SAD and MAD study of ALN-CC5 in Healthy Volunteers and PNH Patients
Research type
Research Study
Full title
A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
164888
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Alnylam Pharmaceuticals
Eudract number
2014-002462-69
Duration of Study in the UK
1 years, 3 months, 27 days
Research summary
We are conducting a First-Time-In-Humans clinical study with an investigational medication known as ALN-CC5 which is being developed for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare, genetically acquired, life-threatening disease of the blood which causes destruction of red blood cells (i.e. hemolytic anemia), blood clot formation (i.e. thrombosis) and prevents the bone marrow from functioning properly; due to an overreaction in the immune system. Some of the common symptoms of PNH include severe abdominal pain, severe headaches, back pain, excessive weakness, fatigue and recurrent infections.
Not all patients can be treated adequately with the currently available medications, such as eculizumab which has to be administered as an intravenous infusion every 2 weeks. Hence there is a need for new drugs such as ALN-CC5 that can be administered more easily and less frequently as it is given subcutaneously (sc). ALN-CC5 targets a blood protein known as complement 5 (C5) and serum complement activity involved in the immune system.
In this study we will assess the safety of ALN-CC5 and how well it is tolerated when it is given as a single increasing dose in the SAD part of the study (Part A - up to 32 healthy volunteers); or multiple doses (Part B - up to 28 healthy volunteers) and Part C (up to 8 PNH patients) via subcutaneous injections. We will also measure how much ALN-CC5 is found in the blood and urine after sc dosing (i.e. pharmacokinetics) and the levels of C5 and Complement activity in the blood, to determine if ALN-CC5 has any impact on these levels (i.e. pharmacodynamics).
The study will be conducted at 2 phase 1 clinical research units and up to 2 hospital sites and is funded by Alnylam Pharmaceuticals Inc (the sponsors of the study) based in the USA.
REC name
London - London Bridge Research Ethics Committee
REC reference
14/LO/1990
Date of REC Opinion
14 Jan 2015
REC opinion
Further Information Favourable Opinion