Hereditary AngioedemaANS-HAENCT06408805
Autonomic Nervous System Profile in Hereditary Angioedema
An observational study of Hereditary Angioedema, run by Istituti Clinici Scientifici Maugeri SpA at 1 site in Italy.
Trial summary
The goal of this study is to compare the autonomic nervous system control of patients with hereditary angioedema to healthy individuals. The main questions it aims to answer are: Are there differences in the autonomic nervous system control between patients with hereditary angioedema and healthy individuals during… Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Hereditary Angioedema
- How many people
- 60 participants
- Where
- 1 siteItaly
- Study dates
- 12 Jul 2023to 18 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: The group of patients with hereditary angioedema were recruited during a out-patient visit in a tertiary care center. The group of healthy individuals were volunteers recruited from community.
Who may be able to join
- Age between 18 and 65 years
- Diagnosis of hereditary angioedema due to C1 inhibitor deficiency (for the group of patients only)
Who cannot join
- Any type of chronic diseases requiring chronic treatment.
- Active acute disease
- Sars-Cov2 infection in the previous three months
- Acute attacks experiences within the previous week and within 72 hours after the enrollment
- Patients in prophylaxis treated off-label.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hereditary Angioedema Due to C1 Inhibitor Deficiency: Evaluation of the Cardiac Autonomic Nervous System Profile in Patients With and Without Prophylactic Treatment
The goal of this study is to compare the autonomic nervous system control of patients with hereditary angioedema to healthy individuals.
The main questions it aims to answer are:
Are there differences in the autonomic nervous system control between patients with hereditary angioedema and healthy individuals during short-term resting period and during orthostatic challenges?
Are there differences in the autonomic nervous system control recorded over long-term periods (i.e. 24 hours)?
Read the detailed description
Angioedema (AE) without wheals is a localized self-limiting edema associated with different mechanisms. The best-known form is hereditary angioedema (HAE) due to C1 inhibitor deficiency (C1-INH), a rare disease with a prevalence of 1:65000 in Italy. Symptoms include swelling of the extremities, genitals, bowel mucosa, face, and upper airway including the larynx. Laryngeal attacks, if not treated, can lead to death. AE attacks are unpredictable and occur episodically upon release of the main mediator of the attack, namely bradykinin, resulting from hyperactivation of the contact system lacking its main control protein C1-INH.
The overall result is an impairment of the endothelial function associated with increased vascular permeability. The release of bradykinin occurs locally, in an unpredictable way, at times facilitated by trauma and different triggers such as stress.
In C1-INH-HAE patients, the autonomic nervous system (ANS) has a role in the regulation of vascular permeability, for example via the baroreflex mechanism. It is also known that sympathetic nervous system inhibition by the α2 agonist clonidine reduces microvascular permeability in endotoxemic animals, suggesting that antagonizing the sympathetic nervous system might prove beneficial in stabilizing capillary leakage during inflammation. Similarly, the vagus nerve has a protective role in models of inflammation such as ischemia-reperfusion injury. The parasympathetic tone, acting on the B2-receptors in the nucleus ambiguous, can also be modulated by bradykinin. The ANS could be investigated using the tilt test and Holter ECG monitoring in current clinical practice when its impairment is suspected. Power spectral analysis of heart rate variability (HRV), i.e. the analysis of the spontaneous fluctuations of the heart period, is exploited to analyze data collected during the tilt test and Holter-ECG, deriving indices of the autonomic control.
In C1-INH-HAE patients the cardiac ANS was investigated during a remission period, during the AE attack and its prodromal phase.
It has been demonstrated that HRV analysis, extended to multiday ECG recordings, may furnish an early marker of an angioedema attack.
In this perspective, the identification of markers, like HRV parameters, could play a crucial role in helping patients and physicians to characterize the ANS control in each patient and individualize treatment based on the ANS balance.
This study aims to compare the cardiovascular ANS control in healthy subjects and HAE patients by head-up tilt test monitoring and evaluate the differences in long-term ANS between healthy controls and HAE patients by Holter ECG monitoring.
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary angioedema, Heart Rate Variability, Autonomic Nervous System, Rare disease
- Medical subject headings
- Angioedemas, Hereditary, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with hereditary angioedema | Patients with documented diagnosis of hereditary angioedema due to C1 inhibitor deficiency with or without prophylactic treatment and aged between 18 and 65 years. | |
| Healthy individuals | Healthy individuals with an age between 18 and 65 and matched with patients with hereditary angioedema by age and gender. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short-term heart rate variability
Changes in heart rate variability indices analyzed during tilt test
Measured: Baseline
Secondary outcomes (2)
Long-term heart rate variability
Changes in heart rate variability indices analyzed during holter ECG recordings
Measured: Baseline
Concentration of catecholamines
Changes in the concentration of catecholamines, quantified in the blood plasma
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2024
- Study start12 Jul 2023
- Primary completion18 Mar 2024
- Study completion18 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
- More trials run by Istituti Clinici Scientifici Maugeri SpAThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Istituti Clinici Scientifici Maugeri SpA
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06408805
- Sponsor's protocol ID
- 2746CE
- Acronym
- ANS-HAE
- Status last verified
- May 2024
- First submitted
- 7 May 2024
- Last updated
- 10 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
De Maria B, Ranucci L, Gino C, Zulueta A, Parati M, Cesoni Marcelli A, Zingale LC, Sideri R, Dalla Vecchia LA, Perego F. Cardiac and vascular autonomic control in patients with hereditary angioedema. Front Physiol. 2025 Dec 16;16:1690915. doi: 10.3389/fphys.2025.1690915. eCollection 2025.
