Long term prophylaxis against acute attacks of Hereditary Angioedema
Research type
Research Study
Full title
HELP Study ExtensionTM: An Open-Label Study to Evaluate the Long-\nTerm Safety and Efficacy of DX-2930 for Prevention Against Acute\nAttacks of Hereditary Angioedema (HAE)
IRAS ID
212411
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Dyax a fully owned subsidiary of Shire plc.
Eudract number
2015-005255-27
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
116647, Investigator New Drug Application (IND)
Duration of Study in the UK
1 years, 2 months, 13 days
Research summary
Safety and efficacy evaluation of DX2930 in the long term prevention of angioedema attacks in Subjects with HAE (Hereditary Angioedema)\n \nHAE 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.\n \nHAE 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.\n \nDX-2930 is a monoclonal antibody that is a highly potent and specific inhibitor of plasma kallikrein. \n \nThis study will look at long term safety and long term efficacy of DX2930 over a period of 12 months, followed by an 4-week follow-up.\n \nPatient who complete DX2930-03 are eligible to roll over into DX2930-04, as well as new patients who may not have been eligible for DX2930-03.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
16/NW/0789
Date of REC Opinion
30 Nov 2016
REC opinion
Further Information Favourable Opinion