Acute Hepatic Porphyria Clinical Trial
Official title:
A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
| NCT number | NCT02240784 |
| Other study ID # | ALN-AS1-NT-001 |
| Secondary ID | |
| Status | Completed |
| Phase | |
| First received | |
| Last updated | |
| Start date | August 2014 |
| Est. completion date | April 26, 2021 |
| Verified date | May 2021 |
| Source | Alnylam Pharmaceuticals |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
| Status | Completed |
| Enrollment | 136 |
| Est. completion date | April 26, 2021 |
| Est. primary completion date | April 26, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Australia | Clinical Trial Site | Camperdown | New South Wales |
| Belgium | Clinical Trial Site | Leuven | |
| Bulgaria | Clinical Trial Site | Sofia | |
| Czechia | Clinical Trial Site | Prague | |
| Finland | Clinical Trial Site | Helsinki | |
| France | Clinical Trial Site | Paris | |
| Germany | Clinical Trial Site | Chemnitz | |
| Israel | Clinical Trial Site | Petach Tikva | |
| Italy | Clinical Trial Site | Milano | |
| Italy | Clinical Trial Site | Modena | |
| Korea, Republic of | Clinical Trial Site | Seoul | |
| Mexico | Clinical Trial Site | Mexico City | |
| Netherlands | Clinical Trial Site | Rotterdam | |
| Norway | Clinical Trial Site | Bergen | |
| Poland | Clinical Trial Site | Warsaw | |
| South Africa | Clinical Trial Site | Cape Town | |
| Spain | Clinical Trial Site | Barcelona | |
| Spain | Clinical Trial Site | Murcia | |
| Switzerland | Clinical Trial Site | Zurich | |
| Taiwan | Clinical Trial Site | Taipei City | |
| United Kingdom | Clinical Trial Site | Cardiff | Wales |
| United Kingdom | Clinical Trial Site | London | |
| United States | Clinical Trial Site | Ann Arbor | Michigan |
| United States | Clinical Trial Site | Galveston | Texas |
| United States | Clinical Trial Site | Miami | Florida |
| United States | Clinical Trial Site | New York | New York |
| United States | Clinical Trial Site | Philadelphia | Pennsylvania |
| United States | Clinical Trial Site | Salt Lake City | Utah |
| United States | Clinical Trial Site | San Francisco | California |
| United States | Clinical Trial Site | Winston-Salem | North Carolina |
| Lead Sponsor | Collaborator |
|---|---|
| Alnylam Pharmaceuticals |
United States, Australia, Belgium, Bulgaria, Czechia, Finland, France, Germany, Israel, Italy, Korea, Republic of, Mexico, Netherlands, Norway, Poland, South Africa, Spain, Switzerland, Taiwan, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Medical history of AHP patients (Part A only) | Baseline to 6-Month Follow-Up Visit | ||
| Primary | Medication usage of AHP patients (Part A only) | Baseline to 6-Month Follow-Up Visit | ||
| Secondary | Plasma biomarkers (Part A only) | 0, 6 months and During Attacks | ||
| Secondary | Porphyria signs and symptoms (Part A only) | 0, 2, 4, 6 months | ||
| Secondary | Quality of Life (Part A only) | 0, 6 months | ||
| Secondary | Urine biomarkers (Part A only) | 0, 6 months and During Attacks | ||
| Secondary | Healthcare Utilization (Part A only) | 0, 6 months | ||
| Secondary | Change in disease activity as measured by Quality of Life (Part B only) | 0, 6, 12, 18, 24, 30, 36 months | ||
| Secondary | Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only) | 0, 3, 6, 9, 12, 18, 24, 30, 36 months | ||
| Secondary | Change in disease activity as measured by Brief Pain Inventory form (Part B only) | 0, 3, 6, 9, 12, 18, 24, 30, 36 months | ||
| Secondary | Change in disease activity as measured by European Organization for Research and Treatment of Cancer (EORTC) Questionnaire (Part B only) | 0, 6, 12, 18, 24, 30, 36 months |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03338816 -
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
|
Phase 3 | |
| Terminated |
NCT03547297 -
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
|
||
| Not yet recruiting |
NCT04923516 -
Prevalence of Acute Hepatic Porphyria
|
||
| Recruiting |
NCT05344599 -
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
|
||
| Recruiting |
NCT04883905 -
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
|
||
| Approved for marketing |
NCT04056481 -
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
|