Genetic DisordersPhase 2OPERANCT00851409
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
A Phase 2 study testing Recombinant Human C1 Inhibitor in people with Genetic Disorders and Hereditary Angioedema, run by Pharming Technologies B.V. at 2 sites in 2 countries.
Trial summary
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Genetic Disorders, Hereditary Angioedema
- What is being tested?
- Recombinant Human C1 Inhibitor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jun 2009to Apr 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged at least 18 years
- Signed informed consent
- Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.
Who cannot join
- A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
- A history of allergic reactions to C1INH products or rabbit protein.
- Any reported SAE related to study drug administration (withdrawal criterium)
- Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
- A diagnosis of acquired C1INH deficiency.
- Woman of child bearing potential, pregnancy or breast-feeding
Show all 10Show fewer
- previous treatment within the last 3 months with plasma-derived C1INH
- Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis
- Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
- Any changes since screening that would exclude subject based on above exclusion criteria.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Netherlands1 site
For information on sites, please contact Pharming Technologies
Leiden
Romania1 site
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Târgu Mureş, 541103
About this study
Official title: An Open-label Exploratory Phase II Study of the Safety and Immunogenicity of Repeated "rhC1INH" Administration of 50 U/Kg in Patients With Hereditary C1 Inhibitor Deficiency ("HAE")
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").
- Conditions
- Genetic Disorders, Hereditary Angioedema
- Being tested
- Recombinant Human C1 Inhibitor
- Medical subject headings
- Genetic Diseases, Inborn, Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human C1 InhibitorDrug
Also called "rhC1INH", Ruconest
50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Human C1 Inhibitor | Other | Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor Receives: Drug: Recombinant Human C1 Inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
HAE Attacks/Week
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
Measured: 8 weeks
Secondary outcomes (1)
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
"PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4)
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2009 (estimated)
- Study startJun 2009
- Primary completionMar 2010
- Study completionApr 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Genetic DisordersStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bruno Giannetti, MD, Study chair, COO Pharming
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00851409
- Sponsor's protocol ID
- C1 1207
- Acronym
- OPERA
- Status last verified
- Apr 2018
- First submitted
- 25 Feb 2009
- Last updated
- 26 Jun 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Exploratory Phase II Study of the Safety and Immunogenicity of Repeated "rhC1INH" Administration of 50 U/Kg in Patients With Hereditary C1 Inhibitor Deficiency ("HAE")
- ClinicalTrials.gov ID
- NCT00851409
- Acronym / sponsor ID
- OPERA / C1 1207
- Conditions
- Genetic Disorders, Hereditary Angioedema
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Jun 2009
- Primary completion
- Mar 2010 (actual)
- Study completion
- Apr 2010 (actual)
- First posted
- 26 Feb 2009 (estimated)
- Results
- Posted 31 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Recombinant Human C1 Inhibitor | Other | Drug: Recombinant Human C1 InhibitorWeekly administration of 50 IU/kg Recombinant Human C1 Inhibitor |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Aged at least 18 years
- Signed informed consent
- Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.
Key exclusion criteria (10 listed)
- A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
- A history of allergic reactions to C1INH products or rabbit protein.
- Any reported SAE related to study drug administration (withdrawal criterium)
- Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
- A diagnosis of acquired C1INH deficiency.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Human C1 InhibitorAlso: "rhC1INH", Ruconest50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period. | Drug | Recombinant Human C1 Inhibitor |
Primary outcome
HAE Attacks/Week
Time frame: 8 weeks
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
1 secondary outcome
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Bruno Giannetti, MD study chair
COO Pharming
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Recombinant Human C1 Inhibitor in people with Genetic Disorders and Hereditary Angioedema, run by Pharming Technologies B.V. at 2 sites in 2 countries.
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 3 inclusion and 10 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 26 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands, Romania. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Jan 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
HAE Attacks/Week (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Jun 2018.