--- title: A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) nct_id: NCT02452372 overall_status: COMPLETED phase: PHASE1 sponsor: Alnylam Pharmaceuticals study_type: INTERVENTIONAL primary_condition: Acute Intermittent Porphyria countries: United States, Sweden, United Kingdom canonical_url: "https://parkinsonspathways.com/agent/trials/NCT02452372.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT02452372" ct_last_update_post_date: 2018-06-14 last_seen_at: "2026-05-12T07:12:48.885Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) **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) **NCT ID:** [NCT02452372](https://clinicaltrials.gov/study/NCT02452372) ## Key Facts - **Status:** COMPLETED - **Phase:** PHASE1 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 40 - **Lead Sponsor:** Alnylam Pharmaceuticals - **Conditions:** Acute Intermittent Porphyria - **Start Date:** 2015-05-06 - **Completion Date:** 2017-09-06 - **CT.gov Last Update:** 2018-06-14 ## Brief 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. ## Eligibility - **Minimum age:** 18 Years - **Maximum age:** 65 Years - **Sex:** ALL - **Healthy Volunteers:** No ``` 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 ``` ## Arms - **givosiran (ALN-AS1)** (ACTIVE_COMPARATOR) - **Sterile Normal Saline (0.9% NaCl)** (PLACEBO_COMPARATOR) ## Interventions - **givosiran (ALN-AS1)** (DRUG) — Single or multiple doses of ALN-AS1 by subcutaneous (sc) injection - **Sterile Normal Saline (0.9% NaCl)** (DRUG) — calculated volume to match active comparator ## Primary Outcomes - **The safety of givosiran 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)_ ## Secondary Outcomes - **Profile of Pharmacokinetics (PK) of givosiran** _(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)_ - **Profile of Pharmacokinetics (PK) of givosiran** _(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)_ - **Profile of Pharmacokinetics (PK) of givosiran** _(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)_ - **Profile of Pharmacokinetics (PK) of givosiran** _(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)_ - **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)_ ## Locations (6) - 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 ## Recent Field Changes (last 30 days) - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.maxAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.clinical trial site|birmingham|alabama|united states` — added _(2026-05-12)_ - `locations.clinical trial site|san francisco|california|united states` — added _(2026-05-12)_ - `locations.clinical trial site|new york|new york|united states` — added _(2026-05-12)_ - `locations.clinical trial site|galveston|texas|united states` — added _(2026-05-12)_ - `locations.clinical trial site|stockholm||sweden` — added _(2026-05-12)_ - `locations.clinical trial site|london||united kingdom` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT02452372.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT02452372* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*