University of Kansas

Researchers are studying children with congenital myotonic dystrophy type 1 CDM, a genetic muscle disorder that begins at birth and affects physical and cognitive development. The study aims to establish reliable clinical assessments and biomarkers to better understand developmental milestones, physical and cognitive function, and quality of life in this population. The study includes children from newborn to just under 4 years old with confirmed CDM, defined by specific early symptoms and genetic testing results. Participants are grouped as children aged from birth to 3 years and 11 months who have early symptoms such as low muscle tone, feeding or breathing difficulties requiring extended hospitalization, and a confirmed genetic expansion in the DMPK gene. The study focuses on tracking motor milestone attainment and other function measures over an 18-month period. No specific treatments are given since this is an observational study. Throughout the study, participants will be evaluated for motor skills and developmental progress using various assessments such as the Gross Motor Function Measure and Vineland scales. The study will also collect data on speech difficulties and other developmental domains. Guardians will provide consent and assist with study procedures. The research team will monitor progress and correlate clinical outcomes with potential biomarkers, with participation lasting up to 18 months.

Researchers are evaluating a drug called sigvotatug vedotin SGN-B6A alone and in combination with pembrolizumab, with or without chemotherapy, to assess its safety and effects in people with advanced solid tumors. This Phase 1 study aims to determine the side effects and whether sigvotatug vedotin works to treat various solid tumors including lung, head and neck, breast, esophageal, skin, pancreatic, bladder, cervical, gastric, and ovarian cancers. The study is divided into four parts to explore dosage, safety, and combination treatments. Participants may receive sigvotatug vedotin alone or combined with pembrolizumab, sometimes alongside chemotherapy drugs carboplatin or cisplatin, depending on the study part. Part A focuses on finding the right dose of sigvotatug vedotin. Part B uses this dose to further test safety and effectiveness. Parts C and D study the drug combined with pembrolizumab and possibly chemotherapy in different tumor types and treatment settings, including people who have not previously received treatment. Treatments are given intravenously, with pembrolizumab administered every 3 or 6 weeks and chemotherapy every 3 weeks. During the study, participants undergo tumor biopsies, clinical evaluations, and monitoring for side effects, including blood tests and safety assessments. Researchers track adverse events, lab abnormalities, and dose-limiting toxicities up to 30-37 days after treatment, with some follow-up extending up to 3 years. They also measure tumor response using standard criteria and monitor survival and drug levels in the body. Participants will have regular visits for treatment and assessments throughout the study duration, which may last several years.

This research aims to evaluate how well amivantamab given by subcutaneous injection works in treating patients with advanced non-small cell lung cancer that has extra copies of the MET gene. This phase II Expanded Lung-MAP treatment trial focuses on patients with MET amplification-positive non-small cell lung cancer. The goal is to see if amivantamab-SC can reduce tumor growth or spread by targeting the extra MET gene copies in these tumors. Participants receive amivantamab subcutaneously on days 1, 8, 15, and 22 during the first 28-day cycle, then on days 1 and 15 of subsequent 28-day cycles as long as the disease does not progress or unacceptable side effects occur. Throughout the trial, patients have regular CT or MRI scans and blood samples taken to monitor treatment effects and disease status. After completing treatment, patients are followed for up to 3 years to assess long-term outcomes. During the study, researchers evaluate how many patients respond to the treatment, how long responses last, progression-free survival, overall survival, and the frequency and severity of side effects. Blood samples are collected before treatment, during therapy, and at disease progression to study tumor DNA and build a tissue and blood repository. Safety labs, physical exams, and performance status assessments are also conducted. The total participation can last up to 3 years including follow-up.

