The Novo Nordisk Investigator Sponsored Studies (ISS) Program will accept Submissions within our current scientific focus areas as noted below. This is a competitive process. Incomplete submissions or protocols that are not within the scope of these focus areas/have low relevance to the therapeutic area may be rejected without further review. Submissions will be reviewed by the relevant Novo Nordisk teams. Decisions will be made based on scientific merit and strategic fit. Please review the submission requirements. The program requests that investigators specify how they will support enrollment of diverse populations in the study.
Scientific Focus areas :
- Haemophilia with and without Inhibitors
- Other Congenital Bleeding Disorders including platelet disorders
- Acquired Haemophilia (AH)
- Combination therapy (NN factor therapies with NFTs)
- New outcome measures in haemophilia (PROs or clinical)
- Joint Health (including biomarkers of joint inflammation and degradation as well as imaging)
- Laboratory and diagnosis in the areas of interest
- Inhibitor prevention research
- Haemophilia and Ageing (with or w/o co-morbidities)
- PK studies in the areas of interest
- Health Economic in the areas of interest
- Haemophilia Covid-19-related studies
- Moderate/mild haemophilia (unmet medical needs / treatment) including women
- Treatment and disease burden; patient unmet needs
- Individualised care
Areas out of Scope:
- Studies conflicting with NN sponsored LCM plans
- Marketing, product promotional and market research studies
- Blood transmitted disorders
- Studies outside of bleeding disorders (except FXIII area)
- Studies on PEG Antibodies or PEG safety (unless required by HAs)
- Non-bleeding related disorders (i.e. malignancies, thrombosis)
Scientific focus areas:
- Laboratory monitoring of rFVIIa therapy including global assays
- Congenital Haemophilia with inhibitors and other congenital rare bleeding disorders
- Bleeding Phenotype
- Inhibitor screening studies (developing countries)
- Combination with antifibrinolytics, bispecific antibodies, novel MoA
- NovoSeven for pre- and during ITI
- Health economic research
- Surgery including other rare bleeding disorders
- Critical bleeding (sPPH, ICH, potentially cardiac surgery)
- Safety (esp. in new indications)
- Long-term experience
Areas out of scope:
- Non-neutralising antibodies research
- Warfarin and tPA overdose reversal
- Continuous infusion
- Comparator studies with FEIBA or biosimilars
- Combination therapy with FEIBA
Scientific focus areas:
- Diagnostic assays and laboratory monitoring including global assays
- Surgery in FXIII CD
- Genotyping
- Multi-centre, National and International Screening programs
- Bleeding phenotype
- Outside of bleeding area ( IBD, osteoporosis, wound healing, enterocolitis, etc.)
- FXIII CD Observational studies
Areas out of Scope :
- Cardiac surgery trial
- Comparator studies with Fibrogammin/Corifact
Scientific focus areas:
- FIX distribution, incl. extravascular distribution
- Real World Data (supporting value of high trough):
- prophylaxis use
- surgery data
- switching studies
- joint health
- PK SHL and EHL factor IX and bleed rates
- Moderate/mild haemophilia (unmet medical needs / treatment) including women
- Adherence to treatment
- Health and Patient Outcomes research
- Health economic research
- Monitoring (validated one-stage and chromogenic assays)
- Genotyping (CRM status)
Areas out of scope:
- Studies on PEG Antibodies or PEG safety (unless required by health authorities)
- Neurocognitive and neurological studies (unless required by health authorities)
- PK-tailored dosing with Refixia
Scientific focus areas:
- Diagnosis (incl new-born screening)
- Laboratory monitoring including global assays
- Genotyping
- Bleeding phenotype
- PK and PK individualized therapy (incl physical activity)
- Immune tolerance induction
- Real World Data:
- switching products
- Joint health outcomes
- Treating breakthrough bleeds in patients on NFTs
- Health and Patient Outcomes research
- Surgery and Continuous infusion for surgical intervention
- Imaging studies
- Moderate/mild haemophilia (unmet medical needs / treatment) including women
- Elderly patients (with or w/o co-morbidities)
- Use of DDVAP, antifibrinolytics + FVIII or other combination therapy
- Inhibitor prevention
Areas out of scope:
- Comparison with BDD products
- Non-neutralising antibodies to turoctocog alfa
- Studies on PEG Antibodies or PEG safety (unless required by health authorities)
Scientific focus areas:
- CZM MoA and TFPI biology
