Investigator Initiated Research Opportunities | Takeda Pharmaceuticals
Investigator Initiated Research
Areas of Interest
Maribavir - US Only
Studies exploring maribavir effectiveness and safety in the real-world setting including:
Immunocompromised patients with high-risk of Cytomegalovirus (CMV) infection
Recurrent CMV
Longer term impact, early treatment (e.g., preemptive, 1st line therapy/treatment)
Diverse demographic populations (e.g., racial, ethnic)
Patterns of use
Studies focusing on effectiveness of maribavir following letermovir use or combination therapy
Studies examining the impact of maribavir on Health Related Quality of Life Measures (HRQoL) and Patient Reported Outcomes (PROs) among thoracic (heart/lung) transplant patients with CMV infection
Lanadelumab
Real-world clinical effectiveness of lanadelumab, for patients newly initiated on lanadelumab or for patients who have previous experience with alternative therapies for HAE
Novel efficacy outcomes (e.g. remission) or composite efficacy end-points (e.g. patient-reported outcomes and biomarkers) in patients with HAE treated with lanadelumab
Investigate potential for lanadelumab to modify the contact system (e.g. changes in trigger sensitivity, sustained control or suppression)
Efficacy of lanadelumab in other conditions where bradykinin or the kallikrein–kinin system may form the pathological basis of disease
HAE
Approaches to facilitating diagnosis and decreasing diagnostic delay
Characterization of non-histaminergic angioedema, including pathophysiology, prodrome, diagnosis and biomarkers
Explore the burden of illness and the burden of treatment, including paediatrics
Ulcerative Colitis (UC), Crohn’s Disease (CD) and Inflammatory Bowel Diseases (IBD)
Understanding the biological mechanisms that drive clinical response to vedolizumab in TNF-naïve/other biologics versus exposed patients with UC/CD
Characteristics (including biomarkers) of optimal vedolizumab patients
Prognostic biomarkers for responders/non-responders of vedolizumab
Transmural healing with vedolizumab
Efficacy of vedolizumab on novel PROs (e.g. bowel urgency)
Efficacy and safety of vedolizumab in combination with advanced/conventional therapeutic agents in UC and CD
Understanding biological mechanisms (including omics/histology) of combining different MoAs
Disease modification by vedolizumab in CD and UC
Real-world disease clearance by vedolizumab in UC
Real-world SC vedolizumab efficacy and safety
Data on SC vedolizumab (e.g. switching patterns from SC to IV and vice versa, different SC dosing regimens)
Data on Diversity, Equity and Inclusion in patients with IBD
Gastrointenstinal Fistulizing Conditions
Clinical outcomes
Effect of long-term seton vs short-term seton on subsequent gastrointestinal fistula treatment success
Impact of radiological healing of gastrointestinal fistula on disease recurrence
Patient care pathways and effect of multidisciplinary teams and/or patient involvement in shared decision making on gastrointestinal fistula outcomes
Gastrointestinal fistula surgeries: post-surgical complications and/or short-term Health related quality of life (HRQoL) and work productivity
Identification of optimal treatment targets in gastrointestinal fistulizing conditions leading to long-term remission
Novel treatment approaches and clinical outcomes in Crohn's-related Rectovaginal Fistula (RVF)
Translational research
Identification, development and characterization of pre-clinical in vivo proof of concept and in vitro cellular models predictive of fistulizing pathobiology
Microbiome composition in fistulizing conditions such as Complex Perianal Fistula (CPF), Complex Cryptoglandular Fistula (CCF), and Rectovaginal Fistula (RVF) (fistula swabs, stool samples)
Cellular composition and characterization of inflammation in Crohn’s disease complex perianal fistula and/or complex cryptoglandular fistula
Celiac Disease (CeD)
Adoption of new methods and technologies to diagnose and manage patients with celiac disease:
use of video capsule endoscopy (VCE) tools for diagnosing and monitoring CeD and its role in clinical guidelines
tools and studies objectively assessing real world gluten exposure
Gluten exposure/challenge design in clinical trials
Biomarkers of the future when monitoring CeD
Disease burden in special populations with highest unmet need (pediatric, adolescents, elderly)
Correlation of histology/serology/symptoms/other risk factors with disease progression/complications
Epidemiology studies
Humanistic burden and patient / healthcare professional (HCP) preferences
Disease pathophysiology (especially advances in immunology)
Optimization of gluten-free diet (GFD) when managing CeD
