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Hereditary angioedema – Trials in Disease

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Ongoing Clinical Trials for Hereditary Angioedema

There are currently 17 clinical trials investigating new treatments for hereditary angioedema, a rare genetic condition causing sudden swelling episodes. These studies are testing various medications including gene-editing therapies, oral preventive treatments, and injectable biologics across multiple European countries.

Clinical trial locations

Long-Term Safety Study of NTLA-2002 for Patients with Hereditary Angioedema, Using Messenger RNA Encoding Cas9 and HU-G012267

This study is designed for individuals who have already received treatment with NTLA-2002, an experimental gene-editing therapy. The trial monitors the long-term safety of this innovative medication by tracking participants over an extended period.

Inclusion criteria: Participants must have completed or discontinued a previous NTLA-2002 treatment study. They need to provide informed consent and commit to attending regular study visits and following the required schedule.

Exclusion criteria: Individuals who have not previously been treated with NTLA-2002 cannot participate. Those outside the specified age range or belonging to vulnerable populations requiring special protection may not be eligible.

Study focus: The primary goal is to evaluate the long-term safety profile of NTLA-2002 by monitoring treatment-related adverse events, serious side effects, and events of special interest. The study also tracks the frequency of swelling attacks, use of emergency medications, and changes in quality of life using validated assessment tools.

Investigational drug: NTLA-2002 is administered through intravenous infusion and contains messenger RNA encoding Cas9. This cutting-edge treatment aims to modify the KLKB1 gene involved in the swelling process characteristic of hereditary angioedema.

Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

This research evaluates deucrictibant, a once-daily oral medication designed to prevent swelling attacks. The 24-week treatment period compares the effectiveness of deucrictibant tablets against placebo.

Inclusion criteria: Participants must be at least 12 years old and weigh a minimum of 40 kg if adolescent. They need a confirmed diagnosis through appropriate laboratory tests and must have experienced at least three attacks within three months before screening. Access to standard acute treatments is required.

Exclusion criteria: Children under 12 years cannot participate. Other exclusions include pregnancy, breastfeeding, severe allergic reactions to similar medications, participation in other trials, uncontrolled medical conditions, and substance abuse problems.

Study focus: The trial tracks the number and severity of swelling attacks during the 24-week period. Participants maintain an electronic diary to record symptoms and attacks, while researchers assess quality of life and treatment satisfaction through questionnaires.

Investigational drug: Deucrictibant is an extended-release tablet taken orally at a dose of 40 mg daily. This medication works as a plasma kallikrein inhibitor to prevent the biochemical cascade that leads to swelling episodes.

Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

This long-term extension study provides continued access to deucrictibant for participants who have previously been enrolled in related trials. The research evaluates the medication’s safety and effectiveness over an extended period.

Inclusion criteria: Participants must be at least 12 years old with a confirmed diagnosis. Those who previously participated in deucrictibant studies may qualify, as well as new participants who have experienced at least one attack in the past three months. Access to acute treatment medications is necessary.

Exclusion criteria: Children under 12 years are excluded. Other exclusions include pregnancy, active liver disease, severe kidney problems, uncontrolled high blood pressure, recent major surgery, cancer or recent cancer treatment, and inability to follow study procedures.

Study focus: The study monitors long-term safety by tracking adverse events, while also assessing attack frequency and severity. Regular health assessments include blood tests, vital signs monitoring, and heart recordings. Participants complete quality of life questionnaires throughout the study.

Investigational drug: Deucrictibant 40 mg extended-release tablets are taken orally once daily. This bradykinin B2 receptor antagonist prevents swelling by blocking the binding of bradykinin, which causes blood vessel dilation and fluid leakage.

Study of Navenibart to prevent attacks in adults and adolescents with Hereditary Angioedema

This six-month study compares navenibart injections against placebo to determine effectiveness in preventing swelling attacks. Participants receive regular subcutaneous injections throughout the treatment period.

Inclusion criteria: Participants must be at least 12 years old with a confirmed diagnosis of Type 1 or Type 2 hereditary angioedema. During an initial observation period, they must experience at least two attacks verified by study doctors. The ability to have attacks confirmed according to study definitions is required.

Exclusion criteria: Exclusions include severe allergic reactions to medications, active liver disease, pregnancy or breastfeeding, recent trial participation, planned major surgery, uncontrolled high blood pressure, blood clotting disorders, severe kidney problems, active cancer, recent substance abuse, and severe mental health conditions.

Study focus: The primary measure is the number of attacks during the six-month period. Researchers track attack severity, need for additional treatment, time between medication and first attack, and quality of life changes.

Investigational drug: Navenibart is administered as a subcutaneous injection for prophylactic treatment. This Phase 3 medication aims to prevent swelling attacks through a mechanism distinct from existing treatments.

