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A Phase I Study of Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Other Hematologic Malignancies
Background: Blood cancers (such as leukemias) can be hard to treat, especially if they have mutations in the TP53 or RAS genes. These mutations can cause the …
RESTORE Study R61 Pilot Phase: Recovery and Engagement for Stimulant Users on Re-entry
Justice-involved individuals face disproportionately high rates of stimulant use disorder and HIV, along with disrupted access to HIV treatment and prevention …
SMART-r: Substance Monitoring and Active Relapse Tracking Repository
Background: About 1.5 million adults in the US enter alcohol or substance use treatment programs each year. Unfortunately, more than half of patients do not f…
The Mechanism of Action Underlying Ketamine's Antidepressant Effects: An Investigation of the AMPA Throughput Theory in Patients With Treatment-Resistant Major Depression
Background: Most drugs that treat mood disorders take a long time to work. Ketamine works within hours. A dose can last for a week or more. Certain receptors …
Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource (ARPKD Database Study)
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and …
Phase II, Randomized, Double-Blind, Placebo-Controlled Trial of Omega-3 Fatty Acid (O3FA) Supplementation for Adult and Juvenile Dermatomyositis (DM/JDM)
Dermatomyositis (DM) is a rare autoimmune disease that causes muscle weakness, skin rashes, and other symptoms. Researchers think both genetic and environmenta…
An Open-label Clinical Trial Investigating the Long-term Safety of Buntanetap in Treating Participants With Parkinson's Disease
This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study. This study will be conduct…
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Confirm Efficacy and Safety of MH002 and to Assess the Effect of Dose in Patients With Mild-to- Moderate Ulcerative Colitis Insufficiently Controlled With 5-Aminosalicylic Acid
The main goal of the study is to check if MH002 works and is safe to use. In a previous study in 45 patients with Ulcerative Colitis, MH002 was found to have f…
NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors
Background: All childhood cancers are rare, but some are called very rare. Very rare cancers are diagnosed in 2 or fewer out of 1 million people each year. Re…
Randomized Controlled Trial of Video-Laryngoscopy Intervention or Direct Laryngoscopy for Delivery of Less Invasive Surfactant Administration for Premature Infants
Many preterm babies born between 22-28+6 weeks' estimated gestational age (EGA) need surfactant, a medicine that helps the lungs. The goal of the study is to c…
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Repeat Subcutaneous Doses of FB825 in Adults With Moderate-to-Severe Atopic Dermatitis
This is a randomized, placebo-controlled and double-blind study to evaluate the efficacy, pharmacokinetics, and Safety of repeat subcutaneous doses of FB825 in…
The Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia: an Ethnographic Study
Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these…