Investigator sponsored studies
An Investigator Sponsored Study (ISS) is an unsolicited preclinical, clinical, outcomes, or disease-state study.
In an ISS, an investigator independently generates a research proposal, and if approved Sobi provides support for the proposal. Support can include, but is not limited to, drug material, expert advice, funding to pay for expenses directly related to the research based on fair market value, or a combination of these items. The investigator, or the institution the investigator is affiliated with, serves as the study sponsor. As such, the sponsor assumes responsibility for initiating and conducting the study, directing the administration of the study drug (if applicable), ensuring compliance with all local laws and regulatory requirements, and analysing and communicating all study results.
Sobi recognises the important role that ISSs can play in expanding the knowledge related to Sobi's products and their associated disease areas. This research can advance science and contribute to the development of better medicines for patients consistent with the company's overall research and global development strategies.
Areas of interest
Haematology
Efmoroctocog alfa/eftrenonacog alfa
Haemophilia
Clinical studies evaluating:
- Personalization of prophylaxis and outcome measures
- Role(s) of factor VIII beyond coagulation
- Biomarkers of microbleeding, bleed resolution, inflammation, and joint damage
- Immunogenicity and induction of immune tolerance
- Joint health and dysfunction
- Management and outcome of mild-moderate hemophilia
- Clinical outcome in special populations (e.g., elderly, with comorbidities)
- Patient Reported Outcomes (PROs) and health-economic studies
Avatrombopag
Immune thrombocytopenia (ITP)
- Clinical studies evaluating the use of avatrombopag in newly diagnosed and persistent ITP patients
- Studies investigating the differentiation of avatrombopag vs other second-line treatments
- Patient-reported outcomes in avatrombopag treatment (e.g. fatigue, HRQoL)
Thrombocytopenia secondary to other diseases/conditions:
- Studies evaluating the use of avatrombopag in other diseases/conditions
Pegcetacoplan
Paroxysmal nocturnal haemoglobinuria (PNH)
Studies evaluating the clinical management of PNH
- Treatment transition strategies & dosing strategies
- Efficacy in specific subpopulations
- PNH in the context of bone marrow failure
- Biomarker analysis
Studies evaluating patient reported outcomes
- Quality of Life (QoL) and effect on Activities of Daily Living (ADL)
- Longer-term treatment effects
Studies evaluating:
- Biomarkers of disease severity
- Epidemiology of PNH
- Further characterisation & measurement of PNH symptoms (e.g., brain fog)
- Iron deposit status at diagnosis and evolution during the course of treatment
- General inflammatory activation at diagnosis and evolution during the course of treatment
Complement 3 glomerulopathy (C3G)/Immune complex membranoproliferative glomerulonephritis (IC-MPGN)
- Studies evaluating the clinical management of C3G/IC-MPGN including natural history of disease, Efficacy in specific subpopulations as well as biomarkers or novel endpoints of disease severity and disease progression
- Studies evaluating patient reported outcomes like Quality of Life (QoL) and effect on Activities of Daily Living (ADL)
- Studies evaluating the Role of the complement system and mechanisms of glomerular injury
Studies evaluating the use of pegcetacoplan for the treatment of C3-mediated diseases/conditions
Loncastuximab tesirine
- Preclinical/translational and clinical studies assessing the sequencing of CD19-targeted therapies in aggressive B-cell malignancies
- Clinical studies in diffuse large B cell lymphoma including combination approaches that may improve the level of care and/or be disease-modifying
- Real-world or health outcomes studies, and preclinical/translational studies exploring markers of response, mechanisms of resistance, or mechanisms of toxicity of loncastuximab tesirine
Pacritinib
- Clinical studies in inflammatory or malignant diseases that may be driven by JAK2, NFkB, CSF1R, or other pacritinib-targeted kinases
- Clinical studies in chronic myelofibrosis including combination approaches that may improve the level of care and/or be disease-modifying
- Real-world or health outcomes studies, and preclinical/translational studies exploring pacritinib’s mechanism of action.
Immunology
Anakinra
Studies assessing the management of severe IL-1 driven autoinflammatory conditions.
Emapalumab
Clinical studies evaluating:
- Inflammatory diseases or conditions that may be driven by IFNγ
Non-interventional studies and preclinical studies evaluating:
- The role of IFNγ in driving disease pathophysiology
- Biomarkers
Review process
Proposals will be reviewed by an ISS Evaluation Committee (ISS EC) consisting of personnel from medical affairs, research and development, biostatistics and safety.
In order to be approved for support by the ISS EC, a study must meet all the following requirements:
- The defined objective of the ISS is aligned with the current ISS strategy for the medicinal product and therapeutic area.
- The study is based on thorough scientific knowledge with careful assessment of risks and benefits, addresses a medical need of the population studied, is conducted by suitably trained investigators using approved protocols, and is subject to independent ethical review and oversight.
- The intent of the study is to obtain scientific data to be shared with the scientific community.
- The Investigator is committed to execute the contractual agreement and to provide regular study updates, as requested by Sobi.
- The Investigator must attest that they are capable of and will comply with all relevant GCP-required Sponsor responsibilities and/or other applicable regulations (clinical studies only).
- The sample size is adequate to the objective of the study and is based on sound scientific, statistical and clinical assumptions (clinical studies only).
- The Investigator´s commitment to follow Sobi's requirements for ISS support including clinical trial registration in public repositories (e.g. clinicaltrials.gov, as applicable), delivery of a final study report no later than 1 year after the Last Patient Out (LPO) date (clinical studies only).
In addition to the scientific evaluation described above, the ISS EC will ensure that requested funds are justified, reasonable and commensurate with fair market value, and that submitting investigators are eligible to conduct the proposed research. Sobi will not compensate for educational and training activities, purchase of equipment related or unrelated to the study or which could generate revenue to the Sponsor and staff that are not dedicated to the study.
Sobi's local medical representative will present the study proposal to the ISS EC. Therefore, it is highly recommended that investigators contact their local Sobi Medical Science Liaison (MSL) or local Medical Director prior to submitting a study proposal, to address any questions and to ensure the proposal and associated budget are complete and compliant. Investigators who do not know their local Sobi contact should email to [email protected] for assistance and contact details.
Official notification of the Sobi ISS EC decision to 1) approve support, 2) approve support with changes, or 3) decline support will be communicated within approximately two months following each submission deadline, by a Sobi representative.
Submission requirements
Using the Sobi ISS portal , investigators must submit a detailed proposal supported by pre-clinical or clinical data with strong scientific rationale and/or an appropriate review of the literature. The link to the portal can be found below, together with a User Guide Reference. An up-to-date CV of the investigator as well as a detailed budget breakdown must be submitted together with the application.
Once a proposal is reviewed and approved, the investigator will be invited to submit a full protocol for review to ensure consistency with the submitted study application. Please note, approval of a proposal does not imply or guarantee approval of a full protocol. For final sign-off of clinical studies, a copy of the ethical approval must be provided, when applicable. Any support approved by Sobi is initiated only upon a fully executed contract and with all necessary documentation in place.
If you have any additional questions, please contact us as [email protected].
Sobi ISS portal support
| ISS portal support document |