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

Status: Recruiting
Location: See all (22) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 3
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 hereditary angioedema attacks

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:

• 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

• 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

Locations
United States
Colorado
Octapharma Research Site
RECRUITING
Centennial
Michigan
Octapharma Research Site
RECRUITING
Farmington Hills
Ohio
Octapharma Research Site
RECRUITING
Toledo
Other Locations
Albania
Octapharma Research Site
NOT_YET_RECRUITING
Tirana
Argentina
Octapharma Research Site
NOT_YET_RECRUITING
Rosario
Armenia
Octapharma Research Site
RECRUITING
Yerevan
Bulgaria
Octapharma Research Site
RECRUITING
Sofia
Mexico
Octapharma Research Site
NOT_YET_RECRUITING
Lima
Octapharma Research Site
NOT_YET_RECRUITING
Mexico City
Montenegro
Octapharma Research Site
NOT_YET_RECRUITING
Podgorica
Peru
Octapharma Research Site
WITHDRAWN
Lima
Octapharma Research Site
NOT_YET_RECRUITING
Lima
Octapharma Research Site
NOT_YET_RECRUITING
Lima
Romania
Octapharma Research Site
RECRUITING
Cluj-napoca
Serbia
Octapharma Research Site
RECRUITING
Kragujevac
Turkey
Octapharma Research Site
NOT_YET_RECRUITING
Ankara
Octapharma Research Site
NOT_YET_RECRUITING
Istanbul
Octapharma Research Site
NOT_YET_RECRUITING
Izmir
Octapharma Research Site
NOT_YET_RECRUITING
Sakarya
Ukraine
Octapharma Research Site
RECRUITING
Kyiv
Octapharma Research Site
RECRUITING
Lviv
Octapharma Research Site
NOT_YET_RECRUITING
Lviv
Contact Information
Primary
Patrick M Murphy
ctgov@clinicalresearchmgt.com
8663371868
Time Frame
Start Date: 2024-04-30
Estimated Completion Date: 2027-06
Participants
Target number of participants: 124
Treatments
Experimental: OCTA-C1-INH
OCTA-C1-INH injection, 20IU/kg BW after first qualifying attack. Treatment to be administered to blinded as well as open-label subjects.
Placebo_comparator: Placebo
0.1 mL/kg BW 0.9% sodium chloride solution injection after first qualifying attack. Only blinded subjects to receive.
Related Therapeutic Areas
Sponsors
Leads: Octapharma

This content was sourced from clinicaltrials.gov