IIT US Areas of Interest

Oncology

Essential factors for selecting patients for Kymriah (tisagenlecleucel) therapy to improve safety and/or response for their approved indications
Essential factors for sequencing Kymriah (tisagenlecleucel) therapy with other therapies and determining outcomes for their approved indications
Novel combinations of therapies with Kymriah (tisagenlecleucel) to improve response and/or safety for their approved indications
Study outcomes of Kymriah (tisagenlecleucel) administered at various sites (e.g., in-patient, out-patient, community hospital, community practice) for their approved indications

CML-CP in Earlier Lines (1L & 2L)

Sequencing of TKIs, clinical efficacy, and safety in real-world setting
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 the 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 of Non BCR-ABL diseases

With Drug

Mechanistic studies in PNH
Studies evaluating complement factors associated with or predictive of treatment outcome in PNH
Studies evaluating effectiveness, safety and management of Iptacopan in PNH patients treated in the real-world setting
Studies evaluating PNH treated patients in the context of bone marrow disorders, such as AA, in the real-world setting
Studies evaluating the role of factor B inhibition in hematologic complement mediated diseases

Without drug

Complement mediated diseases in Hematology
Role of complement system in disease evolution
Approaches to facilitating and expediting diagnosis
Identification of biomarkers that leads to better characterization, management or correlation with outcomes
Burden of disease (clinical, economic, and/or humanistic burden)
Epidemiology studies (incl. registries)

Out of scope

Pediatric studies
Clinical trials exploring different dosing regimens as currently investigated
Clinical trials combining Iptacopan with immunosuppressant and anti-C5 treatments
Head-to-head comparisons
Studies in other non complement mediated hematologic diseases

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

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

GEP & Bronchopulmonary NET

Re-treatment/Re-challenge with Lutathera (after initial 4 cycles)
Combinations with other agents with potential to improve efficacy
Sequencing studies
Long-term safety
Efficacy/Safety of Lutathera in specific patient subgroups

Other SSTR+ Tumors

Role of Lutathera in the management of patients with other SSTR-positive tumors

NETSPOT for Imaging

Role of Netspot in GEP-NET and other SSTR2+ tumors

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/genetic subtype
Understanding FAP expression in benign/inflammatory processes in relation to FAP RLT safety and patient selection for therapy

Therapeutic Studies

Role of 177Lu-FAP RLT in FAP-expressing solid tumors
Use of 177Lu-FAP RLT in combination with standard of care therapies and/or immuno-oncology agents
Studies investigating the effect of 177Lu-FAP RLT in cancer-associated fibroblasts and tumor microenvironment
Evaluation of optimal dosing regimens in subpopulations and combinations
Correlation of RLT efficacy with predictive biomarkers and FAP PET uptake
Studies exploring alternate routes of administration to improve safety and efficacy

Investigating alternative dosing regimens (cycles, frequency) with ²²⁵Ac-PSMA-617 in mCRPC
Radioligand therapy in neoadjuvant setting for localized prostate cancer
Use of ²²⁵Ac-PSMA-617 in adjuvant setting in combination with EBRT + ADT +/- abiraterone in patients with localized prostate cancer post prostatectomy with N1M0 on PSMA PET
Use of ²²⁵Ac-PSMA-617 post definitive therapy for localized prostate cancer with biochemical recurrence and PSMA-PET M0 disease
Use of PSMA-targeted PET imaging agents in prostate cancer (e.g., patient selection, treatment assessment)
Use of ²²⁵Ac-PSMA-617 in combination with other agents in mHSPC or mCRPC
Treatments up-regulating PSMA expression in prostate cancer
Use of >6 cycles of ²²⁵Ac-PSMA-617 in patients with mHSPC or mCRPC
Use of ²²⁵Ac-PSMA-617 in prostate cancer patients with distinct mutations (e.g., PTEN-loss, AKT, DDR)
Use of ²²⁵Ac-PSMA-617 in patients with low or no PSMA expression in mCRPC
Real-world evidence in prostate cancer for ²²⁵Ac-PSMA-617
Health disparities in advanced prostate cancer
Sequencing with 177Lu-PSMA-617

CRM

Non-drug IITs

Epidemiology associated with elevated Lp(a)

Patient characterization, identification, and genetic risk across sub-groups
Association & impact on different types of CVD (ischemic stroke, PAD), polyvascular disease, and other CV-related diseases

Distinct and unique pathophysiology of Lp(a) related to CVD

Insights on the pro-thrombotic mechanisms impacted by Lp(a)
Unique features of Lp(a)

Patient perception on contribution of Lp(a) to CVD and CV risk

Lp(a) testing and global CV risk management

Implementation of Lp(a) testing in CVD risk evaluation
Clinical and economic value of Lp(a) testing

Out of scope

Comparison / association with LDL-C
Non-cardiovascular related diseases

ASCVD MOA – atherosclerotic plaque composition/changes
Differentiation of inclisiran vs other LLTs in Real World setting – e.g., adherence, implementation

Disease-related: including but not limited to IgAN, FSGS, Alport Syndrome, post-kidney transplant

Studies evaluating the role of the endothelin pathway in proteinuric kidney diseases (e.g. IgAN, FSGS, Alport Syndrome, post-kidney transplant).
Innovative diagnostic approaches beyond biopsy and prognostic approaches for IgAN and FSGS.
Approaches to improve profiling (biomarkers, genetics, imaging, histopathology) of patients with IgAN and FSGS.
Epidemiological studies and burden of disease (clinical, economic and/or humanistic burden) in patients with proteinuric kidney diseases.
Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities.

