Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks: An Open-Label Extension Study of the COMPACT Trial

Craig, Timothy J.; Zuraw, Bruce L.; Longhurst, Hilary; Cicardi, Marco; Bork, Konrad; Grattan, Clive; Katelaris, Constance H.; Sussman, Gordon; Keith, Paul K.; Yang, William H.; Hébert, Jacques; Hanzlı́ková, Jana; Staubach‐Renz, Petra; Martinez‐Saguer, Inmaculada; Magerl, Markus; Aygören‐Pürsün, Emel; Farkas, Henriette; Reshef, Avner; Kivity, Shmuel; Neri, Sergio; Crisan, Ioana; Caballero, Teresa; Baeza, M.L.; Hernández, M.D. Jiménez; Li, Henry; Lumry, William R.; Bernstein, Jonathan A.; Hussain, Iftikar; Anderson, J.; Schwartz, Lawrence B.; Jacobs, Joshua; Manning, Michael; Levy, Donald S.; Riedl, Marc A.; Christiansen, Sandra C.; Feuersenger, Henrike; Pragst, Ingo; Mycroft, Sarah; Pawaskar, Dipti; Jacobs, Iris; Investigators, Compact

doi:10.2139/ssrn.3247882

Cited by 1 publication

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“…The OLE of the COMPACT trial was a multicenter, randomized, parallel-arm study and enrolled patients treated in the placebo-controlled COMPACT trial, as well as C1INH (SC)-naive patients. 17 Eligible patients (age ‡6 years with at least 4 attacks over 2 consecutive months before enrollment in the OLE or the placebo-controlled COMPACT trial) were randomly assigned to receive C1INH (SC) 40 or 60 IU/kg twice weekly for 52 weeks. 17 Subjects in the United States could continue therapy for an additional 88 weeks (maximum exposure of 140 weeks) ( Fig.…”

Section: Compact Ole Study Designmentioning

confidence: 99%

“…Dose increases in increments of 20 IU/kg (up to 80 IU/kg) were permitted at the investigator's discretion in case of frequent HAE attacks. 17 The primary objective of the OLE was to assess the longterm safety of C1INH (SC). 17 The secondary efficacy endpoints were the percentage of responders ( ‡50% reduction in attacks compared with the prestudy period) and percentage of subjects with <1 attack per 4-week period.…”

Section: Compact Ole Study Designmentioning

confidence: 99%

“…16 The long-term safety and efficacy of C1INH (SC) were further evaluated in an openlabel extension (OLE) of the COMPACT trial in which subjects were treated for up to 140 weeks (2.7 years). 17 In the OLE study, C1INH (SC) was well tolerated and had a marked and sustained effect, with a median attack rate of 1.0 attack per year in subjects treated with the 60 IU/kg dose. 17 In this analysis, we evaluated the long-term efficacy and safety of C1INH (SC) in 10 pediatric subjects (7 adolescents 12-17 years old and 3 children <12 years old) treated in the OLE of the COMPACT trial, and compared them with adult subjects (>17 years) in the OLE population.…”

Section: Introductionmentioning

confidence: 99%

“… 16 The long-term safety and efficacy of C1INH (SC) were further evaluated in an open-label extension (OLE) of the COMPACT trial in which subjects were treated for up to 140 weeks (2.7 years). 17 In the OLE study, C1INH (SC) was well tolerated and had a marked and sustained effect, with a median attack rate of 1.0 attack per year in subjects treated with the 60 IU/kg dose. 17 …”

Section: Introductionmentioning

confidence: 99%

“… 17 In the OLE study, C1INH (SC) was well tolerated and had a marked and sustained effect, with a median attack rate of 1.0 attack per year in subjects treated with the 60 IU/kg dose. 17 …”

Section: Introductionmentioning

confidence: 99%

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Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema

Levy

Caballero

Hussaın

et al. 2020

Pediatric Allergy, Immunology, and Pulmonology

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Background: Hereditary angioedema (HAE) due to C1 inhibitor (C1INH) deficiency is characterized by recurrent attacks of edema of the skin and mucosal tissues. Symptoms usually present during childhood (mean age at first attack, 10 years). Earlier symptom onset may predict a more severe disease course. Subcutaneous (SC) C1INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. We analyzed the long-term efficacy of C1INH (SC) in subjects £17 years old treated in an open-label extension (OLE) of the pivotal phase III Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1 Inhibitor Replacement Therapy (COMPACT) trial. Methods: Eligible subjects (age ‡6 years, with ‡4 attacks over 2 consecutive months before entry into the OLE or placebo-controlled COMPACT trial) were treated with C1INH (SC) 40 or 60 IU/kg twice weekly for 52-140 weeks. Subgroup analyses by age (£17 vs. >17 years) were performed for key efficacy endpoints. Results: Ten subjects were £17 years old [mean (range) age, 13.3 (8-16) years, 3 subjects <12 years old; exposure range, 51-133 weeks]. All 10 pediatric subjects experienced ‡50% reduction (mean, 93%) in number of attacks versus the prestudy period, with a 97% reduction in the median number of attacks/month (0.11). All subjects had <1 attack/4-week period and 4 had <1 attack/year (1 subject was attack free). No subject discontinued treatment due to a treatment-related adverse event. Conclusions: Data from pediatric subjects treated with C1INH (SC) for up to 2.55 years and adult subjects revealed similar efficacy. C1INH (SC) is effective and well tolerated as long-term prophylaxis in children, adolescents, and adults with HAE.

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Section: Compact Ole Study Designmentioning

confidence: 99%

Section: Compact Ole Study Designmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

“… 17 In the OLE study, C1INH (SC) was well tolerated and had a marked and sustained effect, with a median attack rate of 1.0 attack per year in subjects treated with the 60 IU/kg dose. 17 …”

Section: Introductionmentioning

confidence: 99%

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