This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02352493
First received: January 23, 2015
Last updated: June 2, 2017
Last verified: June 2017
  Purpose
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

Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: ALN-CC5 Drug: Sterile Normal Saline (0.9% NaCl) Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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 ]
    Cmax, tmax, Area Under Curve, t1/2

  • 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 ]

Estimated Enrollment: 76
Study Start Date: January 2015
Estimated Study Completion Date: August 2017
Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-CC5 Drug: ALN-CC5
Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02352493

Locations
Spain
Clinical Trial Site
Barcelona, Spain
United Kingdom
Covance Clinical Research Unit
Leeds, United Kingdom
Richmond Pharmacology
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Nader Najafian, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02352493     History of Changes
Other Study ID Numbers: ALN-CC5-001
Study First Received: January 23, 2015
Last Updated: June 2, 2017

Keywords provided by Alnylam Pharmaceuticals:
PNH
RNAi therapeutic

Additional relevant MeSH terms:
Hemoglobinuria, Paroxysmal
Hemoglobinuria
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms

ClinicalTrials.gov processed this record on June 27, 2017