Utrecht-, Netherlands

A Study to Evaluate the Efficacy and Safety of Tarcocimab Tedromer and Tabirafusp Tedromer Compared to Aflibercept in Participants With Neovascular (Wet) Age-related Macular Degeneration (wAMD) - DAYBREAK

A Study of Zasocitinib in Adults With Psoriatic Arthritis Who Have Not Taken Biologic Medicines

Impella RP Flex with Smart Assist

A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators

People with Cystic Fibrosis (pwCF) who are genetically ineligible and/or not taking cystic fibrosis transmembrane conductance regulator (CFTR) modulators currently face future health that is considerably different from the approximately 90% of pwCF in the United States who benefit from CFTR modulators. New treatments are being advanced for pwCF who are genetically ineligible or not taking CFTR modulators and these therapies will include both nucleic acid-based therapies (NABTs) to address the underlying defect in CFTR and symptomatic therapies aimed at targeting the symptoms of CF. A key concern for this limited and underserved patient population is being able to advance multiple therapeutic programs in parallel. To complete these studies, CF researchers will need to be able to reach this community effectively while also promoting the use of innovative trial designs. The REACH Study is a prospective, longitudinal, observational research study to obtain research quality (i.e., monitored research) CF outcome data. Primary outcome endpoints of the Core study (collected across all study participants) are aligned with anticipated clinical trial outcome endpoints needed in overall development of therapies for the CF population unable to benefit from CFTR modulators. This study will also include sub-studies to obtain specialized measures which may help inform efficacy and safety evaluations of new therapies by providing CF control data. Finally, this study also seeks to assess research solicitation and research participation for the CF population that is modulator ineligible or not taking modulators. The observational data collected within this study may be used in characterizing this CF population, developing innovative trial designs, for comparison when evaluating new or experimental CF therapies, and/or in CF research.

Triptorelin for the Prevention of Ovarian Damage in Adolescents and Young Adults With Cancer

PRIMARY OBJECTIVES: I. Determine the feasibility of conducting a cross network, multi-site, randomized clinical trial of triptorelin among newly diagnosed adolescent and young adult (AYA) female cancer patients age < 40 years (exclusive of breast cancer). II. Measure ovarian reserve via anti-Mullerian hormone (AMH) at 2-years post completion of alkylating agent-containing chemotherapy among randomized patients. SECONDARY OBJECTIVES: I. Collect information on the longitudinal trajectory of change in AMH and other ovarian hormone levels from cancer diagnosis to 2 years post cancer treatment completion among randomized patients. II. Determine the feasibility of measuring estrogen deprivation symptoms (i.e., hot flashes, sexual dysfunction) menstrual pattern, and quality of life among randomized patients. EXPLORATORY OBJECTIVE: I. Establish a unique cohort of female AYA patients treated with alkylating agent chemotherapy and randomized to receive or not receive triptorelin, that can be followed long-term to study reproductive health concerns and outcomes as well as genetic risk factors for premature menopause. OUTLINE: Patients are randomized to 1 of 2 arms. ARM A: Patients receive triptorelin intramuscularly (IM) up to 14 days prior to standard chemotherapy. For patients whose chemotherapy exceeds 24 weeks, a second dose of triptorelin may be given 24 weeks after the first dose at the treating physician's discretion. Patients also undergo blood sample collection throughout the study. ARM B: Patients receive standard chemotherapy. Patients also undergo blood sample collection throughout the study. After completion of study treatment, patients are followed up at 1 and 2 years.

A Study of RSLV-132 in Females With Sjögren's Syndrome

Safety and Efficacy of NMD670 in Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease

RECOVER-AUTONOMIC Platform Protocol

The hypothesis is that some of the autonomic dysfunction symptoms are immune-mediated, so immunotherapy and other applicable therapies will result in improvement in autonomic symptoms. Interventions will be added to the platform protocol as appendices. Each appendix will leverage all elements of the platform protocol, with additional elements described in the individual appendix.

Defining Treatment Outcomes and Genetic Architecture in Idiopathic Toe Walking*

Toe walking is a very common diagnosis in children with a prevalence of 5% -24%. Persistent toe walking in children over 3 years of age often results in parental concern, provoking multiple medical visits, and a range of interventions. Additionally, toe walking has both social implications and concerns for increased foot and ankle pain in those with contracture. Idiopathic toe walking (ITW) is a diagnosis of exclusion and affirming the diagnosis, ascertaining if intervention is indicated, and determining the optimal timing and intervention strategy are not well defined in the literature. As a family history of toe walking is reported in many children with ITW, there is a strong possibility that a subset of children may have a genetic cause for the condition which may impact the clinical course and outcome of treatment. Several approaches to intervention have been suggested to address toe-walking including: observation, therapy, casting, botulinum toxin A as well as surgery to lengthen the gastroc-soleus complex at the level of the calf or Achilles tendon. The purpose of this multi-center study is to examine a well-defined cohort of children with ITW utilizing a combination of quantitative measurement tools, parent reported outcomes, and whole genome sequencing to promote an evidence-based approach to orthopaedic management of this population. One hundred and eighty children who meet the inclusion/exclusion criteria for this study will be recruited from 8 participating SHC sites (POR, NCA, SLC, CHI, PHL, SPO, GRN, LEX) and treated with either serial casting or surgery. Children will be assessed 3 times over 1 year (Baseline, 6-months and 1-year post intervention). A series of screening as well as delineated inclusion/exclusion criteria will be used to ensure the diagnosis of ITW. Clinical assessments, radiographs and 3-D gait analysis utilizing electromyography and a multi-segment foot model will be used to determine if there are differences in the range of motion, gait kinematics and kinetics, motor synergies and foot contact patterns following casting or surgery. Whole genome sequencing will be used to determine if there is a genetic basis for ITW can be identified. Analysis will focus on 1) comparing and contrasting the short and long-term outcomes following non-operative (casting) and surgical intervention to determine if one approach is more efficacious, 2) identify potential genetic determinants for ITWp and 3) identify the factors that mediate and moderate intervention efficacy. The knowledge gained from this study will promote development of an evidence-based and personalized approach to the management of ITW.

A Global Study of Volrustomig (MEDI5752) for Participants With Unresected Locally Advanced Head and Neck Squamous Cell Carcinoma Following Definitive Concurrent Chemoradiotherapy