ong-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder.

Phase 1

• Conditions: Acute Intermittent Porphyria (AIP); MedDRA version: 20.0Level: LLTClassification code 10000818Term: Acute intermittent porphyriaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2016-002638-54-GB
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-08-18
• Study Completion: 2021-11-05
• Last Update Posted: 19 February 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

1. Completed and, in the opinion of the Investigator, tolerated study drug dosing in Part C of study ALN-AS1-001
2. Not on a scheduled regimen of hemin to prevent porphyria attacks at Screening
3. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test, cannot be breast feeding, and must be willing to use 1 highly effective method of contraception 14 days before first dose, throughout study participation, and for 90 days after last dose administration
4. Willing and able to comply with the study requirements and to provide written informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 23
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Alanine transaminase =2.0×ULN or total bilirubin =2 mg/dL (unless bilirubin elevation is due to Gilbert’s syndrome)
2. Estimated glomerular filtration rate =30 mL/min/1.73 m2 (using the Modification of Diet in Renal Disease formula)
3. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or to N-acetyl galactosamine ligand
4. Received an investigational agent, other than ALN-AS1, or who are in follow-up of another clinical study of an investigational agent within 90 days before study drug administration
5. Other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety and tolerability of ALN-AS1 in patients with AIP.;Secondary Objective: • Assess the PD effect of ALN-AS1 over time<br>• Assess the clinical activity of ALN-AS1 over time;Primary end point(s): • Patient incidence of adverse events;Timepoint(s) of evaluation of this end point: The duration of treatment is up to 48 months. The estimated total time on study, inclusive of Screening/Baseline, for each patient is up to 56 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Change in urine ALA and PBG levels<br>• Frequency and characteristics of porphyria attacks<br>• Change in hemin administration.;Timepoint(s) of evaluation of this end point: The duration of treatment is up to 48 months. The estimated total time on study, inclusive of Screening/Baseline, for each patient is up to 56 months