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

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Study Design

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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

Arms and Interventions

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Outcome Measures

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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 ]

Eligibility Criteria

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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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

Spain
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

Investigators

Study Director:	Nader Najafian, MD	Alnylam Pharmaceuticals

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT02352493 History of Changes
Other Study ID Numbers:	ALN-CC5-001
First Posted:	February 2, 2015
Last Update Posted:	September 6, 2017
Last Verified:	September 2017

Keywords provided by Alnylam Pharmaceuticals:

PNH; RNAi therapeutic

Additional relevant MeSH terms:

Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Urologic Diseases; Urological Manifestations	Signs and Symptoms; Anemia, Hemolytic; Anemia; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases