This open-label extension study will provide post-trial access to pelacarsen (TQJ230) to participants who have successfully completed the double-blind parent study (CTQJ230A12301).

Type: Interventional

Start Date: May 2026

open study

The goal of this clinical trial is to learn about the safety and recommended dose of TRI-611 when administered to adults with ALK-positive non-small cell lung cancer (NSCLC). The trial will also evaluate the antitumor activity of TRI-611 in adults with ALK-positive NSCLC. The study will be conducted in two parts. The first part will examine different doses of TRI-611. The second part will look at how well TRI-611 works on ALK-positive NSCLC when administered to three groups of participants that differ based on what type of prior therapy they have received. In this study participants will: - Take TRI-611 on a continued basis, provided it is well-tolerated, for as long as their disease is not progressing - Visit the clinic approximately seven times in the first 3 months and then just once at the start of each 28-day cycle thereafter - Keep a diary of each time they take the study medication

Type: Interventional

Start Date: Mar 2026

open study

The purpose of this study is to evaluate the safety, tolerability, and efficacy of Pumitamig alone or in combination with Ipilimumab or Cabozantinib in participants with advanced Renal Cell Carcinoma (RCC)

Type: Interventional

Start Date: Mar 2026

open study

Researchers are looking for new ways to treat extensive-stage small cell lung cancer (ES-SCLC). ES-SCLC is a type of lung cancer that has spread throughout the lung, to the other lung, or to other parts of the body. A standard (usual) treatment for ES-SCLC uses both chemotherapy and immunotherapy. - Chemotherapy is a treatment that works to destroy cancer cells or stop them from growing. - Immunotherapy is a treatment that helps the immune system fight cancer. Gocatamig and I-DXd (short for ifinatamab deruxtecan) are study medicines. Researchers want to know if giving gocatamig and I-DXd together can treat ES-SCLC. Researchers will also look at giving the study medicines with standard treatment. Gocatamig is a T-cell engager therapy. I-DXd is an antibody drug conjugate. - T-cell engager therapy is a certain type of immunotherapy that uses T-cells to find and destroy cancer cells. - A T-cell is a type of white blood cell, which are cells that help the body fight infection. - An antibody drug conjugate (ADC) is a treatment that attaches to a protein on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn: - About the safety of combining gocatamig and I-DXd and if people tolerate them together - If people who receive gocatamig and I-DXd have ES-SCLC respond, which means the cancer gets smaller or goes away

Type: Interventional

Start Date: Jan 2026

open study

This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).

Type: Interventional

Start Date: Oct 2025

open study

Researchers are looking for new ways to treat types of breast cancer that are both: - High-risk, which means the cancer may have a higher chance of getting worse or coming back after treatment - Early-stage, which means the cancer is in the breast or the lymph nodes around the breast The 2 types of breast cancer in this study are triple-negative breast cancer (TNBC) and hormone receptor (HR)-low positive/human epidermal growth factor receptor-2 (HER2) negative breast cancer. These cancers have zero or a low amount of a protein called HER2 and other proteins that attach to the hormones estrogen or progesterone. Sacituzumab tirumotecan (also known as sac-TMT or MK-2870), the study medicine, is a type of targeted therapy. A targeted therapy is a treatment that works to control how specific types of cancer cells grow and spread. The main goals of this study are to learn if people who receive sac-TMT, pembrolizumab, and chemotherapy: - Have fewer cancer cells found in the tumors and lymph nodes removed during surgery compared to those who receive only pembrolizumab and chemotherapy - Live longer without the cancer growing, spreading, or coming back compared to people who receive only pembrolizumab with chemotherapy

Type: Interventional

Start Date: Jun 2025

open study

The overall goal of the randomized control trial (RCT) will be to evaluate the efficacy of modifications to a web-based tool for patient decision-making regarding return of genomic results that will more closely focus on rare cancers. Participants will be given access to a web-based decision aid (or a standard control) that guides participants in making decisions about what type of genomic results they would like to receive from testing performed in the PE-CGS study (NCT06340646).