New modalities and development targets in CeD
New indications with relevance to gluten sensitivities/wheat allergy
Prucalopride - US Only
Pilot studies in Functional Gastrointestinal Diseases with rationale for prokinetic use
Safety and efficacy of prucalopride in secondary causes of constipation
Real world, retrospective studies of prucalopride in adults with chronic idiopathic constipation
Alpha-1 Antitrypsin Deficiency (AATD) Liver Disease
Studies focusing on identification and validation of non-invasive methods for assessment of liver fibrosis in AATD
Studies exploring predictors of disease progression in AATD liver disease or for the development and or progression of liver disease in patients with or without AATD lung disease
Studies exploring novel markers for the early identification of patients with AATD-LD and diagnostic characterization of disease stage
Studies evaluating correlations between intrahepatic globule accumulation, patient-centered outcomes and/or clinical outcomes
Dravet syndrome or Lennox-Gastaut syndrome
Disease management, treatment patterns, polypharmacy, predictors of treatment response or discontinuation, Health Resources Utilization (HRU), or long-term treatment outcomes
Novel diagnostic approaches or algorithms
Linkage of genotype to clinical phenotype and/or treatment response
Burden of illness, caregiver burden and/or treatment burden
Natural history, seizure patterns or disease progression
Novel tools or methodologies evaluating comorbidities or non-seizure symptoms, e.g. cognitive functioning, behavior, daily functioning
Epidemiology [Prevalence, incidence and diagnostic rates]
Narcolepsy
Explore the role of orexin in the pathophysiology of narcolepsy and idiopathic hypersomnia
Functional outcomes research related to treatment patterns for narcolepsy
Characterize the disease burden and unmet needs of patients with narcolepsy
Attention Deficit Hyperactivity Disorder
C&A and Adult ADHD disease management – recognition, & treatment
Functional outcomes research related to optimal and suboptimal treatment of ADHD symptoms
Lisdexamfetamine dimesylate in C&A and Adult ADHD and other comorbid conditions
Effectiveness of Lisdexamfetamine dimesylate RWE
Guanfacine use in complex ADHD patients
Guanfacine use in combination with stimulants RWE
Guanfacine effect on symptoms and functioning in ADHD patients across ages RWE
Adherence & persistence to treatment options in ADHD
Role of adjunctive psychotherapy
Binge Eating Disorder
Observational Studies on patient characteristics, comorbidities, treatment patterns, outcomes
Role of adjunctive psychotherapy
Hemophilia A
Studies examining the cost efficiency of recombinant Antihemophilic Factor (ADVATE) and PEGylated –recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) with or without myPKFiT
Studies examining the relationship between FVIII levels and the occurrence of bleeds at varying physical activity levels with or without the use of the myPKFiT mobile app
Studies to investigate changes in adherence and quality of life (QoL) in patients using recombinant Antihemophilic Factor (ADVATE) and the myPKFiT patient app
Real World Evidence on use of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI), an extended half-life rFVIII (EHL rFVIII), in clinical practice with or without myPKFiT (including safety, efficacy, utilization, QoL, adherence, patient satisfaction etc.)
Studies on other non-coagulation effects of Factor VIII
Studies looking at the GOAL-HEM (Goal Attainment Scaling for Life – Hemophilia) as a patient-centered reported outcome measure to monitor clinical progress
Non-clinical studies on Polyethylenglycol (PEG) safety
Role of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) for tolerization or in previously tolerized/partially tolerized patients
Studies to investigate appropriate assessment of joint health and prevention/management of all bleeds including subclinical
Studies to investigate benefit of FVIII replacement therapy vs. non-factor therapy in optimising bleed outcomes
Recombinant-porcine Antihemophilic Factor and Acquired Hemophilia (AHA)
Prospective or retrospective studies that provide insights on first-line use, loading dose, dosing over time, FVIII:c and anti-drug antibodies
Explore efficacy and safety of recombinant-porcine Antihemophilic Factor in patient subpopulations (i.e. post-partum or patients with specific comorbidities) with AHA
Relationship between treatment effectiveness, FVIII level and anti-pFVIII inhibitor titer in subjects with AHA receiving recombinant-porcine Antihemophilic factor
Development/validation of dosing algorithms for recombinant-porcine Antihemophilic factor, initial and follow-on, when the anti-porcine FVIII titers are unknown.
