Trial reportAllergy2015
Phase II study results of a replacement therapy for hereditary angioedema with subcutaneous C1-inhibitor concentrate.
Trial report in Allergy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01576523 (An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjects With Hereditary Angioedema), which is not on this map. Cited by 33 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjects With Hereditary Angioedema
Who cites it
33 citing papers in PubMed, 3 syntheses or guidelines pooled it, 75 citations in OpenAlex.
- Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema.Journal of comparative effectiveness research · 2025Pooled it
- Hereditary Angioedema Attacks in Patients Receiving Long-Term Prophylaxis: A Systematic Review.Clinical reviews in allergy & immunology · 2024Pooled it
- Interventions for the long-term prevention of hereditary angioedema attacks.The Cochrane database of systematic reviews · 2022Pooled it
- Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema.Pediatric allergy, immunology, and pulmonology · 2020Trial
- Phase II study results of a replacement therapy for hereditary angioedema with subcutaneous C1-inhibitor concentrate.Allergy · 2015Trial
- Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S.The journal of allergy and clinical immunology. Global · 2026Article
- Treatment of Hereditary Angioedema With Plasma-Derived C1 Inhibitor: A Review.Clinical and translational allergy · 2025Review
- Real-world evidence of the effectiveness and utilization of subcutaneous C1INH long-term prophylaxis in patients with HAE in Spain and Germany.Frontiers in immunology · 2025Article
- [Hereditary angioedema due to C1-inhibitor deficiency, a national disease management programme].Wiener klinische Wochenschrift · 2023Article
- A Comprehensive Management Approach in Pediatric and Adolescent Patients With Hereditary Angioedema.Clinical pediatrics · 2023Review
- A Review of Randomized Controlled Trials of Hereditary Angioedema Long-Term Prophylaxis with C1 Inhibitor Replacement Therapy: Alleviation of Disease Symptoms Is Achievable.Journal of asthma and allergy · 2023Review
- The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.The World Allergy Organization journal · 2022Article
- Real-Life Experience With Subcutaneous Plasma-Derived C1-Inhibitor for Long-Term Prophylaxis in Patients With Hereditary Angioedema: A Case Series.Frontiers in allergy · 2022Article
- Treatment of hereditary angioedema-single or multiple pathways to the rescue.Frontiers in allergy · 2022Review
- Clinical profile and treatment outcomes in patients with hereditary angioedema with normal C1 esterase inhibitor.The World Allergy Organization journal · 2022Article
- Reviewing clinical considerations and guideline recommendations of C1 inhibitor prophylaxis for hereditary angioedema.Clinical and translational allergy · 2022Review
- Racial and Ethnic Disparities in the Research and Care of Hereditary Angioedema Patients in the United States.The journal of allergy and clinical immunology. In practice · 2021Article
- Role of complement and potential of complement inhibitors in myasthenia gravis and neuromyelitis optica spectrum disorders: a brief review.Journal of neurology · 2021Review
- Assessing the cost and quality-of-life impact of on-demand-only medications for adults with hereditary angioedema.Allergy and asthma proceedings · 2021Article
- Safety and Outcomes Associated with the Pharmacological Inhibition of the Kinin-Kallikrein System in Severe COVID-19.Viruses · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 11 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHereditary angioedema (HAE) due to C1 inhibitor deficiency manifests as recurrent swelling attacks that can be disabling and sometimes fatal. Long-term prophylaxis with twice-weekly intravenous injections of plasma-derived C1-inhibitor (pdC1-INH) has been established as an effective treatment. Subcutaneous (SC) administration of pdC1-INH has not been studied in patients with HAE.
methodsThis open-label, dose-ranging, crossover study (COMPACT Phase II) was conducted in 18 patients with type I or II HAE who received two of twice-weekly 1500, 3000, or 6000 IU SC doses of highly concentrated volume-reduced CSL830 for 4 weeks each. The mean trough plasma levels of C1-INH functional activity, C1-INH and C4 antigen levels during Week 4, and overall safety and tolerability were evaluated. The primary outcome was model-derived steady-state trough C1-INH functional activity.
resultsAfter SC CSL830 administration, a dose-dependent increase in trough functional C1-INH activity was observed. C1-INH and C4 levels both increased. The two highest dose groups (3000 and 6000 IU) achieved constant C1-INH activity levels above 40% values, a threshold that was assumed to provide clinical protection against angioedema attacks. Compared with intravenous injection, pdC1-INH SC injection with CSL830 showed a lower peak-to-trough ratio and more consistent exposures. All doses were well tolerated. Mild-to-moderate local site reactions were noted with pain and swelling being the most common adverse event.
conclusionsSubcutaneous volume-reduced CSL830 was well tolerated and led to a dose-dependent increase in physiologically relevant functional C1-INH plasma levels. A clinical outcome study of SC CSL830 in patients with HAE warrants further investigation.
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