Vedec so skúmavkou

Investigator-Initiated Studies

CSL Behring Investigator-Initiated Studies

CSL Behring is committed to saving lives and improving the quality of life for people with rare and serious diseases worldwide. This commitment is reflected in the support for Investigator-Initiated Studies (IIS) that advance medical and scientific knowledge of CSL Behring products and the diseases they have been designed to treat. CSL Behring also supports innovative clinical and basic science studies that address important medical and scientific questions related to our therapeutic areas of focus.

Areas of Focus:

  • Immunology
  • Hematology
  • Respiratory
  • Cardiovascular & Metabolic
  • Transplant

The CSL Behring IIS program is open to physicians, researchers and institutions interested in conducting their own research. We receive many requests for support and carefully evaluate each one with priority given to proposals that align with our interests.

Disclaimer: Any application to participate in CSL Behring’s IIS program, including any information shared in connection with such application, will be used by CSL Behring for the sole purpose of evaluating the application, and will not be shared externally. To the extent that an application contains personal information about an individual, such information will be treated in accordance with CSL’s privacy policy, located at https://www.csl.com/privacy-policy.

Research Priorities and Eligibility

Immunology

We seek to advance science and improve patient care with CSL Behring's products in the field of immunology. Through the provision of total or partial funding, CSL Behring supports high-quality immunoglobulin research that is initiated, designed, implemented, and sponsored by external investigators and involves our immunoglobulin or C1 Esterase inhibitor (C1-INH) products. Applicant proposals may request funding and C1-INH drug supply. Support is provided based on the scientific merit of the proposal as well as alignment with the following specific areas of interest:

Primary Immunodeficiency (PI) Diseases

Proposals that increase the knowledge of the efficacy and safety of immunoglobulins in PI indications, specifically route of administration, subclinical infections in diagnosed PI patients with respiratory tract engagement, quality-of-life, wear-off, manual push, flexible dosing/patient preference, and undiagnosed PI.

Secondary Immunodeficiency (SI) Diseases

Proposals that increase the knowledge of the efficacy and safety of immunoglobulins in SI indications, specifically the combination of immunoglobulins with products that cause drug-induced SI in conditions such as multiple myeloma (MM) and chronic lymphocytic leukemia (CLL).

Peripheral Neuropathy

Proposals that increase the knowledge of efficacy and safety of immunoglobulins, specifically pharmacokinetics in chronic inflammatory demyelinating polyneuropathy (CIDP) including wear-off, dose titration, dose conversion from intravenous immunoglobulins (IVIg) to subcutaneous immunoglobulins (SCIg), loading dose and dosing by body mass index (BMI) or fat percent versus weight, quality-of-life in CIDP patients, health economics in CIDP and specific sub-populations, such as elderly and pediatric patients.

Hereditary Angioedema (HAE)

  • Health Outcome studies on C1 Esterase Inhibitor (C1-INH) including quality of life and adherence
  • Clinical studies and experience with novel treatment strategies for C1-INH Replacement Therapy
  • Use of C1-INH in special patient populations (eg; pregnancy, pediatrics)

Hematology

We seek to advance science and improve patient care with CSL Behring's products in the field of hematology. Through total or partial funding, we support external investigators who initiate, design and implement high-quality hematological research that is focused on our recombinant products, Coagulation Factor IX (Recombinant) Albumin Fusion Protein and Antihemophilic Factor (Recombinant) Single Chain, and our plasma products, Human Plasma Derived von Willebrand Factor (HP-vWF/FVIII) concentrate, Fibrinogen, and Prothrombin complex concentrate [human]. Support is provided based on the scientific merit of the proposal as well as alignment with the following specific areas of interest:

Hemophilia A and B

  • Clinical studies and experience in transitioning to our recombinant coagulation factor products including on-demand treatment and prophylaxis
  • Health outcome studies, including factor consumption, quality-of-life data
  • Health economic outcome studies
  • Clinical studies that focus on long-term joint health and radiographic imaging to assess and monitor joint damage
  • Use in special populations including women
  • Real-world evidence of hemophilia A and B management

Hemophilia A only

  • Role of von Willebrand Factor (vWF) in reducing inhibitor development
  • Clinical studies that increase knowledge of immune tolerance induction with our recombinant Coagulation Factor VIII product

Von Willebrand Disease

  • Clinical studies that generate evidence of efficacy or safety in selected von Willebrand Disease populations specifically: low vWF, type 3, type 2, menorrhagia, pregnancy, or prophylaxis for angiodysplasia, epistaxis and joint bleeds
  • Generate data supporting efficacy & safety, and health economic benefits of high-ratio pdvWF/FVIII vs. 1:1 concentrates
  • Generate data in vWD (including burden of disease), increase awareness of vWD & show benefit of treatment

