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EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02240784
Recruitment Status : Completed
First Posted : September 16, 2014
Last Update Posted : May 17, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.

Condition or disease
Acute Hepatic Porphyria

Study Design
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Study Type : Observational
Actual Enrollment : 136 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
Actual Study Start Date : August 2014
Actual Primary Completion Date : April 26, 2021
Actual Study Completion Date : April 26, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Porphyria

Groups and Cohorts
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Group/Cohort
Acute Hepatic Porphyria


Outcome Measures
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Primary Outcome Measures :
  1. Medical history of AHP patients (Part A only) [ Time Frame: Baseline to 6-Month Follow-Up Visit ]
  2. Medication usage of AHP patients (Part A only) [ Time Frame: Baseline to 6-Month Follow-Up Visit ]

Secondary Outcome Measures :
  1. Plasma biomarkers (Part A only) [ Time Frame: 0, 6 months and During Attacks ]
  2. Porphyria signs and symptoms (Part A only) [ Time Frame: 0, 2, 4, 6 months ]
  3. Quality of Life (Part A only) [ Time Frame: 0, 6 months ]
  4. Urine biomarkers (Part A only) [ Time Frame: 0, 6 months and During Attacks ]
  5. Healthcare Utilization (Part A only) [ Time Frame: 0, 6 months ]
  6. Change in disease activity as measured by Quality of Life (Part B only) [ Time Frame: 0, 6, 12, 18, 24, 30, 36 months ]
  7. 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 ]
  8. 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 ]
  9. 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 ]

Biospecimen Retention:   Samples Without DNA
Urine and plasma samples for known proteins associated with porphyria attacks.

Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Acute Hepatic Porphyria
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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02240784


Locations
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United States, California
Clinical Trial Site
San Francisco, California, United States, 94143
United States, Florida
Clinical Trial Site
Miami, Florida, United States, 33136
United States, Michigan
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
United States, New York
Clinical Trial Site
New York, New York, United States, 10029
United States, North Carolina
Clinical Trial Site
Winston-Salem, North Carolina, United States, 27157
United States, Pennsylvania
Clinical Trial Site
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Clinical Trial Site
Galveston, Texas, United States, 77555
United States, Utah
Clinical Trial Site
Salt Lake City, Utah, United States, 84112
Australia, New South Wales
Clinical Trial Site
Camperdown, New South Wales, Australia
Belgium
Clinical Trial Site
Leuven, Belgium
Bulgaria
Clinical Trial Site
Sofia, Bulgaria
Czechia
Clinical Trial Site
Prague, Czechia
Finland
Clinical Trial Site
Helsinki, Finland
France
Clinical Trial Site
Paris, France
Germany
Clinical Trial Site
Chemnitz, Germany
Israel
Clinical Trial Site
Petach Tikva, Israel
Italy
Clinical Trial Site
Milano, Italy
Clinical Trial Site
Modena, Italy
Korea, Republic of
Clinical Trial Site
Seoul, Korea, Republic of
Mexico
Clinical Trial Site
Mexico City, Mexico
Netherlands
Clinical Trial Site
Rotterdam, Netherlands
Norway
Clinical Trial Site
Bergen, Norway
Poland
Clinical Trial Site
Warsaw, Poland
South Africa
Clinical Trial Site
Cape Town, South Africa
Spain
Clinical Trial Site
Barcelona, Spain
Clinical Trial Site
Murcia, Spain
Switzerland
Clinical Trial Site
Zurich, Switzerland
Taiwan
Clinical Trial Site
Taipei City, Taiwan
United Kingdom
Clinical Trial Site
Cardiff, Wales, United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02240784    
Other Study ID Numbers: ALN-AS1-NT-001
First Posted: September 16, 2014    Key Record Dates
Last Update Posted: May 17, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Alnylam Pharmaceuticals:
Porphyria
AIP
VP
 HCP
Variegate
Coproporphyria
Additional relevant MeSH terms:
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Porphyrias, Hepatic
Coproporphyria, Hereditary
Porphyria, Erythropoietic
Porphyrias
Metabolic Diseases
 Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases