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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02352493
• Date of registration: 23/01/2015
• Prospective Registration: No
• Primary sponsor: Alnylam Pharmaceuticals
• Public title: A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
• Scientific 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
• Date of first enrolment: January 2015
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02352493
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 1/Phase 2

Countries of recruitment

Spain, United Kingdom

Contacts

• Name: Nader Najafian, MD
• Affiliation: Alnylam Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 45 Years
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention(s)

Drug: ALN-CC5

Drug: Sterile Normal Saline (0.9% NaCl)

Primary Outcome(s)

Number of Participants With Adverse Events [Time Frame: Part A: through day 658; Part B: through day 532; Part C: through day 280]

Secondary Outcome(s)

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP) [Time Frame: Part A: through day 70; Part B: through day 140; Part C: through day 140]

Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP) [Time Frame: Part A: through day 70; Part B: through day 140; Part C: through day 140]

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels [Time Frame: Part A: through day 70; Part B: through day 140; Part C: through day 140]

Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer) [Time Frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84]

Secondary ID(s)

ALN-CC5-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02352493