This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Type: Interventional

Start Date: Jul 2026

open study

Background: CAR (Chimeric Antigen Receptor) T cell therapy is a type of cancer treatment in which a person s T cells (a type of immune cell) are changed in a laboratory to recognize and attack cancer cells. Researchers want to see if this treatment can help people with hairy cell leukemia (HCL). Objective: To test whether it is safe to give anti-CD22 CAR T cells to people with HCL. Eligibility: Adults ages 18 and older with HCL (classic or variant type) who have already had, are unable to receive, or have refused other standard treatments for their cancer. Design: Participants will be screened with the following: Medical history Physical exam Blood and urine tests Biopsy sample Electrocardiogram Echocardiogram Lung function tests Imaging scans Some screening tests will be repeated during the study. Participants may need to have a catheter placed in a large vein. Participants will have magnetic resonance imaging of the brain. Participants will have a neurologic evaluation and fill out questionnaires. Participants will have leukapheresis. Blood will be removed from the participant. A machine will divide whole blood into red cells, plasma, and lymphocytes. The lymphocytes will be collected. The remaining blood will be returned to the participant. Participants will get infusions of chemotherapy drugs. Participants will get an infusion of the anti-CD22 CAR T cells. They will stay at the hospital for 14 days. Then they will have visits twice a week for 1 month. After treatment, participants will be followed closely for 6 months, and then less frequently for at least 5 years. Then they will have long-term follow-up for 15 years.

Type: Interventional

Start Date: May 2022

open study

The investigators have designed a 5-week Family Navigator Plus (FN+) educational program to support foster parents in better understanding their youth's mental health needs and in finding mental health services. The purpose of this interventional study is to assess the the program's acceptability (e.g., satisfaction, barriers to participation, and burden), utility (e.g., how useful foster parents found the program to be), and feasibility (e.g., number referred who meet eligibility, time to complete assessments, self-reported feasibility), compared to Attention Control (AC). The investigators hypothesize that our program will be acceptable, useful, and feasible for foster parents of youth with mental health needs.

Type: Interventional

Start Date: Sep 2026

open study

This is a Phase III, multicenter, randomized, double-masked, vehicle-controlled, parallel-group clinical trial designed to evaluate the efficacy, safety, and tolerability of d-MAPPS™ Ophthalmic Solution in adult subjects with chronic ocular graft-versus-host disease (oGVHD). Eligible subjects will receive masked study treatment for 90 days, with the primary efficacy assessment performed at the Day 90 study visit.

Type: Interventional

Start Date: Sep 2026

open study

VY1706 first in human study in early Alzheimer's Disease is a multicenter dose escalation study

Type: Interventional

Start Date: Sep 2026

open study

This study aims to find out whether a study medicine called survodutide helps people control their blood sugar. Adults who live with type 2 diabetes and with a body mass index (BMI) of 23 kg/m2 or higher can join. The study has 3 parts. In each part the study compares survodutide with placebo. Survodutide is being developed to treat several health problems including type 2 diabetes. Placebo looks like survodutide but does not contain any medicine. Depending on a person's diabetes treatment, a person will be assigned either to - Part A: healthy eating and physical activity - Part B: diabetes tablets (no injection of insulin) - Part C: injection of insulin (with or without diabetes tablets) Participants are randomly put into 1 of 3 groups, which means the group is chosen by chance. Two groups of participants get survodutide at different dose levels, and the third group gets placebo as injection under the skin once a week. You have a 2 in 3 chance of getting survodutide. During the study, participants continue their regular diabetes treatment. Participants are in the study for about 1 year and 2 months. During this time, they attend up to 12 visits at the site and receive at least 9 phone calls. Study doctors regularly test participants' blood sugar by checking their HbA1c values and other laboratory test results. The study doctor also regularly checks participants' health and takes note of any changes. For each study part, the results will be compared between the survodutide and the placebo group to see whether the treatment works.

Type: Interventional

Start Date: Aug 2026

open study

The purpose of this study is to investigate the effect of HD on single oral dose PK, safety, and tolerability of balcinrenone/dapagliflozin to male and female participants with kidney failure on intermittent HD.

