NCT02949830

Brief Summary

The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2016

Longer than P75 for phase_1

Geographic Reach
3 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2016

Completed
27 days until next milestone

First Submitted

Initial submission to the registry

October 28, 2016

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 31, 2016

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 5, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 5, 2021

Completed
2.4 years until next milestone

Results Posted

Study results publicly available

March 12, 2024

Completed
Last Updated

March 12, 2024

Status Verified

March 1, 2024

Enrollment Period

5.1 years

First QC Date

October 28, 2016

Results QC Date

November 5, 2022

Last Update Submit

March 11, 2024

Conditions

Acute Intermittent Porphyria

Keywords

RNAi therapeuticPorphyriaAIPHeme/Haem Arginate

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Adverse Events (AEs)

    An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

    Through Month 49

Secondary Outcomes (4)

  • The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline

    Baseline; Month 48

  • The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline

    Baseline; Month 48

  • Annualized Rate of Composite Porphyria Attacks

    Through Month 48

  • Annualized Rate of Hemin Administration

    Through Month 49

Study Arms (1)

Givosiran

EXPERIMENTAL

At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly(QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM.

Drug: Givosiran

Interventions

GivosiranDRUG

Givosiran by subcutaneous (SC) injection.

Also known as: ALN-AS1, Givlaari
Givosiran

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Completed participation in Part C of study ALN-AS1-001 (NCT02452372)
  • Not on a scheduled regimen of hemin
  • Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception
  • Willing and able to comply with the study requirements and to provide written informed consent

You may not qualify if:

  • Clinically significant abnormal laboratory results
  • Received an investigational agent (other than ALN-AS1) 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Clinical Trial Site

San Francisco, California, United States

Location

Clinical Trial Site

New York, New York, United States

Location

Clinical Trial Site

Galveston, Texas, United States

Location

Clinical Trial Site

Stockholm, Sweden

Location

Clinical Trial Site

London, United Kingdom

Location

MeSH Terms

Conditions

Porphyria, Acute IntermittentPorphyrias

Interventions

givosiran

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Chief Medical Officer
Organization
Alnylam Pharmaceuticals Inc.
clinicaltrials@alnylam.com
866-330-0326

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 28, 2016

First Posted

October 31, 2016

Study Start

October 1, 2016

Primary Completion

November 5, 2021

Study Completion

November 5, 2021

Last Updated

March 12, 2024

Results First Posted

March 12, 2024

Record last verified: 2024-03

Locations

GB(1)SE(1)US(3)