Acute Hepatic PorphyriaNCT02240784
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
An observational study of Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals at 30 sites in 20 countries.
Trial summary
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Acute Hepatic Porphyria
- How many people
- 136 participants
- Where
- 30 sitesin 20 countries
- Study dates
- Aug 2014to 26 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Acute Hepatic Porphyria
Who may be able to join
- 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
Who cannot join
- Current participation in a clinical trial of an investigational product
Trial locations and contacts
30 sites in 20 countries.
30 sites in 20 countries
United States8 sites
Clinical Trial Site
Ann Arbor, Michigan, 48109
Clinical Trial Site
Galveston, Texas, 77555
Clinical Trial Site
Miami, Florida, 33136
Clinical Trial Site
New York, New York, 10029
Clinical Trial Site
Philadelphia, Pennsylvania, 19107
Clinical Trial Site
Salt Lake City, Utah, 84112
Clinical Trial Site
San Francisco, California, 94143
Clinical Trial Site
Winston-Salem, North Carolina, 27157
United Kingdom2 sites
Clinical Trial Site
Cardiff, Wales
Clinical Trial Site
London
About this study
Official title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Conditions
- Acute Hepatic Porphyria
- Keywords
- Porphyria, AIP, VP, HCP, Variegate, Coproporphyria
- Medical subject headings
- Coproporphyria, Hereditary, Porphyrias, Coproporphyria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Acute Hepatic Porphyria | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Medical history of AHP patients (Part A only)
Measured: Baseline to 6-Month Follow-Up Visit
Medication usage of AHP patients (Part A only)
Measured: Baseline to 6-Month Follow-Up Visit
Secondary outcomes (9)
Plasma biomarkers (Part A only)
Measured: 0, 6 months and During Attacks
Porphyria signs and symptoms (Part A only)
Measured: 0, 2, 4, 6 months
Quality of Life (Part A only)
Measured: 0, 6 months
Urine biomarkers (Part A only)
Measured: 0, 6 months and During Attacks
Healthcare Utilization (Part A only)
Measured: 0, 6 months
Change in disease activity as measured by Quality of Life (Part B only)
Measured: 0, 6, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)
Measured: 0, 3, 6, 9, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Brief Pain Inventory form (Part B only)
Measured: 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)
Measured: 0, 6, 12, 18, 24, 30, 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2014 (estimated)
- Study startAug 2014
- Primary completion26 Apr 2021
- Study completion26 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hepatic PorphyriaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 136 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02240784
- Sponsor's protocol ID
- ALN-AS1-NT-001
- Status last verified
- May 2021
- First submitted
- 11 Sep 2014
- Last updated
- 17 May 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
- ClinicalTrials.gov ID
- NCT02240784
- Acronym / sponsor ID
- ALN-AS1-NT-001
- Conditions
- Acute Hepatic Porphyria
- Keywords
- Porphyria, AIP, VP, HCP, Variegate, Coproporphyria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 136 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Aug 2014 (actual)
- Primary completion
- 26 Apr 2021 (actual)
- Study completion
- 26 Apr 2021 (actual)
- First posted
- 16 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Acute Hepatic Porphyria | – | – |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Acute Hepatic Porphyria
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- Current participation in a clinical trial of an investigational product
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
9 secondary outcomes
Plasma biomarkers (Part A only)
Time frame: 0, 6 months and During Attacks
Porphyria signs and symptoms (Part A only)
Time frame: 0, 2, 4, 6 months
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
Change in disease activity as measured by Quality of Life (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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2021.
- Individual participant dataSharing planned: No
Related trials for Acute Hepatic Porphyria
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Identification of Acute Intermittent Porphyria Modifying Genes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Alnylam Pharmaceuticals
30 sites in 20 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals at 30 sites in 20 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Italy, Spain, United Kingdom, Australia and 15 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Medical history of AHP patients (Part A only) (time frame: Baseline to 6-Month Follow-Up Visit). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 May 2021.