Researchers are evaluating the combination of abemaciclib and olaparib in treating women with recurrent platinum-resistant ovarian cancer, a type of ovarian cancer that initially responds to platinum-based chemotherapy but returns within six months. This phase IIb trial aims to identify the side effects and best dose of abemaciclib when given together with olaparib. The study also explores the anti-tumor activity of this combination and collects genetic and biomarker data to understand treatment responses better. Participants receive olaparib orally twice daily for 28-day cycles, with abemaciclib added from day 8 to day 28 in the first cycle and throughout subsequent cycles, continuing until disease progression or unacceptable side effects occur. The study includes procedures like blood collection and tumor biopsies. After completing treatment, participants are followed up for 30 days to monitor outcomes and safety. During the study, participants undergo regular assessments including blood tests and tumor biopsies to measure response and side effects. Researchers track the maximum tolerated dose and recommended phase 2 dose after the first 28-day cycle. They also observe overall response rates and duration of response following treatment completion. Genetic and biomarker analyses are performed on collected samples, and safety is monitored throughout participation, which may last several treatment cycles plus follow-up.

Researchers are studying colorectal cancer CRC in participants aged 45 to 70 who have 1 to 2 non-advanced adenomas, which are small colon polyps without high-risk features. The trial compares two surveillance colonoscopy schedules one group receives colonoscopy at 5 and 10 years after polyp detection, while the other group has colonoscopy only at 10 years. This study aims to provide randomized data to guide optimal timing for follow-up colonoscopies to help reduce CRC incidence and improve clinical practices. Participants are randomly assigned to one of two groups the first group undergoes surveillance colonoscopy at both 5 and 10 years following their qualifying colonoscopy, and the second group undergoes colonoscopy at 10 years only. All colonoscopies, including any unscheduled ones, follow current quality guidelines and institutional preparation standards. The qualifying colonoscopy must have been complete and performed within 4 years before randomization, with all polyps fully removed. During the study, participants will undergo colonoscopies according to their assigned schedules. Researchers will monitor the incidence of colorectal cancer over 10 years as the primary outcome. Participants must be able to understand English or Spanish to complete required procedures. The study may last up to several decades, with ongoing monitoring to assess long-term cancer incidence and the effectiveness of different surveillance intervals.

Researchers are evaluating the combination of BP1001 plus venetoclax plus decitabine in adults with acute myeloid leukemia AML who cannot or choose not to receive intensive chemotherapy. The study aims to determine if this combination offers better results, such as complete remission, compared to venetoclax plus decitabine alone or to intensive chemotherapy in patients with untreated or relapsedrefractory AML. This Phase IIa trial focuses on patients who are fragile or ineligible for aggressive treatment, addressing a need for targeted therapies that may improve outcomes while being tolerable. Participants will receive one of three treatment combinations depending on their AML status untreated patients receive BP1001 with venetoclax and decitabine relapsed or refractory patients receive BP1001 with venetoclax and decitabine and a third group of relapsed or refractory patients who are resistant or intolerant to venetoclax receive BP1001 plus decitabine. The study uses an open-label design and will enroll about 19 participants per cohort initially, with potential expansion based on interim data review. The treatment involves multiple drug administrations to assess safety, pharmacokinetics, pharmacodynamics, and efficacy. During the study, participants will undergo regular assessments including bone marrow biopsies or aspirates to measure treatment response over 180 days. Safety will be monitored through evaluations up to 30 days post-treatment, along with pharmacokinetic studies of BP1001. Researchers will also assess minimal residual disease status and overall survival. Participants must meet specific health criteria and will provide informed consent before enrollment. The study is sponsored by Bio-Path Holdings, Inc. and is expected to continue until December 2028.

