Investigator-Initiated Trials / Studies

As part of our commitment to delivering innovative therapies to patients worldwide, Novartis believes in the need to support ethical independent clinical research conducted by qualified third-party investigators.

The value of the scientific research produced by these investigators is key to complementing Novartis-sponsored research by helping to ensure we better understand the benefit/risk profile of our therapies, as well as enabling us to explore new opportunities addressing unmet medical needs.

The proposed clinical research must offer meaningful scientific and/or clinical objectives supported by valid study designs in which the privacy rights, safety and welfare of patients is of paramount importance.

Novartis defines IITs as “studies with scientific and medical merit developed and sponsored by an independent investigator or academic sponsor. An IIT may be a clinical or non-clinical study conducted without the participation of Novartis, for which the IIT sponsor requests Novartis to provide either funding, drug product or both.”

Novartis global investigator-initiated trial guidelines (PDF 0.3 MB) 

IIT guidance document (PDF 0.6 MB)

Strategic areas of interest

We welcome unsolicited research proposals from qualified investigators in our strategic areas of interest which we list below. Well-thought through studies that enhance our delivery of innovative therapies to more patients worldwide, enhance patient care, and align with our strategic areas of interest will be considered. If you have questions on any steps of the process or wish to discuss your study concept, please feel free reach out to your local Novartis contact (e.g. MSL, Medical Advisor) for support.

COVID-19: We are interested in COVID-19 vaccination studies in combination with Novartis products.

Cardiovascular, Renal & Metabolism
  • Studies within the label population:
    • Long-term safety and tolerability
    • Health-related quality of life (HrQol)
    • To support local population health agreement and/or implementation research
      (except CVOT that is out of scope)
    • Studies on apheresis in HeFH
  • Mechanistic Studies in secondary prevention
    • Remodeling, fibrosis, inflammation 
    • Plaque burden regression/modification
    • CABG graft remodeling
  • Mechanistic Studies in primary prevention and/or statins intolerant
    • Remodeling, fibrosis, inflammation 
    • Plaque burden regression/modification

Assessment techniques (IVUS, EKO, CCTA) must be guidelines validated (pending vascular bed assessment)

  • Out of scope:
    • Studies in off-label populations, (with respect to geographies)
    • Efficacy, safety and tolerability studies with inclisiran in pediatric population (<18 y)
    • Studies in different populations than ASCVD, ASCVD equivalent, and FH
    • CVOT trials
    • Pre-Clinical Proposals (separate process)
  • With Drug:
    • Mechanistic studies in IgAN, C3G, aHUS
    • Subgroups of patients that are included in the overall study population in indications pursued with iptacopan (IgAN, C3G, aHUS)
  • Without Drug:
    • Role of complement system in complement-driven renal disease
    • Additional ways to foster diagnosis of glomerulopathies beyond biopsy
    • Identification of biomarkers that leads to better characterization, management or correlation with outcomes in IgAN, C3G, aHUS, MN, LN
    • Burden of disease (clinical, economic, and/or humanistic burden) - IgAN, C3G, aHUS, MN, LN
    • Epidemiology studies (including registries) - IgAN, C3G, aHUS, MN, LN
  • Out of scope:
    • Pediatric studies
    • Studies exploring different dosing regimens as currently investigated
    • Any study, which combines iptacopan with immunosuppressant
    • Head-to-head comparisons
    • Studies including patients with CKD stages 4 and 5
  • Studies with sac/val in Chronic Heart Failure with reduced ejection fraction (EF):
    • Safety and tolerability in populations not well represented in PARADIGM-HF, PIONEER-HF
      • de-novo heart failure
      • ACEi/ARB naive
  • Mechanistic studies looking at:
    • Remodeling, fibrosis, inflammation
    • Cardiac function (including diastolic function)
    • Cardiac biomarkers
  • Population with specific, less well studied/documented HF etiologies
  • Out of scope :
    • Studies in HFmREF or HFpEF or post-MI patients
    • Comparative effectivenes studies vs other MoA, e.g. SGLT-2i
    • Studies in non-cardiovascular disease
    • Studies in patients with valvular disorders not related to HF
    • Studies focused on hypertension including resistant hypertension
    • Studies in patients with severe renal impairment (eGFR < 30 ml/min/1.73 m2)
    • Studies in children (<18 years)
Immunology, Hepatology and Dermatology
  • Registries with data on Familial Mediterranean Fever (FMF):
    • Real-life data on colchicine resistance, lack of tolerance and suboptimal response to colchicine (retrospective)
    • Impact on quality of life (QoL) of suboptimal response to colchicine; impact seen by patients vs that by HCPs (prospective)
  • Clinical studies looking at the window of opportunity in Still’s disease:
    • Early use of canakinumab in Still’s disease
  • Studies in rare systemic autoinflammatory diseases (SAID):
    • Schnitzler Syndrome
    • Periodic Fever, Aphthous stomatitis, Pharyngitis and cervical Adenitis (PFAPA)
    • Pyogenic sterile Arthritis Pyoderma gangrenosum and Acne (PAPA)
    • Chronic Recurrent Multifocal Osteomyelitis (CRMO)
    • Kawasaki
    • Behçet disease
    • Yao syndrome
    • Post-hoc analyses of existing trials
  • Biomarkers in Still’s disease & IL-1ß signature
  • Out of scope
    • Osteoarthritis & other high prevalence indications
    • Pre-clinical IITs

