Acute Hereditary Angio EdemaPhase 3NCT06361537
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
A Phase 3 study testing OCTA-C1-INH in people with Acute Hereditary Angio Edema, run by Octapharma at 25 sites in 13 countries.
Trial summary
Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Acute Hereditary Angio Edema
- What is being tested?
- OCTA-C1-INH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 124 participants
- Where
- 25 sitesin 13 countries
- Study dates
- 30 Apr 2024to Jun 2027
- Phase
- Phase 3
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Is at least 18 years of age (applicable for 1st study phase) or is at least 2 years of age (applicable for 2nd study phase)
- Has confirmed diagnosis of HAE type I or II
- Has had at least 3 moderate or severe HAE attacks (excluding extremity attacks) in the last 3 months before the Screening Visit. For participants ≥2 and ≤12 years of age, has had at least 1 moderate or severe HAE attack (excluding extremity attacks) in the last 6 months before Screening Visit
- Has a documented congenital C1-INH functional activity <50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference range
- Participant or the participant's legally authorized representative(s) has signed informed consent (as required by local law), with the assent of participants legally capable of providing it, as applicable
- States willingness to comply with all study procedures and availability for the duration of the study
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- If the participant is of childbearing potential (CBP), has a negative pregnancy test and must have been using a highly effective method of contraception and continue to do so until at least 2 weeks after their last dose (for both blinded and open-label doses of IMP). Not of CBP is defined as surgically sterilized (hysterectomy, bilateral oophorectomy) or who are postmenopausal (defined as women with no menses for 12 months without an alternative medical cause). Highly effective methods of contraception:
- Combined hormonal contraception (estrogens and progesterone) methods such as oral, implantable, intravaginal, injectable, or transdermal contraceptives at a stable dose for a minimum of 1 full cycle (hormonal contraceptives must inhibit ovulation) and for at least 4 weeks before screening
- Progesterone only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
- Intrauterine device
- Intrauterine hormone-releasing system inserted at least 4 weeks before screening
- Bilateral tubal ligation/occlusion or vasectomized partner (with surgical success confirmed by medical assessment) OR Agrees to abstain from heterosexual intercourse during study participation and to use a highly effective contraceptive (as described above) as backup if they become sexually active during the study. Abstinence is only acceptable if this is the participant's usual lifestyle. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception
- Note: If a participant of CBP has a positive or suspected positive urine pregnancy test within 72 hours prior to treatment, a serum pregnancy test will be required
- Male participants must not plan to father a child or donate sperm for 90 days after their last dose of study drug (for both blinded and open-label doses of the IMP). However, there are no official contraception requirements for male participants during the study.
- Has confirmed QAT per definition criteria
- Has a swelling episode that is new and not the continuation of a previous HAE attack
Who cannot join
- Has a history of clinically relevant antibody development against C1-INH
- Has a medical history consistent with Type 3 HAE (i.e., onset at age above 40 year, no family history, no known HAE mutation, low C1q level in plasma)
- Has a history of allergic reaction to C1-INH or other blood/plasma product
- Has a history of B-cell malignancy that was unresolved in the past 5 years
- Has a narcotic and/or alcoholic addiction
- Has participated in any other investigational drug evaluation within 30 days before screening
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- Is pregnant or breastfeeding
- Has any clinically significant medical or psychiatric condition that, in the investigator's opinion would interfere with the participant's ability to participate in the study
- Has a history of thromboembolic events (TEEs), myocardial infarction, unstable angina pectoris, critical aortic stenosis, cerebrovascular accident, transient ischemic attack, severe peripheral vascular disease, or disseminated intravascular coagulation within one year before screening
- (applicable until IDMC review of the interim preliminary safety and efficacy data): has clinically significant derangement in measurements of cardiovascular status (i.e. uncontrolled arterial hypertension, cardiac insufficiency New York Heart Association (NYHA) class III-IV), pulmonary status (i.e., COPD GOLD classification 3 and 4, severe asthma) and renal status (i.e., eGFR below 90 ml/min per 1.73 m2)
- Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
- started or changed hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) within 14 days before IMP dosing
- Started or changed androgen therapy (e.g. testosterone, dehydro- epiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) within 14 days before IMP dosing or is not willing to maintain a stable dose throughout the study
- Started or changed the dose of monoclonal antibodies e.g. lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
- Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing for a QAT
- Has received OCTA-C1-INH within 14 days before IMP dosing
- Has received blood or a blood product for prophylactic or acute treatment with any C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin and kallikrein pathway inhibitors (e.g., ecallantide, icatibant, berotralstat), or treatment with tranexamic acid within 14 days before dosing with the IMP (or is not willing to abstain from these medications throughout the study)
