Acute Intermittent Porphyria (AIP) MedDRA version: 20.0 Level: LLT Classification code 10000818 Term: Acute intermittent porphyria System Organ Class: 100000004850
Conditions
Sponsors
Alnylam Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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 acceptable methods 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 • 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
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in urine ALA and PBG levels • Frequency and characteristics of porphyria attacks • 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 | — |
Countries
Sweden, United Kingdom, United States
Contacts
Public ContactClinical Trials Hotline
Alnylam Pharmaceuticals Inc
Outcome results
None listed