PubMed 41476578
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hereditary Angioedema Due to C1 Inhibitor Deficiency: Evaluation of the Cardiac Autonomic Nervous System Profile in Patients With and Without Prophylactic Treatment
- ClinicalTrials.gov ID
- NCT06408805
- Acronym / sponsor ID
- ANS-HAE / 2746CE
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary angioedema, Heart Rate Variability, Autonomic Nervous System, Rare disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Istituti Clinici Scientifici Maugeri SpAUniversity, hospital or other
- Study start
- 12 Jul 2023 (actual)
- Primary completion
- 18 Mar 2024 (actual)
- Study completion
- 18 Mar 2024 (actual)
- First posted
- 10 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this study is to compare the autonomic nervous system control of patients with hereditary angioedema to healthy individuals.
The main questions it aims to answer are:
Are there differences in the autonomic nervous system control between patients with hereditary angioedema and healthy individuals during short-term resting period and during orthostatic challenges?
Are there differences in the autonomic nervous system control recorded over long-term periods (i.e. 24 hours)?
Detailed description
Angioedema (AE) without wheals is a localized self-limiting edema associated with different mechanisms. The best-known form is hereditary angioedema (HAE) due to C1 inhibitor deficiency (C1-INH), a rare disease with a prevalence of 1:65000 in Italy. Symptoms include swelling of the extremities, genitals, bowel mucosa, face, and upper airway including the larynx. Laryngeal attacks, if not treated, can lead to death. AE attacks are unpredictable and occur episodically upon release of the main mediator of the attack, namely bradykinin, resulting from hyperactivation of the contact system lacking its main control protein C1-INH.
The overall result is an impairment of the endothelial function associated with increased vascular permeability. The release of bradykinin occurs locally, in an unpredictable way, at times facilitated by trauma and different triggers such as stress.
In C1-INH-HAE patients, the autonomic nervous system (ANS) has a role in the regulation of vascular permeability, for example via the baroreflex mechanism. It is also known that sympathetic nervous system inhibition by the α2 agonist clonidine reduces microvascular permeability in endotoxemic animals, suggesting that antagonizing the sympathetic nervous system might prove beneficial in stabilizing capillary leakage during inflammation. Similarly, the vagus nerve has a protective role in models of inflammation such as ischemia-reperfusion injury. The parasympathetic tone, acting on the B2-receptors in the nucleus ambiguous, can also be modulated by bradykinin. The ANS could be investigated using the tilt test and Holter ECG monitoring in current clinical practice when its impairment is suspected. Power spectral analysis of heart rate variability (HRV), i.e. the analysis of the spontaneous fluctuations of the heart period, is exploited to analyze data collected during the tilt test and Holter-ECG, deriving indices of the autonomic control.
In C1-INH-HAE patients the cardiac ANS was investigated during a remission period, during the AE attack and its prodromal phase.
It has been demonstrated that HRV analysis, extended to multiday ECG recordings, may furnish an early marker of an angioedema attack.
In this perspective, the identification of markers, like HRV parameters, could play a crucial role in helping patients and physicians to characterize the ANS control in each patient and individualize treatment based on the ANS balance.
This study aims to compare the cardiovascular ANS control in healthy subjects and HAE patients by head-up tilt test monitoring and evaluate the differences in long-term ANS between healthy controls and HAE patients by Holter ECG monitoring.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with hereditary angioedema | – | –Patients with documented diagnosis of hereditary angioedema due to C1 inhibitor deficiency with or without prophylactic treatment and aged between 18 and 65 years. |
| Healthy individuals | – | –Healthy individuals with an age between 18 and 65 and matched with patients with hereditary angioedema by age and gender. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- The group of patients with hereditary angioedema were recruited during a out-patient visit in a tertiary care center. The group of healthy individuals were volunteers recruited from community.
Key inclusion criteria (2 listed)
- Age between 18 and 65 years
- Diagnosis of hereditary angioedema due to C1 inhibitor deficiency (for the group of patients only)
Key exclusion criteria (5 listed)
- Any type of chronic diseases requiring chronic treatment.
- Active acute disease
- Sars-Cov2 infection in the previous three months
- Acute attacks experiences within the previous week and within 72 hours after the enrollment
- Patients in prophylaxis treated off-label.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Short-term heart rate variability
Time frame: Baseline
Changes in heart rate variability indices analyzed during tilt test
2 secondary outcomes
Long-term heart rate variability
Time frame: Baseline
Concentration of catecholamines
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2024.
- Derived from this study: PubMed 41476578De Maria B, Ranucci L, Gino C, Zulueta A, Parati M, Cesoni Marcelli A, Zingale LC, Sideri R, Dalla Vecchia LA, Perego F. Cardiac and vascular autonomic control in patients with hereditary angioedema. Front Physiol. 2025 Dec 16;16:1690915. doi: 10.3389/fphys.2025.1690915. eCollection 2025.
Related trials for Hereditary Angioedema
RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema, run by Istituti Clinici Scientifici Maugeri SpA at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Short-term heart rate variability (time frame: Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06408805, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2024.