|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
24 May 2021 |
|
Main ID: |
NCT02240784 |
|
Date of registration:
|
11/09/2014 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
|
|
Scientific title:
|
A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks |
|
Date of first enrolment:
|
August 2014 |
|
Target sample size:
|
136 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT02240784 |
|
Study type:
|
Observational |
|
Study design:
|
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
Australia
|
Belgium
|
Bulgaria
|
Czechia
|
Finland
|
France
|
Germany
|
Israel
|
|
Italy
|
Korea, Republic of
|
Mexico
|
Netherlands
|
Norway
|
Poland
|
South Africa
|
Spain
|
|
Switzerland
|
Taiwan
|
United Kingdom
|
United States
| | | | |
|
Contacts
|
|
Name:
|
Medical Director |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Alnylam Pharmaceuticals |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Males and females aged >/= 12 years
- Diagnosis of AHP [acute intermittent porphyria (AIP), variegate porphyria (VP),
hereditary coproporphyria (HCP) and aminolevulinic acid dehydratase (ALAD) deficient
porphyria (ADP)]
- Porphyria attacks in the past 12 months or receiving treatment to prevent attacks
- Willing to provide written informed consent, medical records, and to comply with study
requirements
Exclusion Criteria:
• Current participation in a clinical trial of an investigational product
Age minimum:
12 Years
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Acute Hepatic Porphyria
|
|
Primary Outcome(s)
|
|
Medical history of AHP patients (Part A only)
[Time Frame: Baseline to 6-Month Follow-Up Visit]
|
|
Medication usage of AHP patients (Part A only)
[Time Frame: Baseline to 6-Month Follow-Up Visit]
|
|
Secondary Outcome(s)
|
|
Change in disease activity as measured by Quality of Life (Part B only)
[Time Frame: 0, 6, 12, 18, 24, 30, 36 months]
|
|
Porphyria signs and symptoms (Part A only)
[Time Frame: 0, 2, 4, 6 months]
|
|
Change in disease activity as measured by Brief Pain Inventory form (Part B only)
[Time Frame: 0, 3, 6, 9, 12, 18, 24, 30, 36 months]
|
|
Change in disease activity as measured by European Organization for Research and Treatment of Cancer (EORTC) Questionnaire (Part B only)
[Time Frame: 0, 6, 12, 18, 24, 30, 36 months]
|
|
Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)
[Time Frame: 0, 3, 6, 9, 12, 18, 24, 30, 36 months]
|
|
Plasma biomarkers (Part A only)
[Time Frame: 0, 6 months and During Attacks]
|
|
Quality of Life (Part A only)
[Time Frame: 0, 6 months]
|
|
Urine biomarkers (Part A only)
[Time Frame: 0, 6 months and During Attacks]
|
|
Healthcare Utilization (Part A only)
[Time Frame: 0, 6 months]
|
|
Secondary ID(s)
|
|
ALN-AS1-NT-001
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|