This trial is active, not recruiting.

Condition paroxysmal nocturnal hemoglobinuria (pnh)
Treatments aln-cc5, sterile normal saline (0.9% nacl)
Phase phase 1/phase 2
Sponsor Alnylam Pharmaceuticals
Start date January 2015
End date December 2016
Trial size 76 participants
Trial identifier NCT02352493, ALN-CC5-001


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

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
(Active Comparator)
Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection
(Placebo Comparator)
sterile normal saline (0.9% nacl)
calculated volume to match active comparator

Primary Outcomes

The safety of ALN-CC5 evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
time frame: Part A: through day 70; Part B: through day 140; Part C: through day 140

Secondary Outcomes

Profile of Pharmacokinetics (PK) of ALN-CC5
time frame: Part A: up to 56 days; Part B: up to 56, days; Part C: up to 85 days
The effect of ALN-CC5 on serum levels of complement activity
time frame: Part A: through day 70; Part B: through day 140; Part C through day 140
The effect of ALN-CC5 on serum levels of C5 protein
time frame: Part A: through day 70; Part B: through day 140; Part C through day 140

Eligibility Criteria

Male or female participants from 18 years up to 45 years old.

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

Additional Information

Official 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
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.