Study on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency

This trial tests OCTA-C1-INH, a plasma-derived treatment given through intravenous infusion to relieve symptoms during acute swelling attacks. The study compares this treatment against placebo in a double-blind design.

Inclusion criteria: Participants must be at least 18 years old for the first phase or at least 2 years for the second phase. A confirmed diagnosis of Type I or II hereditary angioedema is required, with at least three moderate or severe attacks in the past three months. Laboratory tests must show C1-INH functional activity below 50 percent and C4 antigen levels below normal range.

Exclusion criteria: Individuals not experiencing an acute attack cannot participate. Those not willing to receive intravenous treatment are excluded, as are certain age groups depending on the study phase.

Study focus: The primary measure is the time to symptom relief at the attack site, assessed every 15 minutes for four hours after injection. Secondary measures include the percentage of participants responding to treatment and changes in symptom severity.

Investigational drug: OCTA-C1-INH is a human plasma-derived C1 esterase inhibitor concentrate given intravenously. It works by replacing the missing or malfunctioning C1 inhibitor protein in the blood to control swelling.

Study on the Effect and Safety of BW-20805 for Adults with Hereditary Angioedema

This study evaluates BW-20805 Injection administered under the skin to prevent swelling attacks. Participants receive regular injections over several months while being monitored for effectiveness and safety.

Inclusion criteria: Participants must be between 18 and 70 years old with a documented diagnosis of Type 1 or Type 2 hereditary angioedema. They need to have experienced at least two attacks within an eight-week period, confirmed by study doctors. Access to acute attack medications is required, and participants must weigh at least 40 kg.

Exclusion criteria: Those without a hereditary angioedema diagnosis cannot participate. Age restrictions exclude those outside the 18-70 range. Other exclusions are not detailed in the available information.

Study focus: The study measures the reduction in attack frequency from baseline through day 169. Monitoring includes tracking side effects and adverse events throughout the treatment and extension periods.

Investigational drug: BW-20805 is administered as a subcutaneous injection. This Phase 2 medication is being studied for its potential to reduce the frequency of swelling attacks by targeting pathways involved in inflammation and vascular permeability.

Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

This long-term safety study provides continued access to berotralstat for participants who have been part of previous trials. The research monitors safety and tolerability over an extended period.

Inclusion criteria: Participants must have been enrolled in previous berotralstat studies and not discontinued due to adverse events or noncompliance. Written informed consent is required, with assent forms for those aged 12 to 17. The study doctor must believe the participant will benefit from continued treatment.

Exclusion criteria: Those without hereditary angioedema cannot participate. Age restrictions apply to ensure appropriate study groups. Individuals not expected to follow study procedures properly are excluded.

Study focus: The study evaluates long-term safety by monitoring treatment-related adverse events, serious events, and significant laboratory abnormalities. Ongoing assessment continues throughout the study period.

Investigational drug: Berotralstat is taken orally as a capsule. This kallikrein inhibitor works by reducing plasma kallikrein activity, thereby decreasing bradykinin production responsible for swelling and pain in hereditary angioedema attacks.

Safety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema

This study evaluates the safety of switching from current preventive treatments to garadacimab in patients aged 12 and older. The three-month treatment period monitors how participants respond to the medication change.

Inclusion criteria: Participants must be at least 12 years old with confirmed hereditary angioedema. They need documented previous response to acute treatments and must have been using lanadelumab, berotralstat, or plasma-derived C1 inhibitor for at least three months at a stable dose before screening.

Exclusion criteria: Children under 12 years cannot participate. Those not currently using approved preventive treatments are excluded, as are pregnant women, individuals with severe allergic reactions to similar medications, those in other trials, patients with uncontrolled medical conditions, and those unable to follow study procedures.

Study focus: The study monitors participant response to garadacimab, tracking side effects and reactions. Blood samples measure medication levels, and researchers check for antibody development against the drug.

Investigational drug: Garadacimab is administered via subcutaneous injection at doses up to 400 mg per day. This monoclonal antibody inhibits Factor XIIa, offering a different mechanism of action from existing prophylactic treatments.

Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

This long-term study evaluates the safety of donidalorsen, an antisense oligonucleotide given as a subcutaneous injection. Participants receive regular injections for up to 53 weeks.

Inclusion criteria: Participants must provide written informed consent and have access to at least one acute treatment medication. They need to have completed a previous study through Week 25 or be on stable prophylaxis treatment with lanadelumab, berotralstat, or C1-esterase inhibitor for at least 12 weeks. Minimum age is 12 years with a documented diagnosis of Type 1 or Type 2 hereditary angioedema.

Exclusion criteria: Those without hereditary angioedema cannot participate. Age restrictions apply to ensure appropriate study groups. Members of vulnerable populations may not be eligible.

Study focus: The study tracks the incidence and severity of adverse events during long-term treatment. Secondary evaluations include attack frequency, percentage of attack-free patients, and quality of life assessments over 53 weeks.