Type: Interventional

Start Date: Aug 2026

open study

Obesity is a condition where a person has excessive body fat, defined by a body mass index (BMI) of 30 kg/m2 or higher. The goal of this study is to evaluate the effect of various doses of ABBV-295 compared to placebo on reducing body weight at Week 32 in participants with obesity or overweight with weight-related comorbidities. ABBV-295 is an investigational long-acting amylin analog being developed for chronic weight management. Participants are placed into multiple treatment groups with different dosing paradigms of ABBV-295 or placebo. Participants will not know which treatment they are receiving, and there is a chance of being assigned to placebo. Adults with obesity or overweight (as defined by BMI) with weight-related comorbidities will be enrolled. Approximately 360 participants will be enrolled in the study at approximately 70 sites in the United States and Puerto Rico. Participants will receive subcutaneous (SC) injections of ABBV-295 solution for injection or matching placebo administered via pre-filled syringes for 52 weeks during the Double-Blind Period. There may be higher treatment burden for participants in this trial compared to their standard of care due to study procedures. Participants will attend regular visits during the study at a hospital or clinic. The effects of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Type: Interventional

Start Date: Aug 2026

open study

In this study, researchers will learn more about the use of felzartamab in participants with Graves' Disease, also known as GD. GD is an autoimmune disease, which means the body's immune system attacks its own healthy cells. In people with GD, the immune system produces abnormal antibodies, called thyroid-stimulating hormone receptor antibodies (TRAb), that attack the thyroid gland. This causes the thyroid to become too active and produce too much hormone, a condition called hyperthyroidism. Participants with GD are often treated with anti-thyroid drugs, or ATDs, which are medicines that help bring thyroid hormone levels back to normal. Felzartamab is designed to target certain immune cells that produce the abnormal TRAb antibodies. The main goal of the study is to learn whether felzartamab can help bring thyroid hormone levels back to normal and allow participants to stop taking ATDs. Participants will receive either felzartamab or placebo during the study. A placebo looks like the study drug but contains no real medicine. The main question that researchers want to answer is: • How many participants have normal thyroid levels without receiving any ATD medicine at Week 24? Researchers will also learn more about the safety of felzartamab and how the body processes the drug. The study will be done as follows: - Participants will be screened to check if they can join the study. - This is a double-blind study, which means neither the participants, study doctor, or site staff will know if participants are receiving felzartamab or a placebo. - Participants will be placed into 1 of 3 groups. Two groups will receive felzartamab while the other receives placebo. - Participants will receive felzartamab or placebo as intravenous (IV) infusions, which are slow injections into a vein using a needle. The Treatment Period will last 20 weeks. - During the Treatment Period, the study doctor may gradually lower the dose of the ATD if a participant's thyroid levels become normal. - Afterwards, participants will enter a follow-up period which will last 12 weeks. - In total, participants will have 22 study visits. Participants will stay in the study for about 9 months (up to 36 weeks).

Type: Interventional

Start Date: Jul 2026

open study

Quality improvement (QI) initiative to improve uptake and utilization of Connected MOM, particularly among those with limited access to health care

Type: Interventional

Start Date: Jul 2026

open study

This is a phase 1 study, randomized, double-blind, placebo-controlled, 2 treatment, 2 period crossover study to evaluate the pharmacokinetics, safety, and tolerability of a single inhaled dose of ASY202 in adults with stable asthma. Following screening, eligible participants will be enrolled and randomized to one of two treatment sequences. Participants will receive a single inhaled dose of ASY202 and a single inhaled dose of placebo, each administered once during separate study periods. During each treatment period, subjects will stay in the clinical research unit for safety monitoring and PK assessments, including serial blood sampling, spirometry, vital signs, clinical laboratory tests, and ECGs. Each period will include post dose assessments for up to 24 hours. Participants will return for their next treatment period after a washout interval to ensure complete clearance of study medication. All subjects will receive both treatments over the course of the study. A follow-up visit will occur after the last dose to assess ongoing safety and tolerability.

Type: Interventional

Start Date: May 2026

open study

The goal of this observational cohort study is to learn how toxin and occupational exposures, germline genetic variation, and immune dysregulation relate to B-cell non-Hodgkin lymphoma among active-duty service members and other Military Health System beneficiaries. The main questions are whether specific exposures and germline variants are associated with B-cell NHL subtype, immune dysfunction, and clinical outcomes. Participants will complete exposure and medical-history surveys, provide biospecimens for immune and genomic testing, and may be followed annually for up to 3 years.

Type: Observational

Start Date: May 2025

open study

This study is testing an investigational inhaled migraine medication to see how well it works, how safe it is, and how well people tolerate it. Adults with migraine will receive both the study medication (ASY202) and a placebo (inactive treatment) at different times during the study. Neither participants nor study staff will know which treatment is given at the time. The medication is taken using a handheld dry powder inhaler to treat migraine attacks when they occur. Following screening, eligible participants will be enrolled and randomized to one of two treatments sequences i.e. one treatment sequence will receive ASY202 in treatment period 1 followed by placebo in treatment period 2 and other treatment sequence will receive placebo in treatment period 1 followed by ASY202 in treatment period 2. The study lasts about 16 weeks and includes a screening period, two treatment periods (with a minimum of 7 days washout period between the treatment periods), and a safety follow-up visit.