Type: Interventional

Start Date: Apr 2025

open study

This phase III trial compares 6 months of human epidermal growth factor receptor 2 (HER2)-targeted therapy to 12 months of HER2-targeted therapy for the treatment of HER2-positive (+) breast cancer in patients that had a pathologic complete response (pCR) after preoperative (neoadjuvant) chemotherapy with trastuzumab. Trastuzumab and pertuzumab are monoclonal antibodies and forms of targeted therapy that attach to a receptor protein called HER2. HER2 is found on some cancer cells. When trastuzumab or pertuzumab attach to HER2, the signals that tell the cells to grow are blocked and the tumor cell may be marked for destruction by the body's immune system. Giving 6 months of HER2-targeted therapy may work better than giving 12 months for the treatment of HER2+ breast cancer in patients that had a pCR after neoadjuvant chemotherapy with trastuzumab.

Type: Interventional

Start Date: Sep 2025

open study

This is a four-part study evaluating the safety and efficacy of a PSMA-directed radioantibody (rosopatamab tetraxetan, conjugated to either In-111 or Ac-225). Part 1 will consist of one administration of In-111-rosopatamab tetraxetan to characterize the biodistribution of the radioantibody to target organs and prostate cancer lesions. Participants then will be enrolled into either Part 2 (Dose Optimization) or Part 3 (Dose Escalation and Expansion) depending on their prior treatment history. Part 4 will be an extended regimen in dose escalation and expansion. Participants qualifying for Part 2 will be randomized to receive Ac-225 rosopatamab tetraxetan in a single fractionated cycle (dose administration on Day 1 and Day 15) at either 45 or 60 kBq/Kg. Participants qualifying for Part 3 must have received prior Lu-177-PSMA-radioligand therapy and will receive Ac-225 rosopatamab tetraxetan in a single fractionated cycle at 45, 55, or 60 kBq/Kg. Dose limiting toxicities (DLTs) will be monitored in Part 3 to determine the recommended phase 2 dose (RP2D), and the study may enroll additional participants to be treated with the RP2D dose level. Participants qualifying for Part 4 will initially receive two doses (dose administration on Day 1 and Day 15) at the dose level selected in Part 2, followed by a single third dose of Ac-225 rosopatamab tetraxetan administered approximately 12 weeks after completion of the Part 2 fractionated dosing regimen. The starting dose level for Part 4 will be 22 kBq/kg (or fixed activity equivalent). Dose limiting toxicities (DLTs) will be monitored following administration of the third dose in Part 4 to determine the recommended phase 2 dose (RP2D) of a third dose of Ac-225 rosopatamab tetraxetan. Participants enrolled into any part will attend study visits which will include blood samples, electrocardiogram (ECG), radiographic imaging, and physical examinations along with other assessments.

Type: Interventional

Start Date: Aug 2024

open study

This phase III trial compares the effect of giving triptorelin vs no triptorelin in preventing ovarian damage in adolescents and young adults (AYAs) with cancer receiving chemotherapy with an alkylating agents. Alkylating agents are part of standard chemotherapy, but may cause damage to the ovaries. If the ovaries are not working well or completely shut down, then it will be difficult or impossible to get pregnant in the future. Triptorelin works by blocking certain hormones and causing the ovaries to slow down or pause normal activity. The triptorelin used in this study stays active in the body for 24 weeks or about 6 months after a dose is given. After triptorelin is cleared from the body, the ovaries resume normal activities. Adding triptorelin before the start of chemotherapy treatment may reduce the chances of damage to the ovaries.

Type: Interventional

Start Date: Feb 2025

open study

With modern therapy, the survival rate for pediatric brain tumor patients has significantly improved, with over 70% of patients surviving their disease. However, this progress often comes at the cost of substantial morbidity, with cognitive deficits being the primary obstacle to independent living. Robust predictors of cognitive decline and a comprehensive understanding of the underlying mechanisms of cognitive injury remain elusive. This study will prospectively investigate alterations in brain resting state networks following radiation therapy using functional imaging. The hypothesis is that radiation therapy leads to dose-dependent alterations in functional connectivity in the networks associated with higher level cognition, ultimately leading to cognitive decline.