Indications: Psoriasis (PsO), Psoriatic Arthritis (PsA), Axial SpA (ankylosing spondylitis, nr-axSpA)

  • Studies assessing:
    • Clinical outcomes addressing one or several key clinical manifestations of PsA, peripheral SpA or axial manifestations with axial SpA
    • Novel imaging techniques targeting axial or peripheral manifestations (synovitis, enthesitis), structural progression, repair process and bone remodelling in peripheral and axial SpA
    • Implementation of early diagnosis and management of psoriatic disease or SpA to improve long term outcomes
  • Exploratory studies assessing:
    • Treatment effects on selected systemic manifestations and/or comorbidities of psoriatic disease or SpA
    • Novel imaging techniques to investigate the role of secukinumab in limiting progression from PsO to PsA and nr-axSpA to Axial SpA
    • Machine learning techniques to create predictive models for disease trajectories, and IL-17A inhibition responses across disease phenotypes/genotypes
  • Mechanistic studies assessing:
    • Early treatment and disease modification, use of biomarkers to predict disease and treatment outcomes
    • Roles of different pathways (TNFa, IL-17, IL-22/IL-23) in enthesitis, axial disease, dactylitis and peripheral SpA
    • Role of IL 17 pathways in different clinical manifestations of lupus nephritis, Giant cell arteritis, ERA-JIA and JPsA
    • Pathways in diseases with potential role of IL-17A e.g. Hidradenitis Suppurativa, Lichen Planus, Ichthyosis/Netherton syndrome, Pityriasis Rubra Pilaris, non-ocular Bechet’s Disease, Papulopustular Rosacea, JIA uveitis, CRMO/SAPHO, or others
  • RWE studies assessing:
    • Effectiveness, drug survival, cost-effectiveness, resource utilization and treatment patterns across approved indications
    • T2T or guidelines implementation strategies to achieve disease remission
  • Out of scope:
    • Combination studies for secukinumab with other biologic agents
    • Studies in: safety topics e.g. infections (tuberculosis, HIV, viral hepatitis), transplant, sarcoidosis, high-risk patients
    • Life-threatening conditions (evaluated on a case-by-case basis)
    • Studies related to unapproved indications (evaluated on a case-by-case basis)
Neuroscience
  • Vaccination studies 
  • Implementation studies 
  • Telemedicine approaches in MS patient care
  • Patient-reported/relevant outcomes and development of novel and holistic patient improvement measures
  • Clinical impact in cognition, fatigue, ambulation and other functional changes
  • Biomarkers in MS disease
  • Neuroimaging (microglia, brain atrophy, SC, cortical, etc.)
  • Progression and Predictors of response/conversion: Digital tools, clinical, biomarkers
  • Differentiation of S1P Modulators
  • Differentiation of ofatumumab from other RMS DMTs
  • MS pathophysiology studies, including mechanism of CD20+ immune cells in disease
  • Use of ofatumumab in subpopulations not specifically studied in pivotal trial programs 
  • Potential role of ofatumumab in neuroscience diseases/disorders in which dysregulation of B cells may occur
  • Confirmatory studies that high efficacy MS treatment early leads to better patient outcomes
  • Out of scope:
    • Head to head studies
    • Studies in patients with secondary progressive MS (for ofatumumab)
    • Studies in oncological indications
Oncology
  • Studies with alpelisib in HR+/HER2- studies in breast cancer patients exploring:
    • Side effect management trials (e.g., hyperglycemia and rash)
    • Post progression on CDK4/6 inhibitors + endocrine therapy in adjuvant setting
    • Recycling of fulvestrant after use of CDK4/6i plus fulvestrant
    • Rash management optimization trials
  • Studies with alpelisib in other tumor types with PIK3CA mutations 
  • Efficacy and safety of asciminib in 3L+ CML-CP including RWE
  • Efficacy and safety of asciminib in 1L/2L CML including TFR, PRO and QoL
  • Ability of asciminib as monotherapy or in combination to overcome TKI resistance and mutations in CML
  • Predictors of response, dosing strategy, PK/PD and pre-clinical insights into STAMP MOA
  • Exploration of asciminib in high need populations: Ph+ ALL, CML-AP, CML-BC, peri-transplant
  • Out of scope:
    • Combination with ponatinib
  • Real World Evidence generation on capmatinib effectiveness in MET-dysregulated
    • Non-small cell lung cancer (NSCLC)
      • In subpopulations of interest (e.g., brain metastases)
      • Relative effectiveness vs. therapeutic alternatives (e.g., other METi, immune checkpoint inhibitors (ICI), chemotherapy + ICI)
      • Treatment sequencing
  • Clinical trials* in
    • NSCLC
      • Combinations with targeted (e.g., address MET-mediated resistance) and other therapies
      • Subpopulations (e.g., brain metastases) and new settings (e.g., neo/adjuvant)
      • Treatment sequencing, re-challenge
  • Preclinical/mechanistic and other studies*
    • Capmatinib brain penetration and activity
    • Combinations addressing resistance mechanisms
    • To help improve patient experience by addressing adverse events of interest (e.g., peripheral edema)

