CSL Behring

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.Show more

This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.Show more

This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice. Each participant will be followed for 48 months after index date (date of the first administration of garadacimab). Patient data will be collected from the HAE eDiary, patient medical records (MRs) and/or during a routine clinical visit and will be entered into the electronic case report form (eCRF) via an electronic data capture (EDC) system. Data pertaining to HAE attacks, prior HAE treatments, retrospective focused safety data collection, and healthcare resource utilization (HCRU) over a look-back period of 12 months prior to the enrollment will be extracted from the MR, and patients will also record retrospective HAE attack related data over a look-back period of 3 months prior to enrollment in the HAE eDiary. The primary aim of this study is to investigate the real-world effectiveness of garadacimab as measured by HAE attack rate before and after garadacimab initiation in patients with HAE over 24 months of follow-up. The study will aim to complement the data available from the clinical development program on the efficacy, safety, and health-related quality-of-life (HRQoL) in patients with HAE taking garadacimab.Show more

A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed to IVIG treatment

Although there is evidence in the literature that gammaglobulin replacement therapy can lead to a reduction in the prevalence of pulmonary infection and improved lung function, there is no published study to guide immunologists regarding the use of spirometry in titrating IG therapy to assist in the management of immunodeficiency patients with regards to gammaglobulin replacement therapy. The investigators propose to study the use of spirometry to identify patients that could potentially benefit from an increase in IGRT. The investigators will identify 22 common variable immune deficiency (CVID) study subjects on stable IGRT replacement therapy equivalent to 0.40 to 0.60 gm/kg per 4 weeks who have evidence of mild to moderate obstruction as assessed by an FEF25-75% between 50% and 80% of predicted. Patients who are on Hizentra will be preferentially recruited. Of these 22, 11 will be identified at random and treated for 6 months at their current dose (control population). The remaining 11 study subjects (treatment group) will have their level of IGRT increased by the equivalent of 0.05 gm/kg in dose per 4 weeks, adjusted for bioavailability as per manufacturer's instructions. On average, rounded up to the nearest gram, this will typically increase their dose of Hizentra by 2 gm per week.Show more

CSL Behring is a global leader in plasma-derived and recombinant therapies for rare diseases including bleeding disorders, immune deficiencies, and hereditary angioedema. Key products include Hemgenix (etranacogene dezaparvovec), the first gene therapy for hemophilia B, Idelvion (Factor IX) for hemophilia B, Haegarda (C1 esterase inhibitor) for hereditary angioedema, and Hizentra (subcutaneous immunoglobulin) for primary immunodeficiency and CIDP. CSL Behring is part of CSL Limited, which also acquired Vifor Pharma in 2022 to expand into nephrology and iron deficiency.

CSL Behring has active programs across 16 rare diseases: Alpha-1 Antitrypsin Deficiency, Ataxia-Telangiectasia, Chronic Inflammatory Demyelinating Polyneuropathy, Common Variable Immunodeficiency, Cystic Fibrosis, Dermatomyositis, Eosinophilic Esophagitis, Hemophilia A, Hemophilia B, Hereditary Angioedema, Idiopathic Pulmonary Fibrosis, Immune Thrombocytopenia, Myasthenia Gravis, Sickle Cell Disease, Systemic Sclerosis, Von Willebrand Disease. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

CSL Behring is a diversified pharmaceutical company headquartered in Melbourne, Australia, founded in 1916. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 16 active rare disease programs including Alpha-1 Antitrypsin Deficiency, Ataxia-Telangiectasia, Chronic Inflammatory Demyelinating Polyneuropathy. Many of its rare disease treatments hold orphan drug designations from the FDA.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by CSL Behring, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

CSL Behring was founded in 1916 and is headquartered in Melbourne, Australia. The company has been active for 110 years. It trades publicly under the ticker symbol CSL.