(Studies with head to head comparisons with other products are excluded)

Oral Anticoagulant-Associated Bleeding

  • Clinical studies and experience with prothrombin complex concentrates (PCCs) for the management of acute major bleeding in patients treated with Vitamin K antagonists (VKAs) or direct oral anticoagulants (DOACs)
  • Clinical studies and experience with PCCs for urgent reversal prior to emergency surgery or procedures in patients treated with VKAs or DOACs

Acquired Bleeding

  • Clinical studies of PCCs as hemostatic agents in perioperative bleeding and trauma-induced coagulopathy
  • Basic science research with PCCs in trauma-induced coagulopathy

Fibrinogen

  • Clinical Studies of Fibrinogen as a hemostatic agent
  • Basic science studies of Fibrinogen as a hemostatic agent

Respiratory

We seek to advance science and improve patient care with CSL Behring's products in respiratory diseases. Through total or partial funding only, we support external investigators who initiate, design and implement high-quality respiratory research that is focused on the disease alpha-1 antitrypsin deficiency to generate data on:

  • Prevalence, epidemiology, natural course of disease and the development of new biomarkers to monitor disease progression
  • Burden of disease
  • Pathophysiology of Alpha-1 Antitrypsin deficiency

Cardiovascular and Metabolic

CSL is committed to developing novel therapies that save and improve the lives of patients with serious and life-threatening diseases. As part of this commitment, we are focused on the leading cause of death worldwide - Cardiovascular and Metabolic diseases. Our goal in Cardiovascular and Metabolic disease is to address areas of high, unmet medical need and we continue to explore opportunities to work with external investigators to better define and address these needs.

Transplant

We seek to advance science and improve patient care with CSL Behring's products in the field of transplantation. Through total or partial funding, we support external investigators who initiate, design, implement high quality transplantation research focused on areas of high, unmet medical need and involves our Alpha-1 Antitrypsin, Clazakizumab (Claza), or C1 Esterase Inhibitor (C1-INH) products. Applicant proposals may request funding only, or funding and Clazakizumab or C1-INH drug supply. Support is provided based on the scientific merit of the proposal as well as alignment with the following specific areas of interest:

Hematopoietic Cell Transplant (HCT)

  • Burden of disease, natural course of disease, and understanding the role of conditioning regimens in incidence/severity of acute GvHD.
  • Use of biomarkers to identify patients at risk for GvHD
  • Use of Alpha-1 Antitrypsin to prevent/ameliorate acute GvHD through mechanistic and early/pilot clinical studies
  • Studies aimed at defining and understanding the immunomodulatory effects of Alpha-1 Antitrypsin

Ischemia / Reperfusion Injury

  • Use of complement inhibitors and Alpha-1 Antitrypsin to prevent/ameliorate ischemia reperfusion injury as a cause of delayed graft function/diminished organ survival through mechanistic, biomarker and clinical pilot studies

Antibody Mediated Rejection (AMR)

  • Prevalence, epidemiology, burden of disease, natural course of disease, and the development of new biomarkers to monitor disease progression
  • Studies aimed at understanding the role of IL-6 within AMR including prevention and treatment of AMR

Tolerance

  • Studies aimed at understanding the role of tolerance in solid organ transplant

Application

Requirements

A complete Investigator-Initiated Study application must include the following:

  • An online application submitted through the portal
  • Signed and dated Curriculum Vitae
  • Detailed study budget
  • Draft protocol

Request Process

  • Review the "Areas of Focus" sections to identify if the grant request aligns with CSL Behring's areas of interest.
  • To start an IIS application click on "Apply Here".
  • Log in to your account. First time requestors will need to create an account.
  • Your request will be reviewed and we will notify you via email of the decision.
  • If your request is approved, you will be contacted regarding the Agreement and disbursement process.

Frequently Asked Questions (FAQs)

Q: Who should I contact for more information regarding Investigator-Initiated Studies?
A: You can reach out to your local Medical Affairs contact by clicking here.

Q: Should I submit a conceptual outline or a full study proposal?
A: You should submit a full proposal of the study for CSL Behring to consider for review. Conceptual or high-level proposals will not be considered until they are fully developed.

Q: How long does the review process take?
A: Full submissions are submitted to the global review process. You will be notified when the CSL Behring reviewers have arrived at a decision. Decisions are based on medical and scientific value in addition to current resources and research priorities.

Q: What is CSL Behring’s role in a funded Investigator-Initiated Study?
A: CSL Behring is considered the Grant Provider for any Investigator-Initiated Study that it funds. Due to certain legal implications, the role of Sponsor needs to be assumed by either the Investigator or the Investigator’s Organization.