* supported by solid scientific rationale, not overlapping with Novartis-sponsored/supported studies 

  • Melanoma:
    • dabrafenib/trametinib treatment optimization including sequencing and/or biomarkers driven strategies 
    • Melanoma early stages (i.e., neoadjuvant, adjuvant treatment after local/regional relapse) 
    • Acral/mucosal melanoma
    • Real world evidence (i.e., patient characterization, treatment patterns, outcome predictors)
  • Out of scope:
    • NRAS mutated melanoma
    • Pediatric gliomas
  • Myelofibrosis
    • RWE low-int-1 and long-term outcomes
    • Combinations – First line / suboptimal responders
    • Optimizing dose and management
    • Re-challenge
  • Polycythemia Vera
    • Cardiovascular disease outcome / biomarkers
    • Long-term outcomes (RWE)
    • Post Interferon
  • Graft versus Host Disease
    • Burden of Illness / treatment flow (RWE) 
    • Prophylaxis
    • First line acute / chronic
  • Out of scope:
    • IITs competing with ongoing studies
    • Solid tumors
    • Non-oncological indications
  • Studies with Lutathera in well differentiated SSTR+ tumors of neuroendocrine origin
  • Retrospective studies with additional cycles of Lutathera in SSTR+ GEP-NET patients after having progressed on initial Lutathera treatment (“re-treatment”/re-challenge) 
  • Retrospective studies describing treatment sequence in metastatic GEP-NET patients 
  • Studies with Lutathera in metastatic NET patients in combination with other anti-cancer treatments, including chemotherapy, immuno-oncology therapies, tyrosine kinase inhibitors, PARP-inhibitors, CDK4/6 inhibitors or other upcoming treatments (if supported by MoA rationale) 
  • Studies describing the Quality of Life/ Patient reported outcomes related to Lutathera treatment
  • Retrospective studies describing long-term safety or health economic aspects 
  • Studies with neoadjuvant Lutathera use in GEP-NET
  • Intraarterial Lutathera administration studies in GEP-NET
  • Focus on 1/2L mCRPC and nm (localized, loco-regional) and mHSPC (De Novo and Relapse)
    • Chemo-unfit /frail patients population
    • Head to head vs chemotherapy in 1L mHSPC
    • Combination and sequencing strategies
      • with PSMA enhancers / sensitizers in mHSPC 
      • to reduce and control AE management 
      • with PARP inhibitor or Immunotherapeutic in mHSPC
    • Pattern of treatment sequencing to understand potential cross-resistance
    • Re-treatment strategies from localized, loco-regional or mHSPC to mCRPC
    • Long-term safety studies and/or post-progression evaluations 
    • Burden of illness and quality of life studies 
  • Biomarker / translational studies
    • Predictive to efficacy and safety, as well as disease aggressiveness
    • Informative (PSMA-PET)
    • Resistance
    • Regulation of PSMA expression in different disease stages
  • Out of scope:
    • all other tumor types beyond prostate cancer
    • Clinical studies in COVID-19
  • Focus on HR+/HER2- studies in breast cancer patients:
    • Exploring treatment strategies to
      • overcome resistance following initial treatment with a CDK4/6 inhibitor + endocrine therapy
      • personalize treatment selection in early and metastatic breast cancer
      • detect early recurrence/progression
    • Utilizing real world data and/or and digital health technologies
    • Utilizing patient reported outcomes (PRO)
  • Mechanistic studies looking at CDK4 and CDK6 function in cellular senescence, immunomodulation, and endocrine resistance
  • Out of scope: all other tumor types beyond breast cancer
  • Biomarker studies (e.g., TIM-3, LSC, etc.)
  • Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML): Combinations, novel –novel, optimal sequence
  • Burden of illness and quality of life studies in MDS and AML 
  • Pattern of treatment, novel agents treatment in real world (RW) and RW data in MDS and AML
  • Out of scope:
    • Solid tumors and other none-hematology indications
    • Studies in children
    • Clinical studies in COVID-19
  • Identification (± modification) of factors influencing clinical outcomes
  • New combinations and optimized treatment sequencing
  • Strategies for overcoming resistance and relapse
  • Optimization of patient management to reduce toxicity
  • Demonstrating effectiveness, safety and resource utilization profiles in the real world setting
Ophthalmology

