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CF is caused by mutations in the gene that encodes the 'Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)' channel. To re-establish the function of this complex chloride channel, typically two to three drug modes of action are needed. To date, clinical studies of CFTR mo…

1\. To assess treatment efficacy with YCJ-01 by changes in the number of epileptic seizures in patients with refractory epilepsy secondary to Tuberous Sclerosis Complex. 2. To assess the safety of the treatment with YCJ-01 in patients with refractory epilepsy secondary to Tuberou…

The goal of this clinical trial is to evaluate the efficacy, safety and pharmacokinetics of elamipretide in subjects with dry age-related macular degeneration (AMD). The main questions it aims to answer are: what is the rate of change in the macular area of photoreceptor loss in …

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Mosquito-borne diseases cause suffering for hundreds of millions of people and claim more than 700,000 lives yearly. Diseases such as dengue, Zika, and chikungunya cause symptoms similar to malaria and are endemic in many parts of the world, yet there are no treatments for them n…

This is a single-center, double-blind, randomized trial. Patients with relapsed or refractory acute B-lymphoblastic leukemia(r/r B-ALL) experiencing early functional exhaustion of CAR-T cells will be randomly allocated into three groups: the control cell group, the CIK treatment …

The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).

This is a study evaluating the efficacy and safety of calderasib with pembrolizumab as first-line treatment in participants with locally advanced or metastatic non-small cell lung cancer (NSCLC) with identified Kirsten rat sarcoma viral oncogene homolog G12C (KRAS G12C) mutation …

The purpose of this platform study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of novel RAS(ON) inhibitors combined with Standard(s) of Care (SOC) or with novel agents. The current subprotocols include the following: Subpro…

This trial is a registered phase III, randomized, open-label, multicenter study to evaluate the efficacy and safety of BL-B01D1 in patients with locally advanced or metastatic non-squamous non-small cell lung cancer with EGFR-sensitive mutations after EGFR-TKI failure.

This phase II trial tests how well the combination of WP1066 and radiation therapy works in treating newly diagnosed glioblastoma. Glioblastoma is difficult to treat effectively because the cells within the tumor vary widely and are controlled by factors within and around the tum…

This study was designed to compare the efficacy and safety of I-DXd with treatment of physician's choice in participants with relapsed small cell lung cancer (SCLC).

The overall objective of the RESTORATiVE303 study is to evaluate the safety and the Clostridioides difficile infection (CDI) recurrence rate at Week 8 in participants who receive a 14-day course of VE303 or matching placebo. The objectives and endpoints are identical for Stage 1 …

This trial is a registered phase III, randomized, open-label, multicenter study to evaluate the efficacy and safety of BL-B01D1 in patients with locally advanced or metastatic EGFR wild-type non-small cell lung cancer after failure of anti-PD-1/PD-L1 monoclonal antibodies and pla…

This is a prospective, open-labled, randomized controlled study to assess efficacy and safety of treatment with Sintilimab (PD-1 antibody) combined Peg-IFNα-2b in CHB patients on stable NAs treatment.

The goal of this clinical trial is to evaluate CHM-2101, an autologous CDH17 CAR T-cell therapy for the treatment of advanced gastrointestinal (GI) cancers that are relapsed or refractory to at least 1 standard treatment regimen in the metastatic or locally advanced setting.

This trial represents a single institution phase I/II pilot study with the primary objective of establishing the safety and feasibility of generating and infusing ML NK cells after TCRαβ haplo-HCT.

Background: Endometrial cancer (EC) of the uterus is becoming more common in the US. Sometimes EC often has increased levels of a protein called HER2. Cancers with HER2 tend to be more aggressive and have poorer outcomes. Objective: To test 2 study drugs-a vaccine that targets…

ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1A), aged ≥48 months to \<18 years (Part 1B), and aged ≥6 to \<48 months (Part 2). Part 1A follows an …

The purpose of this study is long-term evaluation of long-acting injectable cabotegravir (CAB LA) for HIV pre-exposure prophylaxis (PrEP) in eligible participants who have completed DAIDS (Division of AIDS) sponsored studies HPTN 083 and HPTN 084 and associated sub-studies. Parti…