- TFPI pools/isoforms
- Clinical situation of interest
- Major surgery cases
- Long-term outcome
- Joint Health
- QoL
- Adherence measured
- Assays/Monitoring
- Concizumab haemostatic activity
- FVIII and FIX equivalence
- Potential impact of concomitant therapies
- Switch data to concizumab
- Pre-Clinical & Clinical evidence in other RBDs
- (AH, GT, VWD, FVII-/-, FV-short, …)
Areas out of Scope :
- Studies conflicting with concizumab NN-sponsored LCM plans
- Animal studies
- Head-to-head studies vs other NFT competitors
- Dosing regimen & adjustment (incl. IVD)
Scientific focus areas:
Preclinical
- Mim8 MoA and FVIIIa mimetic biology
- Evaluation of Mim8 in individual donors
- Assays/Monitoring
- Mim8 haemostatic activity (high interest)
- Potential impact of concomitant therapies
Clinical
- Clinical use and indications:
- Major surgery cases(high interest)
- Mild-moderate haemophilia (high interest)
- Subclinical joint bleed (high interest)
- Verification of bleeding
- Biomarker research (high interest)
- Mim8 use in ITI
- Switch data to Mim8
- Other indications e.g. VWD
- Long-term outcome
- Joint Health(high interest)
- Adherence
- Quality of Life (QoL)
- Impact of device on treatment burden and QoL
- Health economic research
Areas out of scope :
Preclinical
- In-vivo pre-clinical studies using haemophilia animal models
- Concomitant use (mixing) of Mim8 with other hemostatic agents.
Scientific focus areas: (AGHD only)
- Sogroya® authorised indications – AGHD
- Adherence / Persistence / treatment initiation
- Traumatic Brain Injury
- Metabolic outcomes (e.g. lipid metabolism)
- Impact on Liver function / hepatic fat (e.g. AGHD with NAFLD / NASH comorbidity)
- Switching from daily GH
- Combination treatment somapacitan & GLP-1
- Titration
- Health Economic (e.g. work productivity), PRO/QoL
- Other proposals synergistic and not in conflict with Novo Nordisk research/scientific commitment in the therapeutic area
Areas out of scope
- Off-label somapacitan use
- Abuse (i.e. anti-ageing, doping)
- Proposals in conflict with Novo Nordisk research or scientific priorities
- Request for placebo
Scientific focus areas:
- Sogroya® authorised indications – GHD where approved
- Sogroya® use in children with GHD below 2.5 years
- Use in paediatric Prader-Willi patients
- Adherence / Persistence / treatment initiation
- Prediction of growth during Sogroya® therapy
- Metabolic outcomes (e.g. lipid metabolism)
- Impact on body composition
- Switching from daily GH
- Switching from weekly GH
- Impact on energy levels, QoL
- Other proposals synergistic and not in conflict with Novo Nordisk research/scientific commitment in the therapeutic area
Areas out of scope
- General off-label use in non-short stature patients
- Use in paediatric non-replacement indications currently under clinical investigation in the REAL programme
- Abuse (i.e. anti-ageing, doping)
- Proposals in conflict with Novo Nordisk research or scientific priorities
- Request for placebo
Scientific focus areas:
Sickle Cell Disease (SCD)
- Markers of disease progression and other biomarkers
- Improving newborn screening
- Advancing the understanding of SCD pathophysiology, management and treatment compliance
- End-Organ damage and other SCD-related complications
- Understanding pain in SCD
- Transition to Care models
- Optimizing outcomes and clinical benefit of Novo Nordisk molecules
- Effect of Novo Nordisk molecules on RBC functions
- (Others): Protocols not in conflict or redundant with NN research/scientific commitment in the therapeutic area.
Thalassemia
Optimization of diagnosis
Understanding burden of disease for NTDT and TDT
Pathophysiology of alpha or beta Thalassemia
Markers of disease progression
Advancing the understanding of disease-related and treatment-related complications
Optimizing outcomes and clinical benefit of NN molecules
Others: Protocols not in conflict or redundant with NN research/scientific commitment in the therapeutic area
Scientific focus areas:
- Optimization of diagnosis
- Pathophysiology of Primary Hyperoxaluria (PH)
- Markers of disease progression
- Improving diagnostics and Monitoring Tools
- Advancing the understanding of PH management
- Optimizing outcomes and clinical benefit of Novo Nordisk Molecules
- Other: (Protocols not in conflict or redundant with NN research/scientific commitment in the therapeutic area)
- Application form.
- Investigator Curriculum Vitae (signed and dated within the last year)
- Active medical license (e.g., if requesting study drug)
- Conflict of Interest form