Investigational drug: Donidalorsen is administered as a subcutaneous injection. This antisense oligonucleotide targets specific genetic material to reduce attack frequency by interfering with protein production that contributes to swelling.

Study on NTLA-2002 for Adults with Hereditary Angioedema Using Ziclumeran and Lonvoguran

This study tests NTLA-2002, an advanced therapy involving nucleic acids delivered through intravenous infusion. The trial has multiple phases evaluating safety, dosing, and effectiveness.

Inclusion criteria: Participants must be at least 18 years old with documented hereditary angioedema and at least three attacks in the past three months confirmed by doctors. They need access to acute treatment medications and must meet specific laboratory criteria during screening. Birth control requirements apply to participants who can have children.

Exclusion criteria: Those without hereditary angioedema cannot participate. Other exclusions include unwillingness to follow procedures, other interfering medical conditions, pregnancy, breastfeeding, recent trial participation, medication allergies, interfering medications, and substance abuse history.

Study focus: Phase 1 assesses safety and determines appropriate doses. Phase 2 evaluates effectiveness in reducing attacks over 16 weeks, comparing NTLA-2002 against placebo. Some participants may receive follow-on dosing to further assess safety.

Investigational drug: NTLA-2002 is administered intravenously as a dispersion for infusion. This gene-editing therapy uses CRISPR technology to target and modify the KLKB1 gene, aiming to correct the underlying genetic defect causing swelling attacks.

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

This double-blind study compares NTLA-2002 against placebo to evaluate effectiveness in reducing attack frequency. The advanced therapy product is delivered through intravenous infusion with active substances ziclumeran and lonvoguran.

Inclusion criteria: Participants must be at least 18 years old with clinical history of hereditary angioedema and appropriate laboratory criteria related to C1-INH levels. At least two confirmed attacks during an eight-week period before the study are required. Access to acute treatment medications is necessary, along with meeting specific laboratory test criteria.

Exclusion criteria: Those without hereditary angioedema due to C1 esterase inhibitor deficiency cannot participate. Age restrictions and other specific criteria apply for eligibility.

Study focus: The primary observation period runs from Week 5 through Week 28, measuring attack frequency compared to placebo. Secondary measures include attacks requiring on-demand treatment, attack severity, and quality of life changes.

Investigational drug: NTLA-2002 is administered intravenously. This gene-editing therapy uses CRISPR technology to edit genes and correct the underlying genetic defect, potentially providing a long-term solution by addressing the root cause of the condition.

Summary

The current landscape of clinical trials for hereditary angioedema demonstrates significant research activity across Europe, with 17 ongoing studies investigating various treatment approaches. Germany leads in trial participation with representation in 11 studies, followed by Poland and Italy with 10 and 9 studies respectively. This concentration suggests strong research infrastructure and medical expertise in these countries for managing this rare condition.

The trials showcase diverse therapeutic strategies including gene-editing therapies, oral preventive medications, subcutaneous injections, and intravenous infusions. Several medications appear in multiple trials, indicating advanced development stages. Notably, deucrictibant features in two separate studies evaluating both initial safety and long-term effectiveness. Similarly, NTLA-2002 appears in three trials examining different aspects of this innovative gene-editing approach.

Research includes both adult and pediatric populations, with some trials specifically designed for children as young as 2 years old. Many studies focus on long-term safety monitoring, reflecting the chronic nature of the condition and the need for sustained treatment effectiveness. The variety of administration methods offers potential options for different patient preferences and clinical situations.

These trials represent important steps toward expanding treatment options for people living with hereditary angioedema, a condition that significantly impacts quality of life through unpredictable and potentially dangerous swelling episodes.

Ongoing Clinical Trials on Hereditary angioedema

  • Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Bulgaria France Germany Hungary Ireland +5

  • Long-Term Safety Study of NTLA-2002 for Patients with Hereditary Angioedema, Using Messenger RNA Encoding Cas9 and HU-G012267

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany The Netherlands

  • Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia France Germany Italy Poland Slovakia +1

  • Study on the Effect and Safety of BW-20805 for Adults with Hereditary Angioedema

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy Poland Spain

  • Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany The Netherlands

  • Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria France Germany Hungary Ireland Italy +4

  • Study on the Safety and Effects of Berotralstat for Children Aged 2 to 12 with Hereditary Angioedema (HAE)

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy Poland Spain

  • Study on the Safety of Donidalorsen for Patients with Hereditary Angioedema

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study on the Long-Term Safety of STAR-0215 for Adults with Hereditary Angioedema

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Czechia Germany Poland

  • Study on Oral PHA-022121 for Preventing Angioedema Attacks in Patients with Hereditary Angioedema

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Austria Bulgaria Germany Ireland Italy Poland

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