IIT US Areas of Interest

Oncology

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

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
TFR and safety biomarkers
Long-term safety and tolerability
Studies aiming to improve deep molecular responses, increase the eligibility for TFR attempts or reduce the risk of relapse after treatment discontinuation.
Response to asciminib in patients with pre-existing 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 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

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

Use of 177Lu-PSMA-617 in combination with other agents in mHSPC or mCRPC
Treatments up-regulating PSMA expression in prostate cancer
Use of 177Lu-PSMA-617 in patients with low or no PSMA expression in mCRPC
Safety and efficacy of 177Lu-PSMA-617 treatment in solid tumors other than prostate cancer
Real-world evidence in prostate cancer for 177 Lu-PSMA-617
Health disparities in advanced prostate cancer
ADT sparing approaches across Prostate Cancer landscape
Investigating alternative dosing regimens (cycles, frequency) with 177Lu-PSMA-617 mCRPC
Sequencing with Actinium

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 or ²²⁵Ac-PSMA-R2 in adjuvant setting in combination with EBRT + ADT +/- abiraterone in patients with localized prostate cancer postprostatectomy with N1M0 on PSMA PET
Use of ²²⁵Ac-PSMA-617 or ²²⁵Ac-PSMA-R2 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 or ²²⁵Ac-PSMA-R2 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

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

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
Real world utilization & implementation of inclisiran post ACS and/or symptomatic PAD
Population modeling of diverse populations in various health care settings (including HCRU,...)
Differentiating attributes of inclisiran versus other LLTs (e.g., safety, drug interaction, adherence, outcomes...) in Real World setting
Effects of inclisiran in high-risk patient population (e.g., diabetes,...)

Role of complement system in complement-mediated kidney diseases
Additional ways to foster diagnosis of glomerulopathies beyond biopsy
Identification of approaches that lead to better characterization, management or correlation with outcomes in IgAN – e.g. identification of biomarkers, genetic analysis or biopsy-based studies
Burden of disease (clinical, economic, and/or humanistic burden) – IgAN
Epidemiology studies (incl. Registries) - IgAN
Mapping or intervening on the patient journey in IgAN to reduce health care costs or improve patient outcomes

Out of scope

Pediatric studies (with drug)
Studies exploring different dosing regimens as currently investigated

Role of complement system in complement-mediated kidney diseases
Additional ways to foster diagnosis of glomerulopathies beyond biopsy
Studies which attempt to clarify the histopathologic complexity/equipoise of C3G
Identification of approaches that lead to better characterization, management or correlation with outcomes in C3G, ICMPGN, aHUS, LN – e.g. identification of biomarkers, genetic analysis or biopsy-based studies
Burden of disease (clinical, economic, and/or humanistic burden) - C3G, ICMPGN, aHUS, LN
Epidemiology studies (incl. Registries) - IgAN, C3G, ICMPGN, aHUS, MN, LN
Studies on Natural History of C3G and ICMPGN in native vs transplant kidney.
Mapping or intervening on the patient journey in ICMPGN and C3G to reduce health care costs or improve patient outcomes

Out of scope

Pediatric studies (with drug)
Studies including patients with CKD stages 4 and 5
Head to head studies comparing Ipta to other treatments

Role of the endothelin system in rare renal diseases, including lgAN, FSGS, Alport
Additional ways to foster (earlier) diagnosis of rare renal diseases, beyond biopsy
Identification of approaches that lead to better characterization and/or correlation with outcomes in rare renal diseases including lgAN, Alport and FSGS
Burden of disease (clinical, economic and/or humanistic burden) in rare renal diseases, including lgAN, Alport, FSGS
Epidemiological studies in rare renal diseases, including lgAN, FSGS, Alport
Studies evaluating the mechanism of hemodilutional anemia with ERAs

Out of scope

Pediatric studies (with drug)
Studies exploring different dosing regimens to those currently being evaluated in Atrasentan CDP
Studies including patients with CKD stages 4 and 5

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

Gene Therapy

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

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
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 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