Indication: neovascular Age-related Macular Degeneration (nAMD)

  • Studies investigating innovative assessment methods:
    • functional efficacy end-points, e.g. reading speed, contrast sensitivity and anatomic end-points
    • new imaging tools e.g. angio-OCT and Ultrawide Field imaging
  • Studies aimed to evaluate effectiveness with real-world usage of brolucizumab, including treatment patterns, anatomical outcomes, treatment burden and adherence/compliance, impact on QoL
  • Studies investigating brolucizumab in sub-populations, e.g. CNV sub-types or PED and other VEGF-driven retinal diseases, e.g.Mac-Tel; Central Serous Chorioretinopathy and similar
  • Studies investigating biomarkers or genetic markers which could better predict outcomes to improve patient care
  • Studies utilizing novel PRO endpoints and evaluations
  • Studies involving use of digital technology for improved patient compliance (e.g. home monitoring devices)
  • Out of scope:
    • Mechanisitc/Pre-clinical studies aimed to evaluate and understand PK/PD or systemic VEGF levels and similar
    • H2H studies with insufficient statistical power (i.e. small sample size) evaluating BCVA as primary efficacy objective
    • Studies (single arm or H2H) targeting safety as primary or key secondary end-point
    • H2H studies versus unlicensed bevacizumab or licensed biosimilars of any anti-VEGF agents

Indication: dry eye disease

  • Characterization of DED and/or DED patient types (i.e. Sjogren’s Disease, Meibomian Gland Dysfunction)
  • Tools to measure DED signs and symptoms objectively
  • Simplifying diagnosis and measuring disease progression (i.e. biomarkers)
  • Functional vision testing
  • Effect of screen use on OSD
  • Tools to improve patient compliance
  • Out of scope:
    • Studies comparing to artificial tears
    • Head to head studies

Indication: Chronic ocular surface pain

  • Characterization of patient cohorts/disease populations with chronic ocular surface pain
  • Patient-Reported Outcomes for chronic ocular surface pain assessments
  • Characterization of treatment paradigm and referral pathway for ocular pain patients
  • Tools to measure chronic ocular surface pain signs and symptoms objectively
  • Out of scope:
    • Studies treating the underlying cause of the ocular surface pain
    • Studies vs. systemic pain management regimes

Indication: ocular gene

  • Epidemiology studies
  • Prospective non-interventional studies to follow-up efficacy & safety of treated patients 
  • Patient-reported outcome projects in IRD patients
  • Patient identification, retina viable cells & peer to peer engagement
  • Molecular diagnosis & genotyping projects (including variances of uncertain significance)
  • Novel surgical procedures for drug delivery
  • Novel approaches to reduce time of diagnosis of IRD patients
  • Out of scope:
    • Studies in non-ocular disease
    • Studies in patients below the age of 12 months
    • Interventional studies in which drug is requested
Respiratory
  • Effectiveness of IND/GLY/MF (LABA/LAMA/ICS), incl. benefits on symptom relief
  • Benefits of the Breezhaler (Concept 1) and/or digital companion (sensor & app), incl. characterization of the patient population who benefits most from digital tools
  • Anti-inflammatory action of GLY (LAMA)
  • Asthma: efficacy/effectiveness/Safety of Xolair on asthma and in comorbid conditions
  • Nasal Polyposis (NP): effectiveness of Xolair in NP, effect of Xolair on the NP biology, AFRS Xolair data (Allergic Fungal RhinoSinusitis)
  • Food Allergy FA: Xolair effectiveness on FA, data on the role of IgE in all FA, data on the food allergen specific IgE
  • Chronic Spontaneous Urticaria: studies on treatment in early CSU disease, Patient reported/patient centric outcomes, effect of Xolair on the CSU biology, RWE & Long-term Safety and efficacy, CINDU, other dosing regimens (eg > 300 mg/4w), digital technology for improved patient compliance; disease modifying capabilities, patients <12 y.o
  • Other indications with rationale to support the role of IgE and with high medical need

How do I submit an IIT request?

IIT requests are submitted via the Novartis Grants, External Studies and Managed Access System or GEMS portal. Please submit your concept by clicking here.

Guidance on using the GEMS portal is available here:

Novartis GEMS portal external user guide (PDF 0.1 MB)

For IIT related questions, please contact the medical team in your Novartis local country office.