Researchers are evaluating a master screening protocol called Lung-MAP for patients with previously treated non-small cell lung cancer. This phase IIIII trial aims to develop a genomic screening method for large cancer populations and assign participants to appropriate sub-studies based on specific cancer biomarkers. The goal is to compare new targeted therapies designed to block cancer growth or spread with standard care, including sub-studies for patients not eligible for biomarker-driven treatments. The study involves screening patient specimens to determine eligibility for various biomarker-driven or non-matched sub-studies within the Lung-MAP umbrella protocol. This is a screening study without direct interventions instead, patients are assigned to different treatment sub-studies, each operating independently. The protocol also includes an optional ancillary study evaluating attitudes about the return of somatic mutation findings suggestive of germline mutations. Participants provide tumor tissue for biomarker testing, including molecular profiling and PD-L1 analysis, and may submit fresh biopsies and blood samples for circulating tumor DNA testing. Researchers will monitor screening success rates up to three years and collect patient and physician feedback on genetic findings. Participation involves signing informed consent, providing smoking history, and possibly completing surveys. The study duration and assessments vary depending on sub-study assignment and patient progression.

Researchers are evaluating the combination of durvalumab, gemcitabine, and cisplatin given before surgery to treat patients with high-risk liver cancer called resectable intrahepatic cholangiocarcinoma. This phase II study aims to see how well patients complete this pre-surgery therapy and proceed to surgical removal of the tumor. The study also investigates tumor response, survival rates, and the safety and feasibility of the treatment approach. Participants receive durvalumab intravenously on day 1, with gemcitabine and cisplatin given intravenously on days 1 and 8, repeated every 21 days for 4 cycles before surgery. After surgery, patients may continue the same regimen for up to 4 more cycles if disease progression or unacceptable side effects do not occur. During the study, patients undergo CT and MRI scans, blood sample collection, and tissue biopsies at screening and during treatment. Throughout the study, participants are closely monitored with imaging and laboratory tests to evaluate tumor response and detect side effects. After completing treatment, patients have follow-up visits every 3 months for 2 years, then every 6 months for 5 years, and yearly afterward to assess survival and cancer recurrence. The main outcome measured is the proportion of patients who complete the pre-surgery therapy and surgery, with additional measures including pathological response, margin status after surgery, radiological response, overall and relapse-free survival, and adverse events.

Researchers are evaluating APVO436 in combination with venetoclax and azacitidine for adults newly diagnosed with CD123-positive acute myeloid leukemia AML. This study is a Phase 1b2 open-label trial designed to find the safest and most effective dose of APVO436 when used with these drugs. The trial focuses on patients who have not received prior treatment and are considered ineligible for standard induction therapy due to factors like age or health conditions. Treatment involves five dose levels of APVO436 given with venetoclax and azacitidine. In the first 28-day cycle, patients receive four initial priming doses of APVO436 to reduce the risk of cytokine release syndrome, followed by doses administered on day 15 and in subsequent cycles. APVO436 is given by a 4-hour intravenous infusion, venetoclax is taken orally on days 1 through 22, and azacitidine is delivered by IV infusion on days 1 to 8 of each 28-day cycle. Participants will attend regular treatment cycles where safety, tolerability, and dosing effects are closely monitored through clinical assessments. Researchers will evaluate the maximum tolerated dose and measure treatment efficacy over approximately one year. The study includes detailed monitoring of side effects and patient response to the combination therapy, with ongoing follow-up until study completion.

Researchers are studying MEN2312, a lysine acetyltransferase 6 KAT6 inhibitor, in adults with advanced breast cancer that is not curable. This first-in-human, phase 1 study evaluates MEN2312 alone and in combination with elacestrant to understand its safety and determine the best dose. Participants have specific genetic alterations in their tumors and have received prior endocrine therapy and cyclin-dependent kinase 4 and 6 inhibitor treatment. Participants will be randomly assigned to receive either MEN2312 by itself or MEN2312 combined with elacestrant, both given as oral tablets. The study follows a sequential design and aims to identify dose-limiting toxicities and recommend the phase 2 dose over several months of treatment. This includes monitoring drug levels in the body and how the body processes the medications. During the study, participants will have regular assessments to monitor side effects, tumor response, and overall health. These include evaluating the number of dose-limiting toxicities within the first 28 days and tracking response rates, progression-free survival, and overall survival for up to nine months after treatment ends. Researchers will also measure drug concentration and excretion to better understand the treatment effects and safety over time.