Genetic DisordersPhase 2/3NCT00262288
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
A Phase 2/3 study testing i.v. recombinant human C1 inhibitor in people with Genetic Disorders, run by Pharming Technologies B.V. at 1 site in Netherlands.
Trial summary
The purpose of this multi-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary angioedema. Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Genetic Disorders
- What is being tested?
- i.v. recombinant human C1 inhibitor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteNetherlands
- Study dates
- Apr 2004to Jan 2007
- Phase
- Phase 2/3
- 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 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Main inclusion Criteria:
- Clinical and laboratory diagnosis of HAE
- Plasma level of functional C1INH of less than 50% of normal
- Severe attack of abdominal, facial-oro-pharyngeal, genito-urinary and/or peripheral HAE.
Main exclusion Criteria:
- Acquired angioedema
- Pregnancy or breastfeeding
- Participation in another clinical study within prior 3 months
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
For information on sites in Europe, please contact Pharming Technologies.
Leiden, 2333 CN
About this study
Official title: A Phase II/III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
The purpose of this multi-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary angioedema.
- Conditions
- Genetic Disorders
- Being tested
- i.v. recombinant human C1 inhibitor
- Medical subject headings
- Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
i.v. recombinant human C1 inhibitorDrug
Also called rhC1INH
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Human C1INH | Experimental | Not described Receives: Drug: i.v. recombinant human C1 inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary outcomes: Relief of angioedema symptoms
Measured: 24 hours
Secondary outcomes (1)
Secondary outcomes: Safety and tolerability; pharmacokinetics/pharmacodynamics
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2005 (estimated)
- Study startApr 2004
- Primary completionJan 2007
- Study completionJan 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jan Nuijens, MD, PhD, Study chair, Pharming Technologies B.V.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00262288
- Sponsor's protocol ID
- C1 1203-01
- Status last verified
- Feb 2013
- First submitted
- 1 Dec 2005
- Last updated
- 22 Feb 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT00262288
- Acronym / sponsor ID
- C1 1203-01
- Conditions
- Genetic Disorders
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Apr 2004
- Primary completion
- Jan 2007 (actual)
- Study completion
- Jan 2007 (actual)
- First posted
- 6 Dec 2005 (estimated)
- 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 purpose of this multi-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary angioedema.
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 C1INH | Experimental | Drug: i.v. recombinant human C1 inhibitor |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| i.v. recombinant human C1 inhibitorAlso: rhC1INH | Drug | Recombinant Human C1INH |
Primary outcome
Primary outcomes: Relief of angioedema symptoms
Time frame: 24 hours
1 secondary outcome
Secondary outcomes: Safety and tolerability; pharmacokinetics/pharmacodynamics
Time frame: 90 days
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 22 Feb 2013.
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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.
Jan Nuijens, MD, PhD study chair
Pharming Technologies B.V.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing i.v. recombinant human C1 inhibitor in people with Genetic Disorders, run by Pharming Technologies B.V. at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 22 Feb 2013. 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 Netherlands. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Primary outcomes: Relief of angioedema symptoms (time frame: 24 hours). 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 22 Feb 2013.