Type: Interventional

Start Date: May 2026

open study

This study is a randomized, double-blind, placebo and active-controlled, multicenter trial conducted to evaluate whether PP-01 mitigates the withdrawal symptoms associated with discontinuing cannabis in participants with moderate to severe cannabis use disorder (CUD). Study participants will receive PP-01, nabilone, or placebo every day for 34 days. The total study duration will be approximately 78 days, including screening and a one-week inpatient stay. Following the initial inpatient portion of the study, participants will return to the clinic for six clinic visits and complete two telemedicine appointments. Participants will complete daily symptom diaries and other study-related questionnaires. Participants who complete the core study may be eligible to participate in a repeat dosing extension study if they meet required criteria.

Type: Interventional

Start Date: Jun 2026

open study

This study will evaluate the efficacy and safety of EVO756 against placebo in adults with migraine.

Type: Interventional

Start Date: Jul 2026

open study

The purpose of this study is to see how LY3439539 affects certain proteins found in the spinal fluid of participants with Alzheimer's disease. Participation in the study will last approximately 9 months with visits about once a month.

Type: Interventional

Start Date: May 2026

open study

Diabetic Retinopathy (DR) is a common eye condition caused by diabetes, where high blood sugar levels damage the blood vessels in the back part of the eye (called the retina). Over time, this damage can lead to vision problems and even blindness if not treated. This study will assess surabgene lomparvovec (sura-vec) as a potential one-time gene therapy administered in the suprachoroidal space (SCS) for the treatment of diabetic retinopathy (DR) and prevention of vision-threatening events (VTEs) in participants with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME). This study will consist of 3 portions: a Phase 2b portion, a Phase 3 portion, and a bilateral treatment portion. Approximately 576 adult participants will be enrolled in the study across multiple sites in the United States and Puerto Rico. In the Phase 2b and Phase 3 portions, participants will be randomized to different groups to receive sura-vec and prophylactic steroids or sham and artificial tears in their study eye. If assigned to sham, participants will be given an opportunity to cross over and receive treatment with sura-vec. In the bilateral treatment portion, participants will be enrolled to receive sura-vec and prophylactic steroids in both eyes. In all 3 portions, follow-up in the study will continue through 5 years following administration of sura-vec in each eye. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Type: Interventional

Start Date: Jun 2026

open study

The purpose of this study is to evaluate the safety and efficacy of atebimetinib in combination with modified GnP compared with SOC GnP alone.

Type: Interventional

Start Date: Jun 2026

open study

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).

Type: Interventional

Start Date: May 2026

open study

Researchers are looking for new ways to treat 2 types of non-Hodgkin lymphoma (NHL) called follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL). FL is a slow-growing type of NHL. DLBCL is a fast-growing type of NHL. NHL is a cancer in the lymphatic system that causes swollen lymph nodes. The lymphatic system is part of the immune system. In this study, researchers want to learn if MK-1045 can treat FL and DLBCL. MK-1045 is a study treatment that is an immunotherapy, which helps the immune system fight cancer. The goals of this study are to learn how safe MK-1045 is and if people tolerate it. Researchers also want to see if FL and DLBCL respond (the cancer gets smaller or goes away) to treatment.

Type: Interventional

Start Date: May 2026

open study

An open-label extension (OLE) study to evaluate the long-term safety and efficacy of ORKA-001 in adult participants with moderate-to-severe plaque psoriasis, who previously participated in an Oruka Therapeutics sponsored study.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this clinical study is to find out if cadonilimab or ateganosine plus cadonilimab is effective and safe in treating resectable hepatocellular carcinoma (HCC).

Type: Interventional

Start Date: Sep 2026

open study

A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD

Type: Interventional

Start Date: Jul 2026

open study

This phase II trial tests whether taking ulipristal acetate prior to a mammogram decreases breast density for people with a history of dense breast tissue. One limitation to breast cancer screening with mammography is dense breast tissue, which decreases the sensitivity of screening as breast density masks cancer and precancerous lesions. Ulipristal acetate lowers the amount of progesterone made by the body which may temporarily decrease the density of the breast tissue, allowing for a more accurate mammogram for people with dense breast tissue.

Type: Interventional

Start Date: Aug 2026

open study

This registration covers only Aim 1b, which focuses on testing usability. The study will include 20 participants who are breast cancer patients. Participants will be observed using the tool on their mobile devices, and their experience will be evaluated using a "think-aloud" method to capture their thoughts and reactions. Interviews will include open-ended questions about the content and format of the decision aid, with follow-up prompts to gather feedback on design elements (length, order, graphics), comprehension, ease of use, acceptance, and engagement with the tool. Knowledge will be measured using the Decision Quality Instrument before and after using the tool. Results will be analyzed to develop recommendations for the final version of the tool.

Type: Interventional

Start Date: Oct 2025

open study