Type: Observational

Start Date: Jul 2024

open study

Aim 1. To determine the factors contributing to pain in patients with chronic pelvic pain with and without endometriosis Aim 2. To determine the changes following endometriosis lesion removal surgery in pain and sex hormone levels. Aim 3. To identify factors predicting clinical pain reduction after lesion removal surgery. Additional exploratory aims might compare subgroups of patients such as patients with vs. without endometriosis, with vs. without additional chronic pain syndromes, and with vs. without hormonal treatment. In addition, the role of lesion-specific immune signatures and psychological factors on pain will be explored. Patients with pelvic pain potentially due to endometriosis will complete questionnaires including social, health, behavioral, and psychological questionnaires. Patients will also complete a 2.5 hours study visit of psychophysical assessments of thermal and pressure stimuli. In addition, blood, urine, and saliva samples will be collected for hormonal, immune, and genetic analyses. Additional samples might be collected and stored for future analyses. For some participants, these procedures will be completed several times, including before the surgery as well as 3 months, 6 months and 1 year after surgery. After the surgery, surveys assessing pain and other symptoms will be sent every week on the first month and then monthly for 1 year. In addition, during the surgery, biopsies will be collected and analyzed to determine hormonal, immune and genetic factors. For adolescents, a parent/legal guardian will be asked to assist in completing some of the health-related surveys (i.e., physical developmental survey, health survey, migraine history survey).

Type: Observational

Start Date: Sep 2023

open study

The main aim of this study is to learn about the effect of treatment with vedolizumab IV (vedolizumab) together with tofacitinib in adults with moderate and severe ulcerative colitis (UC). Another aim is to learn about treatment with Vedolizumab alone after the double treatment. All participants will receive vedolizumab together with tofacitinib for 8 weeks and will be checked for response. Participants who show a response to the treatment after 8 weeks will be treated with vedolizumab alone for an additional 44 weeks. Each participant will be followed up for at least 26 weeks after the last dose of vedolizumab.

Type: Interventional

Start Date: Jun 2024

open study

The goals of this research study are to: 1) understand why some people with obesity are protected from developing conditions such as type 2 diabetes and cardiovascular disease while others are more likely to develop obesity-related conditions; 2) assess the effect of small extracellular vesicles (sEVs also called exosomes), obtained from human participants, on metabolic function in cultured cells and in mice.

Type: Interventional

Start Date: Jun 2023

open study

A study to evaluate the effect of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism (SE) in patients with Atrial Fibrillation (AF) who have been deemed by their responsible physicians or by their own decision to be unsuitable for oral anticoagulation therapy.

Type: Interventional

Start Date: Dec 2022

open study

This is a multicenter trial to establish the efficacy of cooling and the optimal duration of induced hypothermia for neuroprotection in pediatric comatose survivors of cardiac arrest. The study team hypothesizes that longer durations of cooling may improve either the proportion of children that attain a good neurobehavioral recovery or may result in better recovery among the proportion already categorized as having a good outcome.

Type: Interventional

Start Date: Aug 2022

open study

This phase II trial studies the good and bad effects of the combination of drugs called cabozantinib and nivolumab in treating patients with melanoma or squamous cell head and neck cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). Cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. This trial may help doctors determine how quickly patients can be divided into groups based on biomarkers in their tumors. A biomarker is a biological molecule found in the blood, other body fluids, or in tissues that is a sign of a normal or abnormal process or a sign of a condition or disease. A biomarker may be used to see how well the body responds to a treatment for a disease or condition. The two biomarkers that this trial is studying are "tumor mutational burden" and "tumor inflammation signature." Another purpose of this trial is to help doctors learn if cabozantinib and nivolumab shrink or stabilize the cancer, and whether patients respond differently to the combination depending on the status of the biomarkers.

Type: Interventional

Start Date: Dec 2022

open study

DALY II USA is a phase II, multi-center, single arm study to evaluate the efficacy, safety, and pharmacokinetics of zamtocabtagene autoleucel (MB-CART2019.1) in patients with relapsed and/or refractory B cell lymphoma (BCL). Cohorts include subjects with diffuse large B-cell lymphoma (DLBCL) after receiving at least 2 lines of therapy, primary or secondary central nervous system (CNS) lymphoma (PCNSL) and (SCNSL) after receiving at least one line of therapy, mantle cell lymphoma (MCL) and Richter's transformation (RT) after receiving at least one line of therapy, and DLBCL transplant-ineligible after receiving at least one line of therapy.

Type: Interventional

Start Date: May 2021

open study

Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases....

