A Phase 1 Study of ALN-AS1 in Patients With Acute Intermittent Porphyria (AIP)

This study is currently recruiting participants.

See

Contacts and Locations

Verified June 2017 by Alnylam Pharmaceuticals

Sponsor:

Information provided by (Responsible Party):

Alnylam Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT02452372

First received: May 19, 2015

Last updated: June 2, 2017

Last verified: June 2017

History of Changes

Tracking Information

First Received Date ^ICMJE

May 19, 2015

Last Updated Date

June 2, 2017

Start Date ^ICMJE

May 2015

Primary Completion Date

May 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

The safety of ALN-AS1 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 (SAD phase): through day 42; Part B (MAD) phase: through Day 70; Part C (MD) phase: through Day 168 ]

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT02452372 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Profile of Pharmacokinetics (PK) of ALN-AS1 [ Time Frame: Part A (SAD) phase: predose - 42 days post-dose; Part B (MAD) phase: predose - 70 days post-dose; Part C (MD) phase: predose - 168 days post-dose ]
Cmax, tmax, AUC, t1/2
The change in delta-aminolevulinic acid (ALA) from baseline [ Time Frame: Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 168 days post-dose ]
The change in Porphobilinogen (PBG) from baseline [ Time Frame: Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 168 days post-dose ]

Original Secondary Outcome Measures ^ICMJE

Profile of Pharmacokinetics (PK) of ALN-AS1 [ Time Frame: Part A (SAD) phase: predose - 42 days post-dose; Part B (MAD) phase: predose - 70 days post-dose; Part C (MD) phase: predose - 126 days post-dose ]
Cmax, tmax, AUC, t1/2
The change in delta-aminolevulinic acid (ALA) and Porphobilinogen (PBG) from baseline [ Time Frame: Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 126 days post-dose ]

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

A Phase 1 Study of ALN-AS1 in Patients With Acute Intermittent Porphyria (AIP)

Official Title ^ICMJE

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of ALN-AS1 in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 1

Study Design ^ICMJE

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant
Primary Purpose: Treatment

Condition ^ICMJE

Acute Intermittent Porphyria

Intervention ^ICMJE

Drug: ALN-AS1
Single or multiple doses of ALN-AS1 by subcutaneous (sc) injection
Drug: Sterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator

Study Arms

Active Comparator: ALN-AS1
Intervention: Drug: ALN-AS1
Placebo Comparator: Sterile Normal Saline (0.9% NaCl)
Intervention: Drug: Sterile Normal Saline (0.9% NaCl)

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

August 2017

Primary Completion Date

May 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Parts A and B

Inclusion Criteria:

Diagnosis of AIP
Urine PBG at Screening indicating patient is a high excreter
No clinically significant health concerns
Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
Willing to provide written informed consent and willing to comply with study requirements.

Exclusion Criteria:

Porphyria attack within 6 months of screening
Started a new prescription medication within 3 months of screening
Clinically significant abnormal laboratory results
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

Part C

Inclusion Criteria:

Diagnosis of AIP
Patient experienced a porphyria attack or was taking medication to prevent attacks recently
No clinically significant health concerns
Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
Willing to provide written informed consent and willing to comply with study requirements.

Exclusion Criteria:

Stared a new prescription medication within 3 months of screening
Clinically significant abnormal laboratory results
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

Sex/Gender

Sexes Eligible for Study:

All

Ages

18 Years to 65 Years (Adult)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Alnylam Clinical Trials Hotline Call for Complete Site List	617-575-7400
Contact: Alnylam Clinical Trials Hotline Call for Complete Site List	1-866-330-0326

Listed Location Countries ^ICMJE

Sweden, United Kingdom, United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT02452372

Other Study ID Numbers ^ICMJE

ALN-AS1-001

Has Data Monitoring Committee

U.S. FDA-regulated Product

Not Provided

IPD Sharing Statement

Not Provided

Responsible Party

Alnylam Pharmaceuticals

Study Sponsor ^ICMJE

Alnylam Pharmaceuticals

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Amy Simon, MD

Alnylam Pharmaceuticals

PRS Account

Alnylam Pharmaceuticals

Verification Date

June 2017

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

To Top