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NCT06910358
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are: * Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP. * How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment. Researchers will compare bitopertin to a placebo look-alike substance that contains no drug. Participants will complete daily questionnaires and attend study visits for assessments.
NCT03559049
This study is a multi-center, Phase I/II, single arm trial to assess the safety and efficacy of the combination of oral rucaparib plus intravenous pembrolizumab as maintenance therapy in patients with stage IV non-squamous non-small cell lung cancer (NSCLC) without progressive disease (PD), as confirmed on CT scans, after induction therapy with carboplatin/pemetrexed/pembrolizumab (CPP) triplet therapy.
NCT04378790
A randomized trial to determine whether simultaneous treatment with spectacles and patching has an equivalent VA outcome compared with sequential treatment, first with spectacles alone followed by patching (if needed), for previously untreated amblyopia in children 3 to \<13 years of age.
NCT06380517
In children 4 to 7 years of age, to determine if treatment with 1 hour per day 6 days per week of watching dichoptic movies/shows wearing the Luminopia headset is non-inferior to treatment with 2 hours of patching per day 7 days per week with respect to change in amblyopic eye distance VA from randomization to 26 weeks.
NCT07227857
Study CL1-230815-001 (KANDLE) is a Phase Ib/II, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in pediatric participants with KCNT1-related Developmental Epileptic Encephalopathy. To participate in the study, participants must have a diagnosis of Developmental Epileptic Encephalopathy due to a documented pathogenic or likely pathogenic variant in KCNT1 (to be confirmed by central genetic testing at the screening visit). The study consists of a screening period followed by two consecutive interventional parts. Part 1 will evaluate multiple ascending doses of S230815. Part 2 is a long-term treatment extension for participants who have completed Part 1. Participants will seamlessly roll-over from Part 1 to Part 2, resuming the same cohort as they were assigned in Part 1, and will receive S230815 for a maximum of 72 weeks.
NCT06014086
The goal of this clinical trial is to evaluate the safety and tolerability of intratumoral injections of PH-762 in squamous cell carcinoma, melanoma, or Merkel cell carcinomas of the skin, to understand what the body does to the PH-762, and to observe how the tumor responds to the drug. Participants will receive four injections of PH-762 at weekly intervals, into a single tumor, followed by surgical removal of the tumor approximately two weeks later.
NCT06627647
The purpose of ARTEMIDE-Lung03 is to evaluate the efficacy and safety of rilvegostomig compared to pembrolizumab, both in combination with platinum-based doublet chemotherapy, as a first-line treatment of patients with non-squamous mNSCLC whose tumors express PD-L1.
NCT06584357
Bio-Hermes-002 is a 120-day cross-sectional study that will result in a blood, CSF, retinal, digital, MRI, and PET brain imaging biomarker database that can be used to determine the primary objective. Digital biomarkers and blood-based biomarkers will be tested to determine whether a meaningful relationship exists between biomarkers alone or in combination with tau or amyloid brain pathology identified through PET images.
NCT06318169
The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.
NCT04767373
The primary objectives of this phase 2b/3 double-blind, randomized, placebo-controlled study are to evaluate the efficacy and safety of clesrovimab in healthy pre-term and full-term infants. It is hypothesized that clesrovimab will reduce the incidence of respiratory syncytial virus (RSV)-associated medically attended lower respiratory infection (MALRI) from Days 1 through 150 postdose compared to placebo.
NCT06676319
This is a parallel-group, Phase 2, randomized, double-blind, placebo-controlled, 2-arm study for the treatment of asthma. The purpose of this study is to assess the efficacy, safety, and tolerability of add-on therapy with subcutaneous (SC) lunsekimig compared with placebo in male and female participants (aged 18 to 80 years, inclusive) with asthma, who are not currently eligible for biologic treatments. Study details include: * The study duration will be approximately 64 weeks for participants not transitioning into the LTS study and approximately 60 weeks for participants transitioning into the LTS study. * The investigational treatment duration will be up to approximately 52 weeks. * The number of visits will be 18.
NCT06280391
ACT18018 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 study with 3 treatment groups. The purpose of this study is to evaluate efficacy, safety and tolerability with 2 dosing regimens of itepekimab compared with placebo in male and/or female participants with NCFB aged 18 years of age up to 85 years of age (inclusive). Study details include: * The study duration (screening, 24-52-week treatment, 20-week safety follow-up) will be up to 47-77 weeks. * The treatment duration will be up to 24-52 weeks. * The follow-up duration will be 20 weeks. * Site/phone visits are at a monthly interval.
NCT07295847
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA).
NCT06179875
The investigational drug, VRDN-001, is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R). Inhibition of IGF-1R may help to reduce the inflammation and associated tissue swelling that occurs in patients with thyroid eye disease (TED). The primary objectives of this clinical trial are to provide open-label access to VRDN-001 for participants who were previously non-responders at 3 weeks post the fifth IV infusion (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies and assess the safety and efficacy of VRDN-001 in participants who were previously treated with VRDN-001 or placebo.
