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Trial record 3 of 8 for:    patisiran

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

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ClinicalTrials.gov Identifier: NCT02939820
Expanded Access Status : Available
First Posted : October 20, 2016
Last Update Posted : June 6, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Condition or disease Intervention/treatment
TTR-mediated Amyloidosis Amyloidosis, Hereditary Amyloid Neuropathies, Familial Familial Amyloid Polyneuropathies Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related Drug: patisiran (ALN-TTR02)

Detailed Description:
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.

Study Type : Expanded Access
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy



Intervention Details:
  • Drug: patisiran (ALN-TTR02)
    patisiran (ALN-TTR02) administered by intravenous (IV) infusion

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, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • Male or female greater than or equal to 18 years of age
  • Have a diagnosis of hATTR
  • Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
  • Have adequate complete blood counts, liver function tests and coagulation tests

Exclusion Criteria:

  • Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
  • Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
  • Have inadequate cardiac function
  • Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
  • Have known serious comorbidities or considered unfit for the program by the investigator
  • Prior or planned liver or heart transplantation

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): NCT02939820


Contacts
Contact: Alnylam EAP Hotline (617) 715-0200 EAP@alnylam.com

  Show 21 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals

Additional Information:
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02939820     History of Changes
Other Study ID Numbers: ALN-TTR02-007
First Posted: October 20, 2016    Key Record Dates
Last Update Posted: June 6, 2018
Last Verified: June 2018

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
FAP
Familial Amyloid Polyneuropathies
TTR
Transthyretin
Amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis, Familial
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors