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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04056481




Registration number
NCT04056481
Ethics application status
Date submitted
12/08/2019
Date registered
14/08/2019
Date last updated
13/11/2020

Titles & IDs
Public title
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
Scientific title
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
Secondary ID [1] 0 0
ALN-AS1-005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Hepatic Porphyria 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Expanded Access
Description of intervention(s) / exposure
Treatment: Drugs - Givosiran

Treatment: Drugs: Givosiran
givosiran (ALN-AS1) administered as a subcutaneous (SC) injection

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary
Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)

- Have adequate venous access for program sample collections as judged by the
Investigator for study sample collections
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Previously or currently participating in a givosiran clinical trial

- Has any of the following laboratory parameter assessments at Screening:

1. Alanine aminotransferase (ALT) >2×ULN

2. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is
secondary to documented Gilbert's syndrome are eligible if the total bilirubin is
<2×ULN

3. Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2

- On an active liver transplantation waiting list

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Available
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Program Site - Melbourne
Recruitment hospital [2] 0 0
Program Site - Richmond
Recruitment hospital [3] 0 0
Program Site - Wyong
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- Richmond
Recruitment postcode(s) [3] 0 0
- Wyong
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Belgium
State/province [2] 0 0
Leuven
Country [3] 0 0
Belgium
State/province [3] 0 0
Liège
Country [4] 0 0
Luxembourg
State/province [4] 0 0
Luxembourg City
Country [5] 0 0
Netherlands
State/province [5] 0 0
Rotterdam
Country [6] 0 0
Spain
State/province [6] 0 0
Barcelona
Country [7] 0 0
Spain
State/province [7] 0 0
Cieza
Country [8] 0 0
Spain
State/province [8] 0 0
Madrid
Country [9] 0 0
Spain
State/province [9] 0 0
Palma De Mallorca
Country [10] 0 0
Spain
State/province [10] 0 0
Pamplona
Country [11] 0 0
Spain
State/province [11] 0 0
Sabadell
Country [12] 0 0
Spain
State/province [12] 0 0
Sevilla
Country [13] 0 0
Sweden
State/province [13] 0 0
Stockholm

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to provide expanded access of givosiran to patients with Acute
Hepatic Porphyria (AHP).
Trial website
https://clinicaltrials.gov/show/NCT04056481
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alnylam EAP Hotline
Address 0 0
Country 0 0
Phone 0 0
1-877-256-9526
Fax 0 0
Email 0 0
eap@alnylam.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04056481