Type: Observational

Start Date: Sep 2015

open study

The sequential trial compares recovery and mHealth engagement outcomes among Black participants who participated in the mHealth intervention uMAT-R or uMAT-R + enhanced community engagement. The mHealth intervention seeks to support people in various stages of recovery who are also seeking other forms of substance use treatment. Participants are recruited from community-based substance use treatment facilities and recovery services. Participants must be adults who have or are currently using opioids and/or stimulants.

Type: Interventional

Start Date: Aug 2025

open study

This is a prospective specimen collection study evaluating the feasibility of using the ChromoSeq® assay for upfront classification in a real-time clinical setting of pediatric and young adult acute lymphoid leukemia (ALL) patients. Sixty patients will undergo collections of bone marrow and/or peripheral blood for the ChromoSeq® assay at time of initial workup, and the patients will then be followed for clinical outcomes for up to 65 months.

Type: Observational

Start Date: Jun 2026

open study

The goal of this clinical trial is to learn how tests undertaken by people at high risk of developing psychosis (aged 17 to 30 years old) change when those people are given the study drug MT1988 daily for 8 weeks. This will help identify tests that could be used in later trials developing treatments for symptoms in people at high risk of developing psychosis, to measure whether those new treatments are effective. The main question this trial aims to answer is: Can any of the tests (biomarkers) used in this study detect changes in participants dosed with one of two different dose levels of MT1988? Researchers will compare the results from two dose levels of MT1988 to a placebo group. Researchers do not expect to see the test results change in participants taking placebo and this will be compared to changes expected in test results in participants taking MT1988. Participants will: - take a dose of MT1988 or placebo twice per day for 8 weeks - attend clinic appointments every two weeks to undertake assessments - report any side effects they experience to the researchers

Type: Interventional

Start Date: Mar 2026

open study

This phase II trial tests the addition of chemotherapy, with carboplatin and paclitaxel, or chemo-immunotherapy, with carboplatin, paclitaxel and cemiplimab to standard salvage surgery followed by post operative radiation therapy and cisplatin for high risk patients, for the treatment of patients with PD-L1 positive head and neck squamous cell carcinoma that has come back and spread to nearby tissue or lymph nodes after a period of improvement (locally recurrent) or is persistent. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of cancer cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops cancer cells from growing and dividing and may kill them. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Salvage surgery is surgery that takes place to remove tumor tissue after a failure of other treatment. High risk patients also receive radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of cancer cells. Adding chemotherapy or chemo-immunotherapy to standard salvage surgery may kill more tumor cells than salvage surgery alone in patients with PD-L1 positive locally recurrent or persistent head and neck squamous cell carcinoma.

Type: Interventional

Start Date: Apr 2026

open study

This phase III trial compares the effect of immunotherapy (IO) with stereotactic body radiation therapy (SBRT) to IO alone in treating patients with liver cancer (hepatocellular cancer) that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). The usual approach is treatment with IO-based drug combinations, such as atezolizumab and bevacizumab, durvalumab and tremelimumab, or ipilimumab and nivolumab. IO with monoclonal antibodies, such as durvalumab, tremelimumab, atezolizumab, nivolumab and ipilimumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Bevacizumab is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor cells. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. SBRT is a type of external radiation therapy that uses special equipment to position a patient and precisely deliver radiation to tumors in the body (except the brain). The total dose of radiation is divided into smaller doses given over several days. This type of radiation therapy helps spare normal tissue. Giving IO with SBRT may be more effective than IO alone in helping patients with advanced hepatocellular cancer live longer.

Type: Interventional

Start Date: Feb 2026

open study

In this study, patients who are scheduled for breast reconstruction consultation will be randomized into the intervention group (ChatGPT-generated patient education regarding possible reconstruction options) or the control group (usual patient education). All patients will complete a survey following their in-person consultation to assess their experience and overall satisfaction with the consultation process. Additionally, participating surgeons will complete a separate survey to evaluate their consultation experience, satisfaction, and to assess the accuracy and clinical utility of the ChatGPT-generated patient education materials. The surveys are designed to gather information on patient characteristics, organizational health literacy according to Brega et al. Other survey questions have been designed to meet the outcomes of this study and have not been based on previously published surveys.

Type: Interventional

Start Date: Jun 2025

open study