Relationship between treatment effectiveness and recombinant-porcine Antihemophilic factor dosing in subjects with AHA
Explore the potential use of recombinant-porcine Antihemophilic factor as treatment for breakthrough bleeds in patients treated with non-factor therapies [i.e. Emicizumab]
Studies intended to develop flexible and tailored dosing regimens for recombinant-porcine Antihemophilic Factor
Investigate effectiveness, safety and treatment outcomes of the continuous infusion of recombinant-porcine Antihemophilic Factor
Collect long term data on treatment for patients with AHA
Clinical outcomes of Anti-Inhibitor Coagulant Complex for treatment of AHA patients
Hemophilia Gene Therapy
- Measures to evaluate outcomes with hemophilia gene therapy (clinical, humanistic, quality of life, economic, biomarkers, etc.)
Thrombotic Thrombocytopenic Purpura (TTP)
Long-term outcomes and disease progression in TTP (congenital and or/acquired TTP) with current standard of care
Studies of the epidemiology, disease burden, and/or health care utilization of TTP patients
Genotype/phenotype correlations among TTP patients
Correlation of ADAMTS13 levels with outcomes
Investigate effects and adverse events associated with chronic plasma use
Mechanistic investigations into rADAMTS13 and TTP
Interactions of ADAMTS13 with other proteins that might affect clinical outcomes
Understanding the relationship between subclinical events and development of disease-related complications in TTP
Using clinical biomarkers as proxy for on-going development of disease-related complications in TTP
von Willebrand Disease (VWD)
Projects aiming to improve knowledge in the management of prophylaxis, heavy menstrual bleeding (HMB), post-partum bleeding, gastrointestinal (GI) bleeding, severe epistaxis, major surgeries
Comparison of plasma-derived von Willebrand factor (pdVWF) and recombinant von Willebrand factor (rVWF) in the treatment of GI bleeds or HMB
Effectiveness and safety of von Willebrand factor (VWF) in on demand/surgery and prophylaxis in real world setting (including elderly patient or those having a cancer or thrombotic risk)
Personalization of VWD therapy (genetic, bleed prediction, bleeding assessment tools, multidisciplinary approach, …)
Studies to examine the relationship(s) between the rVWF characteristics, its half-life, its multimeric profile and its clinical efficacy
Role of VWF multimeric forms in the control of angiogenesis including biomarkers of angiogenesis (rVWF in angiodysplasia)
Impact of long-term management of joint bleeds on quality of life (QoL) and/or healthcare resource utilization (HRU); impact of the management of HMB on QoL and HRU
Assessment of the efficacy of VWF (pdVWF or rVWF) in acquired VWS – left ventricular assist device (LVAD), extracorporeal membrane oxygenation (ECMO)
Adrenal Insufficiency
We are not accepting new IIRs for this area
Hypoparathyroidism
Hypothesis generating studies on the beneficial effects of Natpar/a on short-term symptomatology and quality of life and long -term function of organs/systems (i.e., Brain, CVD, Renal, Bone, Glucose metabolism, immune function) in chronic HypoPT patients not ade - quately controlled with conventional therapy
Physiology of interaction between PTH N-terminal and C-terminal with the PTH Receptors PTHR1 and PTHR2, circadian rhythm & clinical implications
In-vivo and in-vitro mechanism of action (MOA) including non-clinical studies
Hunter Syndrome
Analyses of natural history of MPSII
Studies involved in the development or evaluation of biomarkers or diagnostic capabilities in MPS II
Studies examining:
Impact of early diagnosis, including screening, and treatment start
Long term treatment effectiveness of Idursulfase (also retrospective)
Early diagnosis of cognitive impairment
Neurodevelopmental assessment
Standardization of cognitive testing
Neuronopathic MPS II Disease course
Studies of genotypes and phenotypes, as well as their correlation, in different geographies
Fabry Disease
Studies for the development or evaluation of biomarkers or diagnostic capabilities to:
Identify high risk populations
Facilitate early disease detection
Detection of early disease progression
Monitor disease progression
Predict/measure therapy effectiveness
Research on cardiac treatment outcomes
Studies of genotypes and phenotypes, as well as their correlation, in different geographies
Studies on disease progression in specific agalsidase alfa treated sub populations or segments (e.g., females and pediatrics subjects).
Studies on early treatment
Studies examining outcomes in patients with amenable mutations
Research fostering understanding of inflammation and immunogenicity in Fabry disease (FD)
Gaucher Disease
Screening for patients with Gaucher disease
Generation of data on patient reported outcomes (PRO)
Generation of data on the role of Lyso-Gb1 as a novel biomarker
Studies fostering understanding of inflammation and immunogenicity in Gaucher disease
Enzyme replacement therapy (ERT) infusion optimization (e.g. location (hospital/home), infusion pumps, etc.) that could have potential positive impact on patient experience and patient Quality of Life (QoL)
Genotypes and phenotypes
Mucopolysaccharidosis Type II (MPS II) / Hunter Syndrome
Studies examining impact of early diagnosis, including screening, and treatment start
Exploring the utility of digital technologies for achieving earlier diagnosis
Areas relating to implementation of newborn screening (NBS) programs e.g. feasibility and pilot studies; follow-up on outcomes in patients identified by NBS programs
Studies of the long-term effectiveness of Idursulfase
Studies on the natural history of MPS II
Studies exploring the development or evaluation of biomarkers or diagnostic capabilities in MPS II
Support lab and imaging research of disease markers and surrogates predictive of MPS II, including its neuronopathic form.
Development of novel methods to diagnose neuronopathic MPS II early, including neurocognitive/behavioral assessments
Studies examining neuronopathic MPS II Disease course
Improve understanding of genotype phenotype relationship in MPS II
Novel utilization of Projected Retained Ability Score (PRAS) methodology in research and clinical practice
Rare autoimmune diseases (RAID), including chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), multifocal motor neuropathy (MMN) and other peripheral neuropathies
Studies intended to improve diagnosis and care
Research that intends to identify biomarkers to improve diagnosis or to measure treatment outcome
Studies that assess and evaluate QoL, personalized health, and perceived health
Immunodeficiencies
Secondary Immunodeficiency (SID)
Studies that intend to understand the course of infections in patients with cancer-and treatment-related SID (especially chimeric antigen receptor (CAR) T-cell therapy, posthematopoietic stem cell transplant (HSCT), and solid organ transplantation (SOT)), for example: timing of infections, frequency, types, and impact on patient outcomes and treatment
Studies that aim to establish or validate existing predictive model or to identify single biomarker that could help to select patients at high risk of developing infections in cancer related and treatment related SID
Studies with a focus on understanding who would benefit the most from prophylactic or on demand immunoglobin (IG) therapy
Analyses that quantify and qualify the impact of SIDs and/or impact of IG therapy (prophylactic and on-demand) vs standard of care (SOC) on clinical and patients’ related outcomes (e.g. efficacy, long term safety, quality of life, personalized health, and perceived health)
Primary Immunodeficiency (PID)
Real World Evidence studies that provide insights into usage and administration parameters of subcutaneous immunoglobulin (SCIG) products, particularly in special populations (pediatric, elderly, obese patients)
Studies that assess and evaluate QoL, personalized health, and perceived health for IG replacement therapy
Studies that assess and evaluate the application of new device or digital health concepts in the management of PID
Studies that explore novel or validate existing approaches in support of earlier PID diagnosis – especially those that could have global impact
Studies with a focus on understanding the full presentation of PID including the impact of comorbidities to PID and/or vice versa
Albumin
Clinical and analytical studies related to the concept of functional albumin
