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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02352493
Recruitment Status : Completed
First Posted : February 2, 2015
Last Update Posted : September 6, 2017
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief 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

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 62 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (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
Study Start Date : January 2015
Actual Primary Completion Date : April 2016
Actual Study Completion Date : August 2017

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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 :
  1. 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

  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 ]
  3. 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 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02352493

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

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Responsible Party: Alnylam Pharmaceuticals Identifier: NCT02352493     History of Changes
Other Study ID Numbers: ALN-CC5-001
First Posted: February 2, 2015    Key Record Dates
Last Update Posted: September 6, 2017
Last Verified: September 2017
Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases