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

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ClinicalTrials.gov Identifier: NCT02240784
Recruitment Status : Recruiting
First Posted : September 16, 2014
Last Update Posted : December 20, 2017
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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 Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
Study Start Date : August 2014
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020


Group/Cohort
Acute Hepatic Porphyria



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.


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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 > 18 years
  • Diagnosis of AHP [acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP)]
  • 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


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


Contacts
Contact: Alnylam Clinical Trials Hotline Call for Complete Site List 617-575-7400
Contact: Alnylam Clinical Trials Hotline Call for Complete Site List 1-866-330-0326

  Show 19 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Amy Simon, MD Alnylam Pharmaceuticals

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02240784     History of Changes
Other Study ID Numbers: ALN-AS1-NT-001
First Posted: September 16, 2014    Key Record Dates
Last Update Posted: December 20, 2017
Last Verified: December 2017

Keywords provided by Alnylam Pharmaceuticals:
Porphyria
AIP
VP
HCP
Variegate
Coproporphyria

Additional relevant MeSH terms:
Porphyrias
Porphyria, Erythropoietic
Porphyrias, Hepatic
Coproporphyria, Hereditary
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases