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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-01-31

Study Completion Date

2017-08-31

Brief Summary

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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

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Detailed Description

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Conditions

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Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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ALN-CC5

Group Type ACTIVE_COMPARATOR

ALN-CC5

Intervention Type DRUG

Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection

Sterile Normal Saline (0.9% NaCl)

Group Type PLACEBO_COMPARATOR

Sterile Normal Saline (0.9% NaCl)

Intervention Type DRUG

calculated volume to match active comparator

Interventions

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ALN-CC5

Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection

Intervention Type DRUG

Sterile Normal Saline (0.9% NaCl)

calculated volume to match active comparator

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes