NCT06361537 · Octapharma
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
What this study is about
forward-looking, conducted at multiple hospitals, randomly assigned, where neither patients nor doctors know which treatment is given, parallel group, placebo- controlled, effectiveness and safety phase 3 study of an given through a vein (IV) 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
View original scientific description
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
Interventions
DRUG
OCTA-C1-INH
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)
OTHER
Placebo
0.1 mL/kg BW 0.9% sodium chloride injection
Primary outcome measures
Time (h) to beginning of unequivocal symptom relief at the defining site in blinded participants.
Time frame: Within 4 hours after injection
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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) 2. Has confirmed diagnosis of HAE type I or II 3. 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 4. Has a documented congenital C1-INH functional activity \<50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference range 5. 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 6. States willingness to comply with all study procedures and availability for the duration of the study 7. If the participant is of childbearin
Where
- Centennial, Colorado
- Farmington Hills, Michigan
- Toledo, Ohio
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 30, 2026 · Source of record for eligibility and locations