A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
Completed · Phase 1
Conditions studied: Acute Intermittent Porphyria
In brief
The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.
Key facts
- Study ID
- NCT02452372
- Run by
- Alnylam Pharmaceuticals
- People needed
- 40
- Starts
- 2015-05-06
- Expected to finish
- 2017-09-06
- Last updated by the study team
- 2018-06-14
Who can join
Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- Clinical Trial Site — Birmingham, Alabama, United States
- Clinical Trial Site — San Francisco, California, United States
- Clinical Trial Site — New York, New York, United States
- Clinical Trial Site — Galveston, Texas, United States
- Clinical Trial Site — Stockholm, Sweden
- Clinical Trial Site — London, United Kingdom
Full record on ClinicalTrials.gov
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