A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
Status and phase
Completed
Phase 1
Conditions
Acute Intermittent Porphyria
Treatments
Drug: Sterile Normal Saline (0.9% NaCl)
Drug: givosiran (ALN-AS1)
Details and patient eligibility
About
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.
Enrollment
40 patients
Sex
All
Ages
18 to 65 years old
Volunteers
No Healthy Volunteers
Inclusion and exclusion criteria
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
Trial design
Primary purpose
Treatment
Allocation
Randomized
Interventional model
Parallel Assignment
Masking
Single Blind
40 participants in 2 patient groups, including a placebo group
givosiran (ALN-AS1)
Active Comparator group
Treatment:
Drug: givosiran (ALN-AS1)
Sterile Normal Saline (0.9% NaCl)
Placebo Comparator group
Treatment:
Drug: Sterile Normal Saline (0.9% NaCl)
Trial contacts and locations
6
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Data sourced from clinicaltrials.gov
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