Clinical experience with Flexbumin in patients with liver disease, and patients managed in intensive care units (ICUs)
Central line-associated bloodstream infections (CLABSIs): Burden of illness, pharmacoeconomic impact and evaluations of prevention strategies
Clinical outcomes of hyperoncotic (25% albumin) Flexbumin in patients with liver cirrhosis and critical care
Impact of closed infusion system in albumin therapy on albumin storage, preparation, and handling
Identification of biomarkers for albumin treatment in liver disease and critically ill patients
Alpha1-antitrypsin (AAT) deficiency (AATD) and AAT Augmentation Therapy:
AATD pathogenesis, mechanisms, and biomarkers
AATD clinical, humanistic, and economic burden of illness and co-morbid conditions
Characterization of MZ patients
Impact of early diagnosis and screening
AAT Augmentation Therapy: for example, but not limited to:
Adherence in AATD patients with emphysema/chronic obstructive pulmonary disease (COPD)
AAT deficient patients in specific populations, (i.e. young adults/adolescents/older/post lung-transplant/forced expiratory volume (FEV) 1 35% Predicted)
Exploratory research on anti-inflammatory, immunomodulatory, and tissue protective effects of AAT therapy in non-AATD individuals (i.e. graft vs host disease, autoimmune, rheumatoid arthritis, cardiac)
C1-esterase inhibitor
Real-world data on effectiveness and safety of on demand use, as well as pre-procedural treatment, short-, and long-term prophylaxis with C1-esterase inhibitor in HAE patients
Understand dosing of C1-esterase inhibitor in the real-world setting
Switching from oral HAE medications to C1-esterase inhibitor (including, but not limited to, reasons and patients` perception/preferences/reported outcomes)
For disease related, non-product specific areas of interest please visit Hereditary Angioedema (HAE) section of the website (see above).
Hemophilia A and B Inhibitor prevention and management
Pre-clinical and clinical studies examining the care and treatment of persons with inhibitors and the use of Anti-Inhibitor Coagulant Complex to control and prevent bleeding episodes and perioperative management in adults and children as well as its impact on quality of life (QoL).
Studies exploring the potential immunotolerance effect of Anti-Inhibitor Coagulant Complex prophylaxis during Immunotolerance induction (ITI) in hemophilia patients with inhibitors.
Studies examining clinical effectiveness and pharmacoeconomic aspects of using Anti-Inhibitor Coagulant Complex in prophylaxis as well as in ITI.
Studies examining the use of Anti-Inhibitor Coagulant Complex in prophy-laxis with low dose regimens: regimens, clinical outcomes, pharmacoeconomic implications, QoL
Studies examining potential correlations between thrombin generation assay (TGA) parameters and clinical outcomes.
Investigations of differences in patient profiles for hemophilia patients with and without inhibitors, and possible correlations with prediction, prevention, eradication and prophylaxis of inhibitors.
Explore whether knowledge from other immunological disease states can be used to develop better management of inhibitor patients.
Investigate safety profile of Anti-Inhibitor Coagulant Complex to identify possible predictors of outcomes.
Investigate the use and cost effectiveness of low dose FVIII ITI with Anti-Inhibitor Coagulant Complex as compared to high dose FVIII ITI alone. Investigate the use and cost effectiveness of low dose FIX ITI with Anti-Inhibitor Coagulant Complex as compared to high dose FIX ITI alone or with other components
Preclinical and clinical studies examining the interaction between the co-administration of Anti-Inhibitor Coagulant Complex and non-factor replacement therapies: regimens and doses, clinical outcomes, safety (TMAs & TEEs), pharmacoeconomic implications, global assays.
Investigate the use of Anti-Inhibitor Coagulant Complex in other diseases where it is required to reestablish the coagulation cascade (i.e. vitamin K deficiency, reversal of NOACs, hepatopathy, etc.)
