Study of the Safety and PK of TT30 in Subjects with PNH
Research type
Research Study
Full title
A Phase 1, Single-Ascending Dose Study of the Safety and Pharmacokinetics (PK) of TT30 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH).
IRAS ID
79503
Contact name
Modupe Elebute
Sponsor organisation
Alexion Pharmaceuticals
Eudract number
2010-022286-98
Clinicaltrials.gov Identifier
Research summary
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that occurs when there is a problem with part of the immune system, called complement, which causes red blood cells to break down too quickly (also known as hemolysis) and lowers red blood cell counts. TT30 is a protein that is being developed for the treatment of (PNH). TT30 is designed to target complement activity to stop the breakdown of red blood cells.The primary purpose of this research study is to understand any side effects of TT30 when given directly into the vein on one singular occasion, to people with PNH. The first participants to receive TT30 will be given a very low dose of the drug, the dose will then be increased with each cohort of participants added. The study will look at the levels of TT30 in the blood, how the drug behaves inside the body and how it is eliminated from the body (pharmacokinetics). The study will determine how TT30 affects a part of the immune system known as complement (pharmacodynamics). About 8 participants from 15 locations in the United States and Europe are expected to take part. This may increase up to 24 participants. Participants will be in this study for approximately 88 days and come to the clinic 11 times to see how they are doing. Participants will be assigned to one of five dose cohorts (0.1 mg, 0.3 mg, 1 mg, 3 mg, and 5 mg/kg) depending on when they enroll in the study.The study involves procedures including:- physical exam, height, weight, vital signs, ECG, Blood and urine tests, telemetry, AE/SAE reporting.Since this is the first time that TT30 is being administered to human subjects, the Safety Review Committee (SRC) will review all data on participants on an ongoing basis to ensure it is safe to continue the study.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
11/LO/0895
Date of REC Opinion
14 Sep 2011
REC opinion
Further Information Favourable Opinion