Phase 2 N=62 Randomized Double-blind Treatment

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

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Bottom Line

View on ClinicalTrials.gov: NCT02352493 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Mar 2020

Primary outcome: Primary: Number of Participants With Adverse Events — 6; 0; 3; 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ALN-CC5 (Drug); Sterile Normal Saline (0.9% NaCl) (Drug)
Age: Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Apr 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Adverse Events	6; 0; 3; 2; 2; 3	—
SECONDARY Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)	18.823; 49.321; 26.337; 64.435; 47.628; 58.525	—
SECONDARY Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)	18.417; 46.953; 43.615; 72.939; 63.582; 72.809	—
SECONDARY Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels	15.88; 77.61; 73.44; 93.17; 88.62; 94.60	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer)	14.4; 13.5; 69.8; 93.9; 111; 166	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer)	27.2; 30.4; 144; 178; 384; 787	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer)	0.50; 0.50; 0.50; 0.50; 0.50; 0.50	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer)	8.00; 8.00; 8.00; 6.00; 12.00; 4.00	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer)	6.63; 7.50; 182; 198; 287; 574	—
SECONDARY Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer)	258; 277; 2290; 2470; 5450; 10040	—

Summary

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

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02352493). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.