- Is receiving hormone replacement therapy or selective estrogen receptor modulators (e.g., tamoxifen) and has had their dose changed within 14 days before IMP dosing
- Is receiving or has received androgen therapy (e.g., testosterone, dehydroepiandrosterone/androstenedione, oxandrolone, danazol, stanozolol) IN ANY DOSE within 14 days before dosing
- Started or changed the dose of monoclonal antibodies e.g lanadelumab within 11 weeks before dosing or not willing to maintain a stable dose throughout the study
- Has used narcotic pain medications or non-opioid analgesics within 7 days before IMP dosing
- Has received IMP within 14 days before IMP dosing
- Has planned dental, medical, or surgical procedures during the PK Period that will require pre-procedural prevention
Trial locations and contacts
25 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
25 sites in 13 countries
India4 sites, 4 recruiting
- Not yet recruiting
Octapharma Research Site, Chhatrapati Sambhajinagar
Aurangabad, 431003
- Not yet recruiting
Octapharma Research Site
Bangalore, 560017
- Not yet recruiting
Octapharma Research Site
Patna, 801507
- Not yet recruiting
Octapharma Research Site
Pune, Maharashtra, 411040
Turkey (Türkiye)4 sites, 3 recruiting
- Not yet recruiting
Octapharma Research Site
Ankara
- Not yet recruiting
Octapharma Research Site
Istanbul
- Withdrawn
Octapharma Research Site
Izmir
- Not yet recruiting
Octapharma Research Site
Sakarya
United States3 sites
- Withdrawn
Octapharma Research Site
Centennial, Colorado, 80112
- Withdrawn
Octapharma Research Site
Farmington Hills, Michigan, 48334
- Withdrawn
Octapharma Research Site
Toledo, Ohio, 43617
Ukraine3 sites, 2 recruiting
- Recruiting
Octapharma Research Site
Kyiv, 03057
- Withdrawn
Octapharma Research Site
Lviv, 79010
- Not yet recruiting
Octapharma Research Site
Lviv, 79035
Mexico2 sites, 2 recruiting
- Not yet recruiting
Octapharma Research Site
México
- Not yet recruiting
Octapharma Research Site
Mexico City, 06720
Peru2 sites, 2 recruiting
- Recruiting
Octapharma Research Site 5102
Lima, 15001
- Recruiting
Octapharma Research Site 5103
Lima, 15001
About this study
Official title: Prospective, Multicenter, Randomized, Double-blind, Parallel Group, Placebo- Controlled, Efficacy and Safety Phase 3 Study of an Intravenous Human Plasma- Derived C1 Esterase Inhibitor (C1-INH) Concentrate in Participants With Congenital C1-INH Deficiency for the Treatment and Pre-procedure Prevention of Acute Hereditary Angioedema Attacks
Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks
- Conditions
- Acute Hereditary Angio Edema
- Being tested
- OCTA-C1-INH, Placebo
- Keywords
- Edema, Swelling, Angio Edema, Hereditary, Congenital Angioedema
- Medical subject headings
- Edema, Angioedema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OCTA-C1-INHDrug
OCTA-C1-INH is a stable, sterile, virus-inactivated, nano-filtered, highly purified concentrate of human C1-INH prepared from pooled human plasma. After reconstitution in 2.5mL water for injection, the solution can be administered as a slow IV injection. OCTA-C1-INH is given as a dose of 20 IU/kg body weight (BW)
PlaceboOther
0.1 mL/kg BW 0.9% sodium chloride injection
| Study group | Role | What they receive |
|---|---|---|
| OCTA-C1-INH | Experimental | OCTA-C1-INH injection, 20IU/kg BW after first qualifying attack. Treatment to be administered to blinded as well as open-label subjects. Receives: Drug: OCTA-C1-INH |
| Placebo | Placebo comparator | 0.1 mL/kg BW 0.9% sodium chloride solution injection after first qualifying attack. Only blinded subjects to receive. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time (h) to beginning of unequivocal symptom relief at the defining site in blinded participants.
Patient will rate symptom relief for the defining attack site (site of swelling or pain) from 15 minutes after start of the IMP injection every 15 minutes over 4 hours. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade SRRS.
This measure will also be used for the secondary outcomes in a different context.
Measured: Within 4 hours after injection
Secondary outcomes (3)
Percentage of participants responding to treatment
Percentage of patients responding to treatment, defined as beginning of unequivocal symptom relief at the defining site within 4 hours after injection (once per participant after first qualifying attack in the study). Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
Measured: within 4 hours after injection
Time to beginning of unequivocal symptom relief at all sites involved
Time to the beginning of unequivocal symptom relief at all sites involved within 4 hours after injection. Unequivocal relief is defined as having 3 consecutive reports of "absent now but present before," "absent now and absent before", or "present, symptoms better" on the 5-grade symptom relief rating.
Measured: Within 4 hours after injection
Changes in symptom severity at the defining site by VAS severity rating
Changes in symptom severity at the defining site by visual analog scale (VAS) rating from pre-injection over 4 hours after injection. The VAS is a self-reported 0-100 pain and swelling rating scale.
Measured: Within 4 hours after injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2024
- Study start30 Apr 2024
- Primary completionDec 2026 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hereditary Angio EdemaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 124 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06361537
- Sponsor's protocol ID
- CONE-02
- Status last verified
- Aug 2026
- First submitted
- 11 Jan 2024
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No