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For Patients
Our Promise to Patients: You Come First
Welcome home
You've likely had a long journey, but now you're among friends. We understand serious and rare diseases because we've specialized in them for decades and we're here to help. By working closely with people like Emily and her family (pictured above), we're able to identify and pursue new innovations that address unmet medical needs. Meanwhile, we continue to enhance our current treatments for rare diseases, including immunodeficiency and autoimmune diseases, hereditary and acquired bleeding disorders, hereditary angioedema, and Alpha 1 antitrypsin deficiency.
We are driven by our promise to save lives and improve the quality of life for our patients and their families, because living with a rare disease impacts more than just one person. Watch this video from our Chief Medical Officer to hear more about our commitment to you. We offer support programs and activities and we partner with patient advocacy organizations to improve and expand educational and outreach efforts. We also work with government authorities to ensure patients have access to the medicines they need to live life to the fullest.
How can we help you?
Immunodeficiency & Autoimmune Diseases
We offer one of the most comprehensive portfolios of high-quality, technically advanced immunoglobulin (Ig) therapies and we are the leading Ig manufacturer in the world. Treatments are available for the following conditions:
Within the United States:
- Primary Immunodeficiency
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Idiopathic Thrombocytopenic Purpura (ITP)
Outside the United States:
- Primary Immunodeficiency
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Idiopathic Thrombocytopenic Purpura (ITP)
- Secondary Immunodeficiency
- Kawasaki Disease (KD)
- Guillain-Barré Syndrome (GBS)
Hereditary Bleeding Disorders
We are a world leader in innovative medicines for the treatment of bleeding disorders. We offer the largest portfolio for the treatment of hereditary bleeding disorders with more than a dozen plasma-derived and recombinant coagulation factor concentrates. Treatments are available for the following conditions:
Within the United States:
- Hemophilia A
- Hemophilia B
- von Willebrand Disease
- Factor XIII Deficiency
- Fibrinogen Deficiency
Outside the United States:
- Hemophilia A
- Hemophilia B
- von Willebrand Disease
- Factor XIII Deficiency
- Fibrinogen Deficiency
- Factor X Deficiency
Hereditary Angioedema (HAE)
We introduced the world's first subcutaneous preventative treatment for attacks of Hereditary Angioedema (HAE), while still offering our intravenous C1-esterase inhibitor therapy for on-demand treatment.
Alpha 1 Antitrypsin Deficiency
Our Alpha 1 proteinase inhibitor treats Alpha 1 Antitrypsin Deficiency (AATD) in patients with Alpha 1-related emphysema.
" Get involved. There are tips and tricks other parents learn over the years in raising children with rare disease. Getting that first-hand advice is helpful. "
- Madonna McGuire-Smith, mother of children with hemophilia
Find a Clinical Trial
A clinical trial is a research study that is done to find out if medical treatments can improve people’s health. A medical treatment can be a drug, medical device, medical procedure, or a change in a person’s behavior such as diet or exercise. People who take part in clinical trials are volunteers. They are also called “participants."
| Therapeutic Area | Condition | Study Number |
Description | Status (Click on link to learn more) |
| Haematology and Thrombosis |
Hemophilia A | CSL627_3001 | An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A | Active, Not Recruiting |
| Hemophilia B | CSL654_3003 | A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B | Recruiting | |
| Hemophilia A & B | CSL689_2001 | Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors | Cancelled | |
| Congenital Fibrinogen Deficiency | BI3023_4003 | An Observational Cohort Study of the Safety and Efficacy of Fibrinogen Concentrate, Human (FCH) in Subjects With Congenital Fibrinogen Deficiency | Completed | |
| Von Willebrand Disease | CSLCT-BIO-12-83 | Study of Voncento® in Subjects With Von Willebrand Disease | Completed | |
| Reversal of acquired coagulation factor deficiency | BE1116_4001 | An Observational Study to Investigate the Risk of Thromboembolic Events in Patients Receiving Kcentra® or Plasma to Reverse Vitamin K Antagonist (VKA) Therapy in the Setting of Acute Major Bleeding | Recruiting | |
| Sickle Cell | CSL200_1001 | CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease | Recruiting | |
| Cardiovascular and Metabolic | Acute Coronary Syndrome | CSL112_3001 | Study to investigate CSL112 in Subjects With Acute Coronary Syndrome (AEGIS-II) | Recruiting |
| Coronary Heart Disease | CSL112_1001 | Assessing the pharmacokinetics, safety, and tolerability of CSL112 in healthy Japanese and Caucasian adults | Completed | |
| Immunology and Neurology | Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | IgPro20_3004 | Extension Study of Maintenance Treatment with Subcutaneous Immunoglobulin (IgPro20) for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | Results |
| Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | IgPro10_5004 | Evaluating the Effectiveness of Telemonitoring System in the Management of Patients With CIDP (HELIPAD 1) | Active, Not Recruiting | |
| Dermatomyositis (DM) | IgPro20_3007 | A study to evaluate the efficacy, safety, and pharmacokinetics of IgPro20 in adults with Dermatomyositis (DM) | Recruiting | |
| Pediatric CIDP | IgPro10_4002 | Randomized Study of Single vs. Multiple Privigen Dose Regimens in Pediatric CIDP | Recruiting | |
| Hereditary Angioedema (HAE) | CSL312_2001 | A study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE) | Recruiting | |
| Hidradenitis Suppurativa and Palmoplantar Pustulosis | CSL324_1002 | A study of the safety and pharmacokinetics of repeat doses of CSL324 in subjects with hidradenitis suppurativa and palmoplantar pustulosis | Recruiting | |
| Primary Immune Deficiency (PID) | IgPro20_4004 | Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra®) in Subjects With Primary Immunodeficiency (PID) | Completed | |
| Primary Immune Deficiency (PID) | IgPro20_4005 | Study of Immune Deficiency Patients treated with Subcutaneous Immunoglobulin (IgPro20, Hizentra®) on Weekly and Biweekly Schedules | Results | |
| Primary Immune Deficiency (PID) | IgPro20_1001 | Comparison of 2 Infusion Devices With Respect to Pharmacokinetics, Safety, and Tolerability of Hizentra: An Investigational Wearable Infusor and the Crono S-PID-50 Infusion Pump | Completed | |
| Systemic Sclerosis | IgPro10_2001 | Efficacy and safety study of IgPro10 in adults with Systemic Sclerosis | Not Yet Recruiting | |
| Healthy Subjects | CSL730_1001 | Assessing the Safety and Tolerability of CSL730 in Healthy Caucasian and Japanese Adults | Active, Not Recruiting | |
| Healthy Subjects | CSL324_1001 | Dose escalation, placebo-controlled phase 1 study to assess the safety and tolerability of CSL324 in healthy adults | Completed | |
| Respiratory | Mild Asthma | CSL311_1001 | A Clinical Study to test the safety, exposure, and markers of Efficacy of CSL311 in patients with mild asthma. | Not Yet Recruiting |
| Transplant | Antibody-mediated kidney transplant rejection | CSL842_3001 | Efficacy and Safety of Human Plasma-derived C1-esterase Inhibitor as add-on to Standard of Care for the Treatment of Refractory Antibody Mediated Rejection (AMR) in Adult Renal Transplant Recipients | Recruiting |
| Acute GVHD (aGVHD) | CSL964_2001 | The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft‑Versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant (MODULAATE) | Recruiting | |
| Graft Versus Host Disease (GVHD) | CSL964_5001 | A study on the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD | Not Yet Recruiting |
Participate in a Clinical Trial
Discover what is involved in participating in one of our clinical trials and how you can enroll.
Learn More