Brentuximab vedotin - Outside the US and Canada
Combination studies in Hodgkin’s Lymphoma with programmed cell death protein 1 (PD-1) / programmed cell death ligand 1 (PD-L1) inhibitors
Studies which evaluate CD30 expression level, binding and resistance, and reversibility of peripheral neuropathy
Studies with CD30 expressing Peripheral T-cell lymphoma (PTCL) in front-line therapy and combination studies in relapsed/refractory PTCL
Contact Seagen for US or Canadian proposals
Brigatinib - Global
- Studies investigating brigatinib in non-small cell lung cancer (NSCLC), evaluation of CNS activity, rational combinations with other agents/modalities, and sequencing of ALK TKIs
Cabozantinib – Japan Only
Studies following solid tumor refractory to standard-of-care (SOC) therapy
Studies exploring predictors of efficacy and to assess novel combination therapies for renal cell carcinoma
Studies examining actual treatment for hepatocellular carcinoma
Studies of side effect management
Contact Exelixis for any proposals outside Japan
Fruquintinib - Global except China, Hong Kong, and Macau
Strong interest:
Combination studies with fruquintinib in metastatic colorectal cancer (mCRC), utilizing agents with a clear scientific rationale
Descriptions of the observed effectiveness and tolerability of fruquintinib in CRC in the clinic population and/or identification of subgroups who may derive the most benefit from the therapy
Generation of observational data characterizing treatment patterns, safety, and outcomes for patients with refractory mCRC to identify optimal sequencing of agents/mechanisms of action (MOAs)
Moderate Interest:
Studies exploring fruquintinib as maintenance therapy following chemotherapy in mCRC with/without single agent chemotherapy
Studies exploring fruquintinib in earlier lines of therapy in mCRC
Studies exploring fruquintinib in pre-metastatic CRC
Studies exploring fruquintinib as adjuvant treatment in patients with resected locally advanced CRC and MRD positive disease
Ixazomib - Global
Currently not accepting proposals
Mobocertinib - Global
Currently not accepting proposals
Modakafusp alfa (TAK-573) – Global
- Preclinical/nonclinical proposals in hematological malignancies
Niraparib – Japan, South Korea and Taiwan Only
Studies on overcoming resistance to PARP inhibitors and platinum-based anti-cancer agents
Studies on improving adherence and promoting proper usage
Studies exploring predictors of efficacy and to assess novel combination therapies for ovarian cancer and PARP inhibitors
Studies following solid tumor refractory to standard-of-care (SOC) therapy
Contact GSK for proposals outside of Japan, South Korea and Taiwan, excluding any prostate cancer proposals in South Korea and Taiwan
Panitumumab – Japan colorectal cancer indication Only
Currently not accepting proposals
Contact Amgen for any colorectal cancer proposals outside Japan
Dengue Disease
Impact of co-circulation of dengue and COVID-19 on the healthcare system and resources, reporting of dengue, on patient outcomes and quality of care.
Impact of long-term persistence of dengue symptoms on quality of life and cost to dengue patients and their families; out of pocket financial impact related to dengue borne by families/households
Studies examining predicted relative morbidity gain (QALY/DALY) in dengue prevention vs other infectious disease preventions
Impact of socioeconomic factors and urban/rural location on probability of dengue patients to present for medical care
Epidemiology studies to determine Dengue seroprevalence (national, rural, urban, by age); age groups most at risk of severe dengue outcomes
Studies to determine extent of dengue under-reporting in Dengue endemic setting
Burden of Dengue on tourism and migration (Travelers)
Questions about our IIR program?
Please contact
Rare Diseases, Neuroscience, Gastroenterology, and Plasma Derived Therapies (excluding US): [email protected]
Clinical Oncology: [email protected]
Preclinical Oncology: [email protected]
Vaccines: [email protected]
US (excluding Oncology): [email protected]
Japan: More information available on this page (Japanese)
Disclaimer
Takeda and its alliance partners are committed to improving patient care by supporting scientific advances in medicine and increasing our understanding of important diseases. As part of this commitment, the IIR program supports innovative interventional and noninterventional and basic science studies that address important medical and scientific questions related to our compounds and therapeutic Areas of Interest (AOI).
IIR is defined as an unsolicited research study where the Investigator, organization or institution (academic, private, or governmental) serves as the Sponsor, and Takeda provides support only in the form of funding, study product, safety information and/or authorization to reference Takeda’s NDA or other regulatory submissions (e.g., IND). Takeda does not provide input in an IIR other than providing the above support.
Takeda reviews completed proposals both within and outside of the Areas of Interest (AOI) listed throughout this document.
All proposals will be evaluated in adherence with Takeda policies and all applicable laws and regulations. Decisions are based upon scientific merit as well as alignment with research areas of interest and availability of resources. Submission of a proposal does not imply or guarantee approval. Support of a study in no way implies any obligation toward or is any way connected to the recommendation or prescribing of products.