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| Sponsor: | Boston University |
|---|---|
| Collaborators: |
Food and Drug Administration (FDA) National Institute of Neurological Disorders and Stroke (NINDS) |
| Information provided by: | Boston University |
| ClinicalTrials.gov Identifier: | NCT00294671 |
Purpose
The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy.
| Condition | Intervention | Phase |
|---|---|---|
|
Familial Amyloid Polyneuropathy Familial Amyloidosis |
Drug: diflunisal Other: placebo |
Phase II Phase III |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
| Official Title: | The Effect of Diflunisal on Familial Amyloidosis |
| Estimated Enrollment: | 200 |
| Study Start Date: | February 2006 |
| Estimated Study Completion Date: | August 2010 |
| Estimated Primary Completion Date: | May 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Active Comparator
diflunisal
|
Drug: diflunisal
given twice daily for 24 months
|
|
2: Placebo Comparator
placebo
|
Other: placebo
an inactive substance given twice daily for 24 months
|
Familial amyloidosis polyneuropathy (FAP) is a rare, lethal, autosomal dominant, neurodegenerative disease characterized by misfolding of variant transthyretin tetramer (TTR) — a transport protein produced by the liver. The disease causes TTR to become unstable, triggering amyloid fibrils to form and leading to peripheral and autonomic nerve dysfunction.
Currently, the only treatment for FAP is a liver transplant, which is expensive and risk-filled. Medicines are needed to treat this disease. Previous in vitro (in a test tube) studies have shown that a common anti-inflammatory drug called diflunisal stabilizes TTR, preventing the formation of amyloid fibrils.
The goal of this 2-year randomized, double-blind, placebo-controlled research study is to establish whether diflunisal can stop the nerve damage, or peripheral neuropathy, resulting from amyloid production in patients with FAP. Scientists already know that diflunisal prevents formation of amyloid in the test tube. This study will determine if the drug can block amyloid production in FAP patients.
Participants will be randomly chosen to receive either diflunisal or an inactive (placebo) pill twice daily for 24 months. Participants will be carefully monitored through 7 follow-up visits, either at the study center or with individual primary care physicians. Participating in the study does not preclude patients from being listed for liver transplantation.
Eligibility| Ages Eligible for Study: | 18 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Melissa Rosenberg | 617-638-4494 | merose@bu.edu |
| United States, California | |
| The Scripps Research Institute | Not yet recruiting |
| La Jolla, California, United States, 92035 | |
| United States, Massachusetts | |
| Amyloid Treatment and Research Program, Boston Medical Center | Recruiting |
| Boston, Massachusetts, United States, 02118 | |
| Contact: Melissa Rosenberg 617-638-4494 merose@bu.edu | |
| United States, Minnesota | |
| Mayo Clinic Rochester | Recruiting |
| Rochester, Minnesota, United States, 55905 | |
| Contact: Steven Zeldenrust, MD, PhD 507-284-2865 zeldenrust.steven@mayo.edu | |
| United States, New York | |
| Mount Sinai School of Medicine, Department of Medicine | Recruiting |
| New York, New York, United States, 10029-6574 | |
| Contact: Susan Comninel, BA 212-241-0059 | |
| Italy | |
| IRCCS Policlinico San Matteo | Recruiting |
| Pavia, Italy, 27100 | |
| Contact: Laura Obici, MD 39-0382-502-983 l.obici@smatteo.pv.it | |
| Japan | |
| Kumamoto University | Recruiting |
| Kumamoto, Japan, 860-0811 | |
| Contact: Taro Yamashita, MD, PhD 096-373-5893 taro-yamashita@fc.kuh.kumamoto-u.ac.jp | |
| Shinshu University | Recruiting |
| Matsumoto, Japan, 390-8621 | |
| Contact: Yoshi Sekijima, MD 81-263-37-2673 sekijima@hsp.md.shinshu-u.ac.jp | |
| Portugal | |
| Hospital Santo Antonio | Not yet recruiting |
| Porto, Portugal, 4099-001 | |
| Contact: Teresa Coelho, MD 351-91-8840370 tcoelho@netcabo.pt | |
| Sweden | |
| Umea University Hospital | Recruiting |
| Umea, Sweden, SE-901 86 | |
| Contact: Hans Eric, MD 46 90 785 1415 HansErik.Lundgren@vll.se | |
| Principal Investigator: | John L. Berk, MD | Boston University |
More Information
| Responsible Party: | BOSTON UNIVERSITY MEDICAL CENTER ( JOHN BERK, MD, PRINCIPAL INVESTIGATOR ) |
| Study ID Numbers: | R01NS051306-01, FD-R-002532-01 |
| Study First Received: | February 21, 2006 |
| Last Updated: | July 30, 2009 |
| ClinicalTrials.gov Identifier: | NCT00294671 History of Changes |
| Health Authority: | United States: Food and Drug Administration |
|
familial amyloid polyneuropathy familial amyloidosis diflunisal amyloidosis |
transthyretin peripheral neuropathy autonomic neuropathy amyloid cardiomyopathy |
|
Anti-Inflammatory Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Neurodegenerative Diseases Amyloid Neuropathies, Familial Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Neuromuscular Diseases Sensory System Agents Therapeutic Uses Anti-Inflammatory Agents, Non-Steroidal Analgesics Diflunisal Metabolic Diseases |
Amyloidosis, Familial Amyloid Neuropathies Nervous System Diseases Cyclooxygenase Inhibitors Polyneuropathies Enzyme Inhibitors Pharmacologic Actions Amyloidosis Genetic Diseases, Inborn Analgesics, Non-Narcotic Peripheral Nervous System Diseases Peripheral Nervous System Agents Antirheumatic Agents Central Nervous System Agents |