Drug-related

Studies evaluating pre-clinical, mechanistic and clinical effects of atrasentan in patients with proteinuric kidney diseases (included, but not limited to IgAN, and post-kidney transplant), including biomarker analyses, specific patient subgroups, and broader physiological impacts (e.g. inflammation, fibrosis, and pain).
Studies on combination strategies with atrasentan in IgAN.

Out of scope

Pediatric studies (with drug).
Studies exploring different dosing regimens than currently FDA-approved dose.
Head-to-head comparisons with other approved products.
Studies including patients with eGFR <15 ml/min/1.73m² (CKD stage 5).

Disease-related: including but not limited to IgAN, C3G, IC-MPGN, aHUS, LN, AAV, FSGS, post-kidney transplant, IgAV with nephrits

Studies evaluating the role of the complement system in complement-mediated kidney diseases.
Innovative diagnostic approaches beyond biopsy and prognostic approaches for complement-mediated kidney diseases.
Approaches to improve profiling (i.e. biomarkers, genetics, imaging, histopathology) of patients with complement-mediated kidney diseases.
Epidemiological studies and burden of disease (clinical, economic and/or humanistic burden) in patients with complement-mediated kidney diseases.
Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities.

Drug-related

Studies evaluating pre-clinical, mechanistic, and clinical effects of iptacopan in patients with complement-mediated kidney diseases (e.g. IgAN, C3G, IC-MPGN, aHUS, post-kidney transplant, IgAV with nephritis) including biomarker analyses, and specific patient subgroups (e.g. high crescent, RPGN, corticosteroid-resistant).
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/1.73m².

Neuroscience

Multiple Sclerosis

The experience of use of OMB in sub-populations of RMS (e.g., AA and Hispanic patients, and age)
The impact of OMB on MS comorbidities and patient-centric outcomes
The therapeutic role of OMB in MS: Efficacy, safety, tolerability, use in treatment naive patients
The impact of OMB on both fluid and digital biomarkers in MS
The MS pathophysiology (including MoA of OMB and its effects on MS pathophysiology) and burden of disease of MS (including impact of OMB)
The innovative neuroimaging techniques used to measure biomarkers of MS disease/MS inflammation/axonal integrity and function (including effects of OMB)
The long-term impact on the immune system and long-term safety with B-cell therapies
Different B-cell depleting therapies have a differential impact on the functioning of the immune system over time, especially on the non-B-cell compartment

Multiple Sclerosis

The impact on CNS – BBB transmigration, microglial impact (activation)
The impact on biology of progression – PET imaging impact, cognition, fatigue, depression outcomes
The impact on imaging – SELs, PRLs, cortical lesions impact
The role for remibrutinib in sequencing of treatments
The proteome profiling effects of remibrutinib

Myasthenia Gravis

Impact of remibrutinib on gMG
Development of biomarkers and endpoint exploration for clinical trial use

In Scope

Demonstrating or validating care needs for SMA populations post Zolgensma IV Treatment-safety related items
Value of Zolgensma IV : Cost of care, Quality of life, and Caregiver Burden-Cost effectiveness
Methods/Processes to assess the efficacy and durability of Zolgensma IV (e.g. bulbar function)
Biomarkers for efficacy

Out of Scope

Clinical Trials involving Zolgensma IV re-dosing
Study of Zolgensma IV alternative doses/maximum dose
Basic Science research that request use of Zolgensma IV

In Scope

Interventional Studies of OAV101 IT in patients not included in clinical trials (e.g. independently ambulant SMA patients, patients >18 years, severe scoliosis) and patients with AAV9 titers >1:50
Non-interventional Studies of OAV101 IT assessing sleep, bulbar function, scoliosis and respiratory function, head steadiness and independence.
Studies on biomarkers assessing clinical response to OAV101 IT

Out of Scope

Clinical Trials involving OAV101 IT re-dosing or OAV IT dosing following OAV IV
Study of OAV101 IT alternative doses/maximum dose
Head-to-head comparison with other therapies and combination with other MDT
Studies of OAV101 IT in patients under 2 years of age.
Comparative studies between Zolgensma IV and OAV101 IT

Immunology

Sjogren's disease US epidemiology
Sjogren's disease classification and clinical assessment
Sjogren's disease progression: use of ultrasounds, clinical assessments and or biomarkers
Sjogren’s symptoms: evidence generation with existing PROs or new SjD symptom modalities
Sjogren's disease organ domains: generation of evidence in key disease domains
Sjogren's disease and concomitant conditions (i.e., rheumatoid arthritis, lupus, etc.) and outcomes
Sjogren's disease in subpopulations (AA, Hispanic, etc.) and outcomes
Sjogren's disease burden: clinical, social, economical, and humanistic aspects

Disease Related Research

Population-based epidemiology studies; studies investigating the impact of urticaria on patients; studies of real-world treatment patterns (incl. e.g. overuse of corticosteroids, impact on sleep); studies of patient preference, patient experience and satisfaction (qualitative).
Studies employing digital technology e.g. in silico models, AI-enabled/machine learning techniques, telemedicine etc. to predict disease trajectories, treatment response, disease modification, etc.
Studies investigating innovative tools to support urticaria management, e.g. digital applications, sleep related.
Long-term CSU/CindU observational studies and secondary use of data.

Clinical Studies

Studies with Remibrutinib in CSU/CindU,
Poof of concepts 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.

Optional Out of Scope

Head-to-head comparisons of Remibrutinib with other active treatments.
Studies investigating Remibrutinib in combination with biologics.
Alternative dosing regimens to 25 mg b.i.d developed in the CSU phase 3 clinical program.