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RNAi Therapeutics

givosiran (ALN-AS1) for Acute Intermittent Porphyria

Phase 1
Waitlist Available
Research Sponsored by Alnylam Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 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
Awards & highlights
All Individual Drugs Already Approved

Summary

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.

Eligible Conditions
  • Acute Intermittent Porphyria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~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
This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Profile of Pharmacokinetics (PK) of givosiran

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: givosiran (ALN-AS1)Active Control1 Intervention
Group II: Sterile Normal Saline (0.9% NaCl)Placebo Group1 Intervention

Find a Location

Who is running the clinical trial?

Alnylam PharmaceuticalsLead Sponsor
76 Previous Clinical Trials
15,226 Total Patients Enrolled
Amy Simon, MDStudy DirectorAlnylam Pharmaceuticals
1 Previous Clinical Trials
32 Total Patients Enrolled
~4 spots leftby Oct 2025
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