A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

Acute Intermittent Porphyria Clinical Trial

Official title:

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02949830
• Other study ID #: ALN-AS1-002
• Secondary ID: 2016-002638-54
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: October 2016
• Est. completion date: November 5, 2021

Study information

• Verified date: March 2024
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: November 5, 2021
• Est. primary completion date: November 5, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Acute Intermittent Porphyria
• Porphyria, Acute Intermittent
• Porphyria, Erythropoietic
• Porphyrias

Intervention

Drug:
• Givosiran
Givosiran by subcutaneous (SC) injection.

Locations

Country	Name	City	State
Sweden	Clinical Trial Site	Stockholm
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Galveston	Texas
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline	The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels.	Baseline; Month 48
Secondary	The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline	The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels.	Baseline; Month 48
Secondary	Annualized Rate of Composite Porphyria Attacks	Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. Composite porphyria attacks included porphyria attacks that required hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home. The annualized attack rate (AAR) was calculated as the number of composite porphyria attacks/total person-years.	Through Month 48
Secondary	Annualized Rate of Hemin Administration	The annualized rate of hemin administration was evaluated by annualized days of hemin use, which is calculated as the number of doses of hemin administered/total person-years.	Through Month 49