Rare Diseases Investigator Sponsored Studies Program

Considerations for submissions to the Novo Nordisk, Inc. (NNI) Investigator-Sponsored Studies (ISS) Program:

We accept protocols aligned to the Areas of Interest listed below. Protocols outside the Areas of Interest are also accepted, but are not as likely to be awarded.
Please review the FAQ section for additional information
Please review and utilize the NNI ISS protocol template located in the online system (inRich). Links can be found in the “Submit your ISS” section

Areas of interest

Preclinical
- In-vitro studies on FVIII equivalency
- In-vitro studies on assay interference and activity in non-HA rare bleeding disorders
Clinical
- Clinical use and indications:
  - Surgical cases, especially major surgery
  - Subclinical joint bleed
  - Biomarker research
  - Real world outcomes
  - Quality of life (QoL)
- Long-term outcome
  - Joint Health
    - Synovitis resolution and/or prevention
    - Prevention of joint damage
- Physical activity

Areas not in scope

Clinical
- Studies conflicting with NN sponsored trial program (Acquired Haemophilia and VWD)
- Head-to-head studies with clinical outcomes
- Monitoring of denecimig (Mim8) level and its clinical impact
- Reduced dosing frequency
- Assessment of clinical samples from FRONTIER trial patients
- Studies that overlap with completed, ongoing or planned research (Find Study Results)

Areas of interest

Clinical situations of interest such as major surgery cases
Long-term outcomes: Joint health, quality of life, adherence
Data related to switching from a product to Concizumab
In vitro and clinical evidence in other RBDs (including but not limited to Acquired Hemophilia, Glanzmann’s thrombasthenia, von Willebrand Disorder, congenital FVII deficiency)

Areas not in scope

Optimization of diagnosis
Pathophysiology of Primary Hyperoxaluria (PH)
Improving diagnostics and Monitoring Tools
Advancing the understanding of PH management
Optimizing outcomes and clinical benefit
Protocols not in conflict or redundant with NNI research/scientific commitment in the therapeutic area

Areas not in scope

Studies that overlap with completed, ongoing or planned research (Find Study Results)

Somapacitan authorized 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 MAFLD/MASH comorbidity)
Switching from daily GH; switching between long-acting growth hormone brands
Combination treatment somapacitan & GLP-1
Titration and Insulin-like growth factor -1 (IGF-I) excursions
Health Economic outcomes (e.g., work productivity), PRO/QoL
Transition GHD to adulthood
Other protocols not in conflict or redundant with NNI research/scientific commitment in the therapeutic area

Studies that overlap with completed, ongoing or planned research (Find Study Results)
Anti-aging or performance enhancing purposes
Request for placebo only

Somapacitan authorised indications – GHD where approved
Somapacitan use in children with GHD younger than 2.5 years of age
Use in paediatric Prader-Willi patients
Adherence/persistence/treatment initiation
Prediction of growth during somapacitan therapy
Metabolic outcomes (e.g., lipid metabolism)
Impact on body composition
Switching from daily GH
Switching from weekly GH
Impact on energy levels, QoL
Transition GHD to adulthood quality of life
Endocrine sequalae in cancer survivor and long-acting growth hormone use
Other protocols not in conflict or redundant with NNI research/scientific commitment in the therapeutic area

Studies that overlap with completed, ongoing or planned research (Find Study Results)
Use in paediatric non-replacement indications currently under clinical investigation in the REAL program
Anti-aging or performance enhancing purposes
Request for placebo only