A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH

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

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH

Inclusion Criteria:

• Adequate complete blood counts, liver and renal function
• 12-lead electrocardiogram (ECG) within normal limits
• Female subjects of child bearing potential agreeing to use a protocol specified method of contraception
• Male subjects agreeing to use protocol specified methods of contraception
• Willing to provide written informed consent and willing to comply with study requirements

Exclusion Criteria:

• Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
• Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
• History of multiple drug allergies or intolerance to subcutaneous injection
• Parts A and B of the study: Used prescription medications within 14 days or 7 half-lives of administration of the first dose of study drug.
• History of meningococcal infection