A Phase 2 interventional study of rhC1INH in Hereditary Angioedema, sponsored by Pharming Technologies B.V.. Completed at 16 sites in 10 countries. Open to participants aged 2 Years to 13 Years. Per ClinicalTrials.gov, last updated 2024-03-29.
Sponsored by Pharming Technologies B.V. (part of Pharming Group N.V.) · Phase 2, Interventional, and Treatment
This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.
This study was an open-label, Phase 2, non-comparative, multinational, multicenter clinical study in pediatric patients from 2 up to and including 13 years of age, with a confirmed diagnosis of HAE. Patients were eligible for treatment with recombinant human C1-inhibitor (rhC1INH) if they presented to the clinic within 5 hours of onset with an acute attack of at least moderate severity without signs of spontaneous regression. Patients received rhC1INH at a dose of 50 U/kg body weight up to a maximum of 4200 U. The reconstituted solution was administered as a slow intravenous (iv) injection over approximately 5 minutes. The patients remained in hospital and were closely monitored in the study center for at least 4 hours after study medication administration.
171 studies on the registry are indexed under Angioedemas, Hereditary; 24 are open to participants now.
This study's enrollment of 57 is above the median of 48 across 116 interventional studies indexed under Angioedemas, Hereditary.
Browse Angioedemas, Hereditary studies →Pharming Technologies B.V. is the lead sponsor of 22 studies on the registry; 3 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
Drug: rhC1INH
Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
Also known as: Ruconest
Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief.
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
Time frame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.
Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations
Time to minimal symptoms was defined as the time at which the Overall VAS score fell below 20 mm for all locations where VAS Scores were recorded. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines provided to answer each question related to the severity of symptoms and the patient's condition. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the OPL VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination.
Time frame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.
Male and female patients, from 2 up to and including 13 years of age with a clinically suspected and/or confirmed diagnosis of HAE will be recruited for this study. Patients will be identified and invited to participate by the investigators at the respective study centers.
| Milestone | Recombinant Human C1 Inhibitor |
|---|---|
| Started | 57 |
| Completed | 20 |
| Not completed | 37 |
| Withdrew: Study continued until at least 20 patients had been treated for at least one treatment. | 37 |
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
| minutes | Recombinant Human C1 Inhibitor |
|---|---|
| Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief. | 60 (60 to 65) |
Time to minimal symptoms was defined as the time at which the Overall VAS score fell below 20 mm for all locations where VAS Scores were recorded. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines provided to answer each question related to the severity of symptoms and the patient's condition. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the OPL VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination.
| minutes | Recombinant Human C1 Inhibitor |
|---|---|
| Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations | 122.5 (120 to 126) |
Collected over Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).. Non-serious events are listed at a 3% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Recombinant Human C1 Inhibitor | 0/20 (0%) | 3/20 (15%) | 11/20 (55%) |
| Event | Recombinant Human C1 Inhibitor |
|---|---|
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 1/20 |
| Pharyngeal oedemaRespiratory, thoracic and mediastinal disorders | 1/20 |
| VomitingGastrointestinal disorders | 1/20 |
| BronchitisInfections and infestations | 1/20 |
| PneumoniaInfections and infestations | 1/20 |
| TonsillitisInfections and infestations | 1/20 |
| Viral infectionInfections and infestations | 1/20 |
| Eye swellingEye disorders | 1/20 |
| UrticariaSkin and subcutaneous tissue disorders | 1/20 |
| Event | Recombinant Human C1 Inhibitor |
|---|---|
| NasopharyngitisInfections and infestations | 3/20 |
| VomitingGastrointestinal disorders | 3/20 |
| Lymphocyte morphology abnormalVascular disorders | 2/20 |
| Viral infectionInfections and infestations | 2/20 |
| PallorVascular disorders | 1/20 |
| Body temperature increasedVascular disorders | 1/20 |
| CatarrhRespiratory, thoracic and mediastinal disorders | 1/20 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 1/20 |
| Pharyngeal oedemaRespiratory, thoracic and mediastinal disorders | 1/20 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 1/20 |
| Age, Categorical(Participants) | Recombinant Human C1 Inhibitor |
|---|---|
| <=18 years | 20 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Recombinant Human C1 Inhibitor |
|---|---|
| Median | 9.55 ± 3.304 |
| Sex: Female, Male(Participants) | Recombinant Human C1 Inhibitor |
|---|---|
| Female | 9 |
| Male | 11 |
| Race (NIH/OMB)(Participants) | Recombinant Human C1 Inhibitor |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 19 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Recombinant Human C1 Inhibitor |
|---|---|
| Romania | 2 |
| Hungary | 1 |
| Czechia | 2 |
| United States | 1 |
| Poland | 2 |
| North Macedonia | 2 |
| Israel | 9 |
| Germany | 1 |
| Height at screening(centimeters) | Recombinant Human C1 Inhibitor |
|---|---|
| Mean | 125.6 ± 25.78 |
| Weight at presentation(kilograms) | Recombinant Human C1 Inhibitor |
|---|---|
| Mean | 34.81 ± 20.554 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2012 to primary completion in Jul 2017, 66 months.
Results were first posted in Mar 2024, 6 years 8 months after primary completion. A results delay was requested in Jul 2018.
Of 17 phase 2 interventional studies under Angioedemas, Hereditary, half reached primary completion within 19 months of starting (the median), counting those still running; a quarter within 11 and three quarters within 35. This study's 66 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Conestat alfa