We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study is currently recruiting participants.
Verified June 2017 by Alnylam Pharmaceuticals
Sponsor:
ClinicalTrials.gov Identifier:
NCT02240784
First Posted: September 16, 2014
Last Update Posted: June 5, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
  Purpose
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.

Condition
Acute Hepatic Porphyria

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • 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 Measures:
  • 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 ]

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

Estimated Enrollment: 300
Study Start Date: August 2014
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Groups/Cohorts
Acute Hepatic Porphyria

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

  Contacts and Locations
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 20 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Amy Simon, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02240784     History of Changes
Other Study ID Numbers: ALN-AS1-NT-001
First Submitted: September 11, 2014
First Posted: September 16, 2014
Last Update Posted: June 5, 2017
Last Verified: June 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