## [IIT US Areas of Interest](/node/662881 "IIT US Areas of Interest") Oncology Ribociclib (Kisqali) **HR+/HER2- Studies in Breast Cancer** - Exploring ribociclib with novel/emergent compounds - Utilizing real-world data (RWD) and/or digital health technologies - Enhances the treatment experience of patients **Out of Scope** - Any area outside HR+/HER2- breast cancer - Any study in overlap with ongoing Novartis-sponsored/supported studies 177LuDOTATATE (Lutathera) / 68GaDOTATATE (Netspot) **GEP & Bronchopulmonary NET** - Re-treatment/Re-challenge with 177LuDOTATATE (after initial 4 cycles) - Combinations of 177LuDOTATATE with other agents with potential to improve efficacy - Sequencing studies with 177LuDOTATATE - Long-term safety of 177LuDOTATATE - Efficacy/Safety of 177LuDOTATATE in specific patient subgroups **Other SSTR+ Tumors** - Role of 177LuDOTATATE in the management of patients with other SSTR+ tumors **NETSPOT for Imaging** - Role of 68GaDOTATATE in GEP-NET and other SSTR2+ tumors [68Ga]Ga-FAP-2286 **Imaging Studies in FAP-expressing Solid Tumors** - Role of FAP PET in diagnosis, staging, clinical decision-making, and treatment response - Studies exploring FAP PET as an imaging biomarker: correlation with other biomarkers such as histological, molecular, and genetic subtypes - Understanding FAP expression in benign/inflammatory processes in relation to FAP RLT safety and patient selection for therapy 225Ac-PSMA-617 - Use of PSMA-targeted PET imaging agents in prostate cancer (e.g., patient selection, treatment assessment) - Treatments up-regulating PSMA expression in prostate cancer - Methods of preventing and/or managing RLT-induced xerostomia - Real-world evidence in prostate cancer for 225Ac-PSMA-617 - Health disparities in advanced prostate cancer - Sequencing with 177Lu-PSMA-617 Tisagenlecleucel (Kymriah) - Essential factors for selecting patients for tisagenlecleucel therapy to improve safety and/or response for their approved indications - Essential factors for sequencing tisagenlecleucel therapy with other therapies and determining outcomes for their approved indications - Novel combinations of therapies with tisagenlecleucel to improve response and/or safety for their approved indications - Study outcomes of tisagenlecleucel administered at various sites (e.g., inpatient, outpatient, community hospital, community practice) for their approved indications Asciminib (Scemblix) **CML-CP in Earlier Lines (1L & 2L)** - Sequencing of TKIs, clinical efficacy, and safety in real-world settings - Patient-reported outcomes (PROs) and quality-of-life issues with current CML therapies - Biomarkers for TFR and safety - Long-term safety and tolerability - Studies aiming to improve eligibility to attempt TFR and reduce the risk of relapse after TFR attempts - Response to asciminib in patients with pre-existing BCR::ABL1 mutations other than T315I, or treatment approaches in patients with emerging mutations under asciminib, including compound mutations **CML-BC and Ph+ ALL** - Efficacy and safety of asciminib in selected ALL settings (Ph+, Ph-like) - Exploratory high-risk advanced phase CML populations such as patients with additional genomic alterations - TKI-based combinations addressing high unmet need populations (CML-AP/BC) **Out of Scope** - Use in non-BCR::ABL diseases [177Lu]Lu-PSMA-617 (Pluvicto) - Investigating retreatment or extended treatment with 177Lu-PSMA-617 - Real-world evidence in prostate cancer for 177Lu-PSMA-617 - Health disparities in advanced prostate cancer CRM Inclisiran (Leqvio) - ASCVD MOA – atherosclerotic plaque composition changes - Differentiation of inclisiran vs. other LLTs in a real-world setting (e.g., adherence, implementation) Pelacarsen / Lp(a) **Non-drug IITs** Epidemiology associated with elevated Lp(a) - Patient characterization, identification, and genetic risk across sub-groups. - Association and impact on different types of CVD (e.g., ischemic stroke, PAD), polyvascular disease, and other cardiovascular-related diseases. Distinct and unique pathophysiology of Lp(a)-related CVD - Insights on the pro-thrombotic mechanisms impacted by Lp(a). - Unique features of Lp(a). Patient perception of the contribution of Lp(a) to CVD and cardiovascular risk Lp(a) testing and global cardiovascular risk management - Implementation of Lp(a) testing in CVD risk evaluation. - Clinical and economic value of Lp(a) testing. **Out of Scope:** - Relationships between Lp(a) and LDL-C, and their independent contributions to Lp(a)-related disease risk. - Lp(a) in non-cardiovascular-related disease. Atrasentan (Vanrafia) Disease-related - Studies evaluating the role of the endothelin pathway in proteinuric kidney diseases - Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities Drug-related - Studies on combination strategies with atrasentan in IgAN **Out of Scope:** - Pediatric studies involving drug treatment - Studies exploring dosing regimens other than the currently FDA-approved dose - Head-to-head comparisons with other approved products - Studies including patients with eGFR <15 mL/min/1.73 m2 (CKD stage 5) Iptacopan (Fabhalta) **Disease-related** Studies evaluating the role of the complement system in complement-mediated kidney diseases. Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities. **Drug-related** Studies on combination strategies with iptacopan in IgAN. **Out of Scope** Pediatric studies (with drug). Head-to-head comparisons with other approved products. Studies including patients with GFR <20 mL/min/1.73 m2. Iptacopan PNH (Fabhalta) **With drug** - Mechanistic studies in PNH. - Studies evaluating factors associated with or predictive of treatment outcome in PNH. **Without drug** - Approaches to facilitating and expediting diagnosis - Identification of biomarkers that lead to better characterization, management, or correlation with outcomes. **Out of Scope** - Pediatric studies. - Clinical trials exploring different dosing regimens than those currently being evaluated in the clinical development program. - Clinical trials combining iptacopan with immunosuppressant and anti-C5 treatments. - Head-to-head comparisons. - Studies in other non-complement-mediated hematologic diseases. Neuroscience Ofatumumab (Kesimpta) **Multiple Sclerosis** - The experience of use of OMB in sub-populations of RMS (e.g., African American and Hispanic patients, and different age groups). - The impact of OMB on MS comorbidities and patient-centric outcomes. - The therapeutic role of OMB in MS, including efficacy, safety, tolerability, and use in treatment-naive patients. - The impact of OMB on both fluid and digital biomarkers in MS. - The pathophysiology of MS (including the mechanism of action of OMB and its effects on MS pathophysiology) and the burden of disease associated with MS (including the impact of OMB). - Innovative neuroimaging techniques used to measure biomarkers of MS disease, MS inflammation, axonal integrity, and function (including the effects of OMB). - The long-term impact of B-cell therapy on the immune system. - Impact of B-cell-depleting therapies on immune system function over time, with particular focus on the non-B-cell compartment. Remibrutinib **Multiple Sclerosis** - The role for remibrutinib in sequencing of treatments. - The impact on imaging, including SELs, PRLs, and cortical lesions. - The impact on the biology of progression, including PET imaging, cognition, fatigue, and depression outcomes. - Effect of remibrutinib on CNS processes, including blood–brain barrier (BBB) transmigration and microglial activation. - The proteome profiling effects of remibrutinib. **Myasthenia Gravis** - Development of biomarkers and endpoint exploration for clinical trial use. - Impact on macrophage involvement in disease pathophysiology at the neuromuscular junction (NMJ). - Effect of remibrutinib on steroid use and its potential to reduce corticosteroid exposure. Onasemnogene abeparvovec-xioi (Zolgensma) **In Scope** - Holistic and societal impact of Zolgensma IV, including clinical transformation, economic impact, quality of life, and societal effects. - Biomarkers for efficacy. **Out of Scope** - Clinical trials involving Zolgensma IV re-dosing. - Studies of alternative Zolgensma IV doses or maximum dose. - Basic science research requesting the use of Zolgensma IV. - Methods or processes to assess the efficacy and durability of Zolgensma IV (e.g., bulbar function). Onasemnogene abeparvovec-brve (Itvisma) **In Scope** - Long-term, multi-domain functional and clinical outcomes, including sleep, bulbar function, head control, scoliosis, respiratory function, and overall independence. - Reproductive outcomes, including fertility and reproductive health. - Biomarker and mechanistic evidence generation, including biomarkers predictive of clinical and functional response and durability. - Overall survival, caregiver and societal impact, long-term care needs and cost/resource implications, and the impact of delaying treatment. **Out of Scope** - Interventional studies of OAV101 IT in patient types not included in clinical trials. - Clinical trials involving OAV101 IT re-dosing or OAV101 IT dosing following OAV101 IV. - Studies of alternative OAV101 IT doses or maximum dose. - Head-to-head comparisons with other therapies and combination with other MDTs. - Studies of OAV101 IT in patients under 2 years of age. - Comparative studies between Zolgensma IV and OAV101 IT. Immunology Remibrutinib - Urticaria **Disease Related Research** - Population-based epidemiology studies; studies investigating the impact of urticaria on patients; studies of real-world treatment patterns (including, for example, overuse of corticosteroids and impact on sleep); and studies of patient preference, patient experience, and satisfaction (qualitative). - Studies employing digital technology, such as in silico models, AI-enabled or machine learning techniques, telemedicine, and related approaches to predict disease trajectories, treatment response, disease modification, and similar outcomes. - Studies investigating innovative tools to support urticaria management, including digital applications and sleep-related tools. - Long-term CSU/CIndU observational studies and secondary use of data. **Clinical Studies** - Studies with remibrutinib in CSU/CIndU. - Proof-of-concept studies in new allergic and dermatologic indications. **Mechanistic Studies** - Mechanistic studies assessing the effects of remibrutinib on mast cells, basophils, B cells, and other relevant immune pathways in vitro or ex vivo. **Out of Scope** - Head-to-head comparisons of remibrutinib with other active treatments. - Studies investigating remibrutinib in combination with biologics. - Alternative dosing regimens to the 25 mg b.i.d. regimen developed in the CSU Phase 3 clinical program. Ianalumab - Sjogren’s US epidemiology and systemic disease characterization. - Sjogren's classification, systemic disease recognition, and clinical assessment. - Sjogren's disease progression: use of ultrasound, clinical assessments, and/or biomarkers. - Sjogren’s symptoms: evidence generation using existing PROs or new SjD symptom assessment modalities. - Sjogren's disease organ domains: generation of evidence in key disease domains. - Sjogren's disease and concomitant conditions (e.g., rheumatoid arthritis, lupus) and associated outcomes. - Sjogren's disease in subpopulations (e.g., African American, Hispanic) and associated outcomes. - Sjogren's disease burden: clinical, social, economic, and humanistic aspects. - Biomarker-informed assessment and monitoring in SjD. - SjD serologic and immunologic characterization and association with clinical outcomes. - Tissue-level immune activity and disease progression in SjD. - [Print](/node/662881/printable/print) - [PDF](/node/662881/printable/pdf)