Hereditary angioedema with C1Inh deficiency
MONDO:0033946Forms of hereditary angioedema that occur due to mutations in the gene for complement C1 inhibitor protein. Type I hereditary angioedema is associated with reduced serum levels of complement C1 inhibitor protein. Type II hereditary angioedema is associated with the production of a non-functional complement C1 inhibitor protein.
Also known as: angioedema, hereditary, 1 and 2, angioedema, hereditary, type 1/2, C1 esterase inhibitor, deficiency of, HAE1, angioedema, hereditary, type 1, angioedema, hereditary, type 2, angioedema, hereditary, type I, angioneurotic edema, hereditary
32 clinical trials for this condition and its sub-types.
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Broader categories
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Real-World data to show if HAE drug works in kids
Disease control CompletedThis study tracks 40 children aged 2 to 12 with hereditary angioedema (HAE) who are taking lanadelumab to prevent sudden, painful swelling attacks. Researchers will look at medical records to see how long kids stay attack-free and how the drug performs in daily life. No new treat…
Sponsor: Takeda • Aim: Disease control
Last updated Jul 16, 2026 00:00 UTC
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Teens with rare swelling disorder get new drug tested
Disease control CompletedThis study looked at how a new oral medication (sebetralstat) works in the bodies of 11 teenagers aged 12 to 17 with hereditary angioedema (HAE) types I or II. The goal was to measure drug levels and timing in the blood. All participants were already in a larger HAE trial. The st…
Phase: PHASE3 • Sponsor: KalVista Pharmaceuticals, Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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New pill shows promise for controlling rare swelling disorder
Disease control CompletedThis study looked at the long-term safety of an oral medication called KVD900 for treating swelling attacks in people with hereditary angioedema (HAE) types I or II. The trial enrolled 145 adolescents and adults who had already completed a previous study. Participants took KVD900…
Phase: PHASE3 • Sponsor: KalVista Pharmaceuticals, Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New shot could stop painful swelling attacks in kids
Disease control CompletedThis study tested a drug called Garadacimab in 22 children aged 2 to 11 with hereditary angioedema, a condition that causes sudden, painful swelling. The drug is given as a shot under the skin to prevent attacks. The study looked at safety and how well it works. It has already be…
Phase: PHASE3 • Sponsor: CSL Behring • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New injection shows promise for long-term prevention of rare swelling disorder
Disease control CompletedThis study tested the long-term safety and effectiveness of garadacimab, a monthly injection, for preventing attacks of hereditary angioedema (HAE) — a rare condition causing severe swelling. 171 people aged 12 and older who had frequent HAE attacks took part. The goal was to see…
Phase: PHASE3 • Sponsor: CSL Behring • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New drug navenibart aims to tame hereditary swelling attacks
Disease control CompletedThis early trial tested a new drug called navenibart in 29 people with hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. Participants received one or two doses of the drug to check its safety and how well it might reduce attacks. The study is …
Phase: PHASE1, PHASE2 • Sponsor: Astria Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC
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Real-world study shows lanadelumab keeps HAE attacks at bay for many patients
Disease control CompletedThis study followed 48 people with hereditary angioedema (HAE) in Poland who were taking lanadelumab as part of a national drug program. Researchers tracked how many participants remained attack-free over 24 months, how many attacks still needed rescue treatment, and the severity…
Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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New pill could stop swelling attacks in hereditary angioedema
Disease control CompletedThis study tested an oral medication called deucrictibant for quickly treating attacks of hereditary angioedema (HAE), a condition causing sudden, painful swelling. The trial involved 134 adolescents and adults with HAE types 1, 2, or 3. Participants took either the drug or a pla…
Phase: PHASE3 • Sponsor: Pharvaris Netherlands B.V. • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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New daily pill could stop painful swelling attacks in rare disease
Disease control CompletedThis study tested an oral drug called BCX4161 to prevent attacks of hereditary angioedema (HAE), a condition that causes sudden, painful swelling. Over 12 weeks, 110 adults with HAE took either a low dose, high dose, or placebo pill. Researchers tracked how many swelling attacks …
Phase: PHASE2, PHASE3 • Sponsor: BioCryst Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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New hope for kids with rare swelling disorder: sebetralstat shows promise
Symptom relief CompletedThis study tested a drug called sebetralstat in 36 children aged 2 to 11 with hereditary angioedema (HAE) types I or II, a condition that causes sudden, painful swelling. The goal was to see if the drug is safe and how well it works to relieve attacks. The trial was open-label, m…
Phase: PHASE3 • Sponsor: KalVista Pharmaceuticals, Ltd. • Aim: Symptom relief
Last updated Aug 07, 2026 00:00 UTC
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New pill could stop painful swelling attacks in rare disease
Symptom relief CompletedThis study tested an oral medication called deucrictibant for quickly relieving swelling and pain attacks in people with hereditary angioedema (HAE) types I and II. 74 adults received either one of three doses of the drug or a placebo during an attack. The main goal was to see if…
Phase: PHASE2 • Sponsor: Pharvaris Netherlands B.V. • Aim: Symptom relief
Last updated Jun 27, 2026 08:04 UTC
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HAE patients and caregivers speak out: the hidden costs of On-Demand treatment
Knowledge-focused CompletedThis completed French survey of 300 hereditary angioedema (HAE) patients and their caregivers aimed to understand the challenges of using on-demand treatments for HAE attacks. Researchers collected data on attack frequency, severity, treatment delays, hospitalizations, and the fi…
Sponsor: University Hospital, Grenoble • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC
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Scientists probe gene activity in rare swelling disorder
Knowledge-focused CompletedThis study looked at gene activity in immune cells from 30 people with hereditary angioedema and healthy volunteers. The goal was to see if certain receptors on monocytes are linked to inflammation and blood vessel problems. No treatment was given; the study was purely observatio…
Phase: NA • Sponsor: University Hospital, Rouen • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:02 UTC
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Real-World data on HAE attack treatment collected in completed study
Knowledge-focused CompletedThis study followed 88 people with hereditary angioedema (HAE) types I or II who already use icatibant for sudden attacks. Participants filled out diaries for 48 hours after each attack to report symptom changes, severity, and anxiety levels. The goal was to gather real-world inf…
Sponsor: KalVista Pharmaceuticals, Ltd. • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:10 UTC