silevertinib combined with temozolomide in patients whose tumors have certain genetic features (unmethylated MGMT and EGFRvIII). This is an early-stage study to see if this combination works better than current treatments.
WHY IT MATTERSThis trial targets newly diagnosed GBM patients with unmethylated MGMT and EGFRvIII mutations—a subgroup with historically poor prognosis—and offers access to a potentially more effective treatment combination before it becomes widely available.
Clinical trialUNITERAREApr 3
Researchers are testing a new combination of three drugs (RBS2418, tremelimumab, and durvalumab) to treat advanced hepatocellular carcinoma, a type of liver cancer that cannot be surgically removed. This is an early-stage clinical trial (Phase 2) that is currently accepting patients. The study aims to see if combining these drugs works better than existing treatments.
WHY IT MATTERSThis trial offers eligible patients with advanced unresectable hepatocellular carcinoma access to a novel three-drug combination that may provide a new treatment option beyond current standard therapies.
Clinical trialUNITERAREApr 3
Researchers are testing a new drug called Xaluritamig to treat Ewing sarcoma, a rare bone cancer that has come back or stopped responding to previous treatments. This is an early-stage study (Phase 1) that will include children, teenagers, and adults. The study is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with relapsed or refractory Ewing sarcoma, a rare pediatric cancer with limited treatment alternatives after standard therapies fail.
Clinical trialUNITERAREApr 3
Lumos Pharma is testing a new drug called LUM-201 in children who don't produce enough growth hormone naturally. This Phase 3 trial is the final stage of testing before the drug might be approved by the FDA. Researchers are actively recruiting children to participate and see if this treatment helps them grow taller.
WHY IT MATTERSThis is now actively recruiting children with growth hormone deficiency — families interested in potentially accessing LUM-201 before FDA approval should check eligibility requirements and contact the trial sites listed on ClinicalTrials.gov.
Clinical trialUNITERAREApr 3
Researchers are testing a new combination treatment for advanced kidney cancer that has spread to other parts of the body. The treatment combines two immunotherapy drugs (nivolumab and ipilimumab) with an experimental live biotherapeutic product called EXL01. This Phase 1 trial is now accepting patients to see if adding EXL01 to the standard immunotherapy improves outcomes.
WHY IT MATTERSThis trial offers metastatic renal cell carcinoma patients access to a novel combination approach that may enhance the effectiveness of existing immunotherapies by incorporating a live biotherapeutic product.
Clinical trialUNITERAREApr 3
Researchers are looking for patients with a type of blood cancer called acute lymphoblastic leukemia (ALL) to test a new treatment combination. The study will test whether adding a drug called JZP458 to standard chemotherapy works better than chemotherapy alone. This trial is just starting and will recruit patients beginning in April 2026.
WHY IT MATTERSThis Phase 2 trial is now actively recruiting newly diagnosed ALL patients without the Philadelphia chromosome, offering access to an investigational asparaginase formulation (JZP458) that may have improved tolerability compared to standard asparaginase.
Clinical trialUNITERAREApr 3
Prelude Therapeutics is testing a new drug called PRT12396 in people with certain blood cancers called myeloproliferative neoplasms. This is an early-stage study (Phase 1) to see if the drug is safe and how it works in the body. The study is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers access to a potentially new treatment option for patients with myeloproliferative neoplasms who may have limited alternatives, particularly if they have specific subtypes targeted by this drug.