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Browse 40,629 clinical trials for rheumatoid arthritis. Find studies that match your criteria and connect with research centers.
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NCT06424301
Retinoblastoma (RB) is the most common intraocular malignancy in children, accounting for approximately 11% of all cancers diagnosed in children under the age of one. Although its incidence is relatively low-about 1 in 15,000 to 20,000 live births-RB has a high risk of intracranial metastasis via the optic nerve, often leading to poor prognosis in advanced cases. Recent advances in administration routes, such as intravitreal and intra-arterial chemotherapy, have significantly improved eye preservation rates. However, these strategies are limited by cumulative retinal toxicity and drug resistance. In refractory cases, enucleation remains the only definitive treatment to prevent extraocular spread and death. In light of these challenges, current research efforts are focused on developing novel targeted therapies that enhance anti-tumor efficacy while minimizing local toxicity. In this context, we introduce a first-in-class siRNA-based drug targeting NUDT21, which promotes tumor regression by modulating the 3'UTR tail of SMC1A, thereby suppressing tumor cell proliferation. Importantly, the siRNA drug selectively targets tumor cells, offering a favorable safety profile compared to conventional chemotherapeutic regimens. Given that both the target (NUDT21) and the mode of administration (intraocular siRNA injection) are novel in retinoblastoma treatment, there is an urgent need for early-phase investigator-initiated clinical research. This study is therefore designed to assess the short-term safety and preliminary efficacy of NUDT21 siRNA in patients with refractory retinoblastoma, and to provide an evidence base for future large-scale clinical trials.
NCT06467136
Treatment of post-stroke apraxia of speech (AOS) requires frequent and ongoing practice with a speech-language pathologist to facilitate lasting behavioral change, which is costly and, therefore, inaccessible to many patients. Thus, there is a critical need to identify novel, cost-effective ways to supplement speech therapy to increase opportunities for practice and optimize treatment outcomes. Our long-term goal is to develop an effective, home-practice, computer-based, motor imagery protocol Motor Imagery for Treatment Enhancement and Efficacy (MI-TEE) which will serve as an adjunct to routine speech therapy to optimize treatment response in persons with AOS. The overall objectives of this application are to (i) evaluate the acceptability and feasibility of MI-TEE as a home practice program and (ii) determine the efficacy of MI-TEE with speech therapy, compared to speech therapy alone, in improving speech production in people with AOS. Our central hypothesis is that MI-TEE will be an accessible, feasible, and efficacious adjunct to speech therapy. To attain our objectives, the following specific aims will be pursued using two single-subject experimental designs with multiple baselines across participants (n=18): 1) Evaluate the acceptability and feasibility of MI-TEE as an adjunct to speech therapy for the rehabilitation of AOS; and 2) Compare the efficacy of adjunctive MI-TEE plus standard speech therapy to standard speech therapy alone. Under the first aim, observational data, surveys, and semi-structured interviews will be employed to assess the acceptability (perceived satisfaction, appropriateness, and intent to continue use) and feasibility (recruitment, retention, and intervention adherence rates) of MI-TEE. For the second aim, accuracy of articulation for trained words and untrained words (generalization) will be measured pre-treatment, repeatedly during the treatment phase, and post-treatment. Improvements in speech accuracy will be documented using a binary scoring system (correct/incorrect). Multilevel analyses will be used to address rate of acquisition, overall change, and response variation across participants.