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New treatment option for Acute Hereditary Angio Edema
Official title 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
ClinicalTrials.gov ID: NCT06361537
What this study is testing
What is OCTA-C1-INH?
OCTA-C1-INH is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for acute hereditary angio edema.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- 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...
- Has a documented congenital C1-INH functional activity \<50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference...
- Participant or the participant's legally authorized representative(s) has signed informed consent (as required by local law), with the assent of...
You likely can't join if
- 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
See the full eligibility criteria
Who can 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...
- 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
- 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...
- 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...
- 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...
- 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...
- Has confirmed QAT per definition criteria
- Has a swelling episode that is new and not the continuation of a previous HAE attack
What rules you out
- 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
- 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...
- (applicable until IDMC review of the interim preliminary safety and how well it works data): has clinically significant derangement in measurements of cardiovascular status (i.e. uncontrolled arterial hypertension...
- 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...
- 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...
- 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 Exclusion Criteria for IMP Dosing for PK:
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Centennial, Colorado, United States
- Farmington Hills, Michigan, United States
- Toledo, Ohio, United States
- Tirana, Albania
- Rosario, Argentina
- Yerevan, Armenia
- Sofia, Bulgaria
- Bangalore, India
- Patna, India
- Mexico City, Mexico
- México, Mexico
- Podgorica, Montenegro
- Lima, Peru
- Cluj-Napoca, Romania
- Kragujevac, Serbia
- Ankara, Turkey (Türkiye)
- Istanbul, Turkey (Türkiye)
- Izmir, Turkey (Türkiye)
- Sakarya, Turkey (Türkiye)
- Kyiv, Ukraine
+ 1 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Centennial, Colorado, United States; Farmington Hills, Michigan, United States; Toledo, Ohio, United States; Tirana, Albania; Rosario, Argentina; Yerevan, Armenia and 15 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.