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Eligibility
for people ages 18–65
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:
Principal Investigator

Description

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.

Official Title

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)

Keywords

Acute Intermittent Porphyria RNAi therapeutic Porphyria AIP

Eligibility

For people ages 18–65

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

Locations

  • Clinical Trial Site accepting new patients
    San Francisco, California, United States
  • Clinical Trial Site accepting new patients
    Galveston, Texas, United States
  • Clinical Trial Site accepting new patients
    Birmingham, Alabama, United States
  • Clinical Trial Site accepting new patients
    New York, New York, United States
  • Clinical Trial Site accepting new patients
    London, United Kingdom
  • Clinical Trial Site accepting new patients
    Stockholm, Sweden

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alnylam Pharmaceuticals
ID
NCT02452372
Phase
Phase 1
Lead Scientist
Dwight Bissell
Study Type
Interventional
Last Updated
June 1, 2017
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