Prophylaxis against acute attacks of Hereditary Angioedema (HAE)
Research type
Research Study
Full title
HELP Study (TM): A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX 2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)
IRAS ID
184624
Contact name
Ryan Iarrobino
Contact email
Sponsor organisation
Dyax Corp
Eudract number
2015-003943-20
Clinicaltrials.gov Identifier
Investigator New Drug Application (IND), 116647
Duration of Study in the UK
1 years, 0 months, 27 days
Research summary
Evaluation of DX2930 in the prevention of angioedema attacks in Subjects with HAE (Hereditary Angioedema)
HAE is a rare disease of the immune system that causes attacks of spontaneous swelling that can be painful and sometimes severe. The most common locations of HAE attacks are the hands, feet, face, genitals, abdomen and larynx. The frequency, severity and location of HAE symptoms can vary widely even in the same patient. Swelling can occur without warning and may last up to five or more days; most patients suffer multiple attacks per year. Abdominal attacks are often misdiagnosed and can results in unnecessary surgery or invasive procedures. Because laryngeal swelling can result in death from asphyxiation, HAE mortality has been estimated at up to 30% in undiagnosed individuals.
HAE is a genetic disorder that is caused by a mutation in the protein, C1-esterase inhibitor, or C1-INH. One of the functions of this protein is to regulate the activity of a pathway called the kallikrein-kinin pathway. Low levels of functioning C1-INH causes a reaction in the pathway and leads to the over-activation plasma kallikrein. When plasma kallikrein is activated, it causes the generation of a potent vasodilator, bradykinin, that leads to swelling. If the pathway is not regulated properly, too much activity of plasma kallikrein and over-accumulation of bradykinin lead to HAE attacks.
DX-2930 is a monoclonal antibody that is a highly potent and specific inhibitor of plasma kallikrein. In the proposed study the primary question being asked is,how effective is DX-2930 in preventing HAE attacks? The study will include patients with HAE, 18 years of age and above, who meet the entry criteria. The duration of the study can be between 30 and 44 weeks (approximately 7 to 10 months).
REC name
London - Central Research Ethics Committee
REC reference
15/LO/1945
Date of REC Opinion
11 Jan 2016
REC opinion
Further Information Favourable Opinion