NCT06621602
This study aims to assess the levels of phosphorylated alpha-synuclein (P-SYN) in patients with Parkinson's disease and REM Behavior Disorder using a minimally invasive skin punch biopsy. It seeks to understand the natural progression of P-SYN deposition over time to explore the potential of P-SYN quantification as a biomarker for disease progression.
NCT04715139
The objective of the registry is to evaluate the continued safety and performance of Arthrex foot and ankle products, including the ProStop® implant for hyperpronated foot; Bio-Compression Screw for reconstruction surgeries of the foot; TRIM-IT Drill Pin® system and/or TRIM-IT Spin Pin™ system for fixation of fractures and fusion (bunionectomy osteotomies) of the foot/ankle; Headless Compression Screws and Compression FT Screws for fixation of small bone fragments of the foot/ankle; DynaNite® nitinol staple for midfoot and hindfoot arthrodeses or osteotomies, first metatarsophalangeal arthrodesis, and mono or bi-cortical osteotomies in the forefoot; BioComposite SutureTak® anchor for medial ankle stabilization; Beveled FT Screws for hallux valgus repair; KreuLock™ screws for ankle fractures; ArthroFLEX® dermal allograft for hallux rigidus arthroplasty; and DualCompression Hindfoot Nail for tibiotalocalcaneal arthrodesis.
NCT07396376
ELU42 01 01 (SuperHealer42) is a Phase I/IIA open label study sponsored by Eluciderm, Inc. that evaluates the safety and preliminary effectiveness of ELU42, a topical small molecule designed to modulate Wnt signaling, for the treatment of chronic diabetic foot ulcers (DFU). ELU42 combines a tankyrase inhibitor (XAV939) with a novel derivatized hyaluronic acid excipient (DHA77) and is applied as a topical spray to the index ulcer. Fifteen adults with Wagner grade 1-2 diabetic foot ulcers that have been present for at least 4 weeks and up to 52 weeks will be enrolled across up to four U.S. clinical sites. After a 2 week run in period during which standard of care (SOC) is provided (off loading, dressings, and debridement), subjects will receive ELU42 applied on site by trained study staff or the investigator three times per week (Monday, Wednesday, Friday) for six weeks (up to 18 applications). Subjects will be followed weekly during treatment and for up to 6 weeks after the final dose; additional Healing Confirmation visits are scheduled if the wound closes to evaluate the subjects over a course of a 3 month period. The study's co-primary objectives are to assess safety (incidence and severity of adverse events and infections) and to measure percent area reduction (PAR) of the index ulcer at Weeks 4 and 6. Efficacy assessments will be performed by the site investigator using direct two axis planimetry and by automated evaluation using the Tissue Analytics platform (surface area, volume, and PAR). Secondary and exploratory assessments include patient reported outcomes (Wound Q and SF 36), frequency of complete wound healing, wound hydration metrics, and pharmacokinetic sampling in a subset of subjects. Contact information for potential participants and referring clinicians is available at each participating site.
NCT03750409
This study will gather data to see if infrared and near infrared light frequency can increase the activity of brain cells and provide support for the cell's ability to repair and protect themselves against further damage.
NCT05574335
This is a multicenter, Phase Ib, open-label, siplizumab dose-finding study in individuals aged 8-45 years with a Type 1 diabetes mellitus (T1DM) diagnosis. within 18 months of V0. Participants will be randomized 1:1:1:1 to one of four possible siplizumab dosing arms. All dosing arms will receive weekly siplizumab doses for a total of 12 weeks. After the completion of treatment, participants will undergo follow-up visits at weeks 12, 24, 36 and 52 which include longitudinal MMTTs. If indicated, participants will enter into long-term safety monitoring for up to an additional 48 weeks. Blood samples for mechanistic analyses will be obtained during the treatment phase and thereafter. Adults aged 18- 45 will be enrolled initially at the study sites. The primary objective is to identify a safe, metabolically favorable, dosing regimen for siplizumab in patients with type 1 diabetes that induces changes in T cell phenotypes observed with alefacept therapy in new-onset T1DM. The secondary objectives are to: 1. Assess the safety profile of siplizumab in recently diagnosed T1DM. 2. Assess the effects of siplizumab on residual beta cell function in recently diagnosed T1DM participants.
NCT05937854
The investigators will study whether the drug tadalafil improves shortness of breath in 126 Veterans with Chronic Obstructive Pulmonary Disease (COPD) and high blood pressure in the lungs. The investigators will also assess whether tadalafil improves quality of life, home daily physical activity, exercise endurance, the frequency of acute flares of COPD, blood pressure in the lungs, and lung function. Veterans who enroll in the trial will be allocated by chance to either active tadalafil or an inactive identical capsule (placebo). Neither the Veteran nor the investigator will know whether the Veteran is taking tadalafil or placebo. Veterans will be followed closely in clinic or by telephone at 1, 2, 3, 4, 5, and 6 months, with attention to side effects and safety. At 1,3, and 6 months the investigators will repeat the questionnaires and testing of blood pressures in the lung and lung function. The investigators anticipate that the results of this study will determine whether tadalafil improves shortness of breath when added to usual medications for COPD.