Proof of Concept Study of ACH 0144471 in Untreated Patients With PNH
Research type
Research Study
Full title
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH 0144471 in Untreated Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)
IRAS ID
235225
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Achillion Pharmaceuticals Inc.
Eudract number
2016-002652-25
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 10 months, 4 days
Research summary
This study is being done to find out how safe and effective the study drug ACH-0144471 is at controlling the complement system, by inhibiting Factor D, in patients with untreated paroxysmal nocturnal haemoglobinura (PNH). The complement system is found in the blood and helps or "complements" the ability of the body to get rid of many disease-causing organisms. It is part of the body’s defence system. Many diseases are associated with either an inefficient control of complement or too much activity of the complement system. Factor D is one of the proteins within the complement system and the sponsor (Achillion Pharmaceuticals) believes that blocking these proteins will help treat certain diseases, such as PNH, affecting the complement system.
Following a screening period of up to 60 days (including vaccinations, when applicable), patients will enter the treatment portion of the study which will be conducted in two parts.
Each patient will receive doses of ACH-0144471 tablets three times a day for a total of 28 days during Part 1, and may be treated for an additional 8 weeks if they participate in Part 2. Additionally there will be a 6-day taper of study drug at the end of treatment. Including the taper, the total study duration for patients who complete Part 1 will be 48 days and for patients who complete both Part 1 and 2, the total study duration will be up to 104 days (not including screening).
This study will include 4-12 patients from UK, Europe, South Korea and New Zealand.
A long-term extension study may be offered to patients, if supported by clinical and nonclinical data. Pending regulatory approval of such a study, patients who, in the opinion of the Principal Investigator, are receiving benefit from ACH 0144471 may be enrolled directly into that study without interruption from this study.
REC name
London - City & East Research Ethics Committee
REC reference
17/LO/1989
Date of REC Opinion
2 Feb 2018
REC opinion
Further Information Favourable Opinion