IIT US Areas of Interest

Lymphoid

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

Myeloid/Malignant Hematology

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

Non-Malignant Hematology

With drug:

Mechanistic studies in PNH
Studies evaluating complement Factors associated with or predictive of treatment outcome in PNH
Studies exploring Factor B inhibition in other disease states where complement plays a role

Without drug

Role of complement system in complement-mediated PNH
Approaches to facilitating and expediting diagnosis of PNH
Identification of biomarkers that leads to better characterization, management or correlation with outcomes in PNH
Epidemiology studies (incl. registries) – PNH

Breast and Gynecological Cancers

Breast cancer

Ribociclib in early breast cancer and metastatic breast cancer (limited scope)

Out of scope

All other studies and tumor types

RLT

Investigating alternative dosing regimens (cycles, frequency) with ¹⁷⁷Lu-PSMA-617 in mCRPC
Radioligand therapy in neoadjuvant setting for localized prostate cancer
Use of ¹⁷⁷Lu-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 ¹⁷⁷Lu-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 ¹⁷⁷Lu-PSMA-617 in combination with other agents in mHSPC or mCRPC
Treatments up-regulating PSMA expression in prostate cancer
Use of >6 cycles of ¹⁷⁷Lu-PSMA-617 in patients with mHSPC or mCRPC
Use of ¹⁷⁷Lu-PSMA-617 in prostate cancer patients with distinct mutations (e.g., PTEN-loss, AKT, DDR)
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

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 other non-GEP NET SSTR2+ tumors

CVM

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 (e.g. kidney disease, diabetes)

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

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

Quantification of Lp(a) role in CV risk assessment tools

Quantification of Lp(a) contribution to global CV risk and in light of other CV risk factors
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
Guidance on management of currently modifiable risk factors in the setting of elevated Lp(a)

Out of scope

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

Mechanistic studies in post ACS/ symptomatic PAD
Real world utilization & implementation of inclisiran post ACS/symptomatic PAD
Inclisiran in under-represented patient population (eg, women, pts with treatment disparities in LDL-C lowering, patient types who tend to have worst outcomes)
Characterization of the effect of inclisiran on lipoprotein metabolism: particle synthesis/secretion, including Lp(a)
Populaton modeling of diverse populations in various health care settings (including HCRU,...)
Effects of inclisiran in high-risk patient population (e.g., diabetes,...)
Differentiating attributes of inclisiran versus other LLTs (e.g., safety, drug interaction, adherence,..)
Preclinical studies evaluating non-LDL-C-lowering effects

Iptacopan for Igan Indication

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 regiments as currently investigated
Any study, which combines iptacopan with immunosuppressant

Iptacopan for C3G indication

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

Atrasentan for IgAN Indication

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

In scope

Demonstrating or validating care needs for SMA populations post OAV101 Treatment-safety related items
Expansion of treatment with OAV101 to patient populations not included in clinical trials (e.g. older/heavier, 4 copies, switch therapy, ambulatory)
Value of OAV101: Cost of care, Quality of life, and Caregiver Burden-Cost effectiveness
Methods/Processes to assess the efficacy and durability of OAV101 (e.g. bulbar function)
Biomarkers for efficacy

Out of scope

Clinical Trials involving OAV 101 re-dosing
Study of OAV101 alternative doses/maximum dose
Basic Science research that request use of OAV101

In scope

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

Out of scope

Clinical Trials involving OAV 101 re-dosing
Study of OAV101 alternative doses/maximum dose
Head-to-head comparison with other therapies and combination with other MDT
Studies of OAV IT in patients under 2 years of age.

Dermatology

Approaches to facilitating and expediting diagnosis and treatment of HS to modify disease progression
Development of novel imaging techniques to investigate the role of secukinumab in limiting progression of HS and PsO
Machine learning techniques to create predictive models for disease trajectories, and IL-17A inhibition responses across disease subpopulations (e.g. disease phenotypes, Black/African American, super-responders, etc.)
Essential factors (such as biomarkers) for predicting disease and treatment outcomes
Burden of disease (clinical, economic, and/or humanistic burden) – HS and PsO
Development and validation of scoring tools and patient reported outcomes in HS

Out of scope

Dermatologic conditions other than psoriasis and hidradenitis suppurativa

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 (i.e., rheumatoid arthritis, lupus, etc.)

IITs in these products are no longer supported