Investigator-Initiated Studies

We seek to advance science and improve patient care with CSL Behring products in the field of immunology. Through the provision of total or partial funding, CSL Behring supports high-quality Immunology research that is initiated, designed, implemented, and sponsored by external investigators and involves our immunoglobulin products. Support is provided based on the scientific merit of the proposal as well as alignment with the following specific areas of interest:

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

Secondary Immune Deficiency (SID)
Proposals that increase the knowledge of the efficacy and safety of immunoglobulins in SID indications, e.g. hematologic malignancies, autoimmune diseases, and specifically the combination of immunoglobulins with products/procedures that cause drug-induced SID in conditions such as multiple myeloma (MM), chronic lymphocytic leukemia (CLL) and solid organ transplant/hematopoietic stem-cell transplantation (HSCT).

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, quality-of-life in CIDP patients, health economics in CIDP and specific sub-populations, such as elderly patients.

We seek to advance science and improve patient care with CSL Behring 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] and our gene therapy etranacogene dezaparvovec-drlb. 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
• Basic, translational, or clinical research to gain more understanding on the response to gene therapy, including the immune and the liver-related factors, as well as the role of neutralizing antibodies
• Studies that assess gene therapy in real-world settings, including factors such as the impact of stable factor expression levels, innovative efficacy measurements using biomarkers, joint health, and patient-reported outcomes
• Clinical studies that increase knowledge of immune tolerance induction with our recombinant Coagulation Factor VIII product

Von Willebrand Disease

• Role of von Willebrand Factor (vWF) in reducing inhibitor development
• 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 pd-VWF/FVIII vs. 1:1 concentrates
• Generate data in VWD (including burden of disease), increase awareness of VWD & show benefit of treatment
• Generate data in VWD in orthopedic status and joint health

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 / Congenital Fibrinogen Deficiency

• Clinical studies of PCCs as hemostatic agents in perioperative bleeding and trauma-induced coagulopathy
• Basic science research with PCCs in trauma-induced coagulopathy
• Clinical Studies of Fibrinogen as a hemostatic agent
• Basic science studies of Fibrinogen as a hemostatic agent
• The role of FXIII in the pathophysiology of acute bleeding

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

Alpha-1 antitrypsin Deficiency

• Prevalence, epidemiology, natural course of Alpha-1 Antitrypsin deficiency and development of new biomarkers to monitor disease progression
• Burden of disease of Alpha-1 Antitrypsin deficiency
• Pathophysiology of Alpha-1 Antitrypsin deficiency
• Characterization of long-term outcomes during Alpha-1 Antitrypsin substitution treatment versus other treatment approaches
• Characterization of outcome in patients following lung transplant and treated with Alpha-1 Antitrypsin substitution versus other treatment approaches

Hereditary Angioedema (HAE)

• Health Outcome studies on C1 Esterase Inhibitor (C1-INH) including quality of life and adherence
• Health Outcome studies in HAE on treatment patterns
• Clinical studies and experience with novel treatment strategies for C1-INH replacement therapy
• Use of C1-INH in special patient populations (eg; pregnancy, pediatrics)
• Use of C1-INH replacement therapy as short-term prophylaxis
• Unmet needs and outcomes in patients with HAEnC1-INH, including in the context of long-term prophylaxis use
• Prevalence, epidemiology, and natural course of disease in patients with HAEnC1-INH
• Pathophysiology of FXIIa in HAE
• Role of FXIIa inhibition in comorbidities occurring in HAE patients
• Role of FXIIa inihibition in hemostasis, thrombosis, and inflammation in HAE patients
• Data showing the association between treatment response/attack-free status and QoL/productivity improvements
• Outcome studies with garadacimab on quality of life and patient reported outcomes including measures used
• Outcome studies in HAE on treatment patterns with long-term prophylaxis, patient satisfaction and adherence
• Biomarker studies in HAE
• Characterization of full (attack-free)/partial/non responders to long-term prophylaxis: stratification, predictors, and treatment patterns
• Burden of disease in patients with HAE and their caregivers, including those specific to special patient populations

We seek to advance science and improve patient care with CSL Behring products in the field of transplantation. Through total or partial funding, we support external investigators who initiate, design and implement high-quality transplantation research focused on areas of high unmet medical need and involves our Alpha-1 Antitrypsin.

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)

• Real-world evidence of prevention and treatment of acute Graft versus Host Disease (GvHD), including health outcomes data and QoL
• Burden of disease of acute GvHD

Solid Organ Transplant

• Prevalence, epidemiology, and natural course of disease of chronic active Antibody Mediated Rejection (AMR)
• Burden of disease of chronic active AMR
• Basic science research and clinical pilot studies aimed at preventing rejection of solid kidney or lung transplants

A: You can contact your regional Medical Affairs representative by clicking here.

A: You should submit a first draft protocol of the study for CSL Behring to consider for review.

The grant application process consists of two parts:

1. The initial study synopsis/draft protocol submission.
2. Submission of a full protocol.

If you are accepted for a full protocol submission, you will be notified by email and a new Full Protocol request will become available on your profile in the “Required Reporting” section.

A: Full submissions are reviewed on a monthly basis. You will be notified when the CSL Behring Review Committee has arrived at a decision. Decisions are based on medical and scientific value in addition to current resources and research priorities.

A: CSL Behring is considered the Grant Provider for any Investigator-Initiated Study that we fund. The role of Sponsor-Investigator needs to be assumed by either the Investigator or the Investigator Organization.