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

Inclisiran (Leqvio®)

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)

Iptacopan (LNP023)

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

Sacubitril/Valsartan (Entresto)

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

Canakinumab (Ilaris)

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

Secukinumab (Cosentyx)

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

Ofatumumab (Kesimpta) and siponimod (Mayzent) - Multiple sclerosis (MS)

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

alpelisib (Piqray)

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

Capmatinib (Tabrecta ®)

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

crizanlizumab (Adakveo)

Burden of illness and quality of life studies in sickle cell disease (SCD)
SCD biomarker studies
Long term impact of vaso-occlusive crises (VOCs) (i.e., adults, adolescents and pediatrics)
Mechanistic studies in SCD (e.g., inflammation, vascular remodeling, sudden cardiac death)
Studies in other p-selectin driven hematology disease areas
Microvascular perfusion studies evaluating p-selectin inhibition in SCD patients
SCD treatment management studies (e.g., infusion speed, premedication, adherence)
Studies of medical digital devices in SCD patient care
Out of scope:
- Organ protection specific clinical studies in SCD patients (i.e., renal, retina, brain, liver, heart)
- Combination studies with steroids and other monoclonal antibodies (e.g., Crohn’s disease, RA)
- Studies in non-hematology disease areas

dabrafenib (Tafinlar) + trametinib (Mekinist)

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

Jakavi

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

[177Lu] Lu-DOTA-TATE (Lutathera ®)

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

[177Lu] Lu-PSMA-617

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

ribociclib (Kisqali)

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

sabatolimab (MBG453)

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

tisagenlecleucel (Kymriah)

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

Brolucizumab (Beovu)

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

Lifitegrast (Xiidra®) and ECF843

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

SAF312

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

Voretigene neparvovec (Luxturna®)

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

Enerzair Breezhaler (QVM149; indacaterol acetate, glycopyrronium bromide and mometasone furoate [IND/GLY/MF])

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)

Omalizumab (Xolair)

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.

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