The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)
![]() |
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: NCT01432587 |
Recruitment Status :
Completed
First Posted : September 13, 2011
Last Update Posted : August 24, 2015
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
An ongoing trial of diflunisal has been closed for enrollment, thus, patients suitable for the study can no longer participate or receive treatment by diflunisal; and patients, who have participated in the trial can not continue their treatment. The investigators want to continue to monitor the effect of the drug on transthyretin (TTR) amyloidosis in an open label observational study.
Primary endpoint will be a composite score of the manifestations of the disease (Kumamoto scale) and secondary end points will be measurements of neurological impairment, heart involvement and nutritional status.
Condition or disease | Intervention/treatment |
---|---|
Amyloidosis | Drug: Diflunisal |
Study Type : | Observational |
Actual Enrollment : | 55 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | The Effect of Diflunisal on Familial Transthyretin Amyloidosis: An Open Label Extension Study of "the Diflunisal Trial" (IND 68092), and an Open Label Observational Study on Previously Untreated Patients With Familial Transthyretin Amyloidosis. |
Study Start Date : | August 2011 |
Actual Primary Completion Date : | December 2014 |
Actual Study Completion Date : | December 2014 |

- Drug: Diflunisal
Film-coated tablet, 250 mg twice daily, orally for approximately 2 years
- Changes in the Kumamoto scale [ Time Frame: Enrollment, 12 month and annual follow-up ]Composite score of the manifestations of the disease (Kumamoto Scale). Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in modified body mass index (mBMI) [ Time Frame: Enrollment, 12 month and annual follow-up ]Changes in nutritional status measured by mBMI.Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in paraneoplastic neurological disorders (PND) scale [ Time Frame: Enrollment, 12 month and annual follow-up ]Neurological impairment measured by the PND-score. Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in cardiac function [ Time Frame: Enrollment, 1 month, 2 month, 3 month, 6 month, 9 month 12 month, 18 month and annual follow-up ]Cardiac impairment is measure by echocardiographic measurement of septal thickness and by proBNP in blood samples. Results at enrollment will be compared to results during the study and annual follow-ups.
- Safety follow-up Blood Work [ Time Frame: 1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months ]To follow-up the patient safety during the study and follow-up the blood samples for (B-Hb), blood platelets, s-creatinine, liver enzymes [aspartate transaminase (ASAT),alanine aminotransferase (ALAT), s-bilirubin and alkaline phosphatase (ALP)],serum proBNP (S-proBNP) are drawn. Results at enrollment will be compares to results during study and every 6-month follow-ups.
Biospecimen Retention: Samples Without DNA

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 |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
Inclusion Criteria:
- Biopsy and genetically proven systemic transthyretin amyloidosis caused by a TTR gene mutation. The amyloid shall be proven to be of transthyretin type, and the fibril composition settled.
- Age ≥ 18 years.
- Negative pregnancy test and contraception for sexually active women of child bearing potential.
Exclusion Criteria:
- Concomitant use of non-study non-steroidal anti-inflammatory drugs (NSAIDs)
- Heart failure with symptoms at daily activities (NYHA class ≥III)
- Renal insufficiency (creatinine clearance < 30 ml calculated from the Cockcroft-Gault formula)
- Active non-haemorrhoidal bleeding within the last 18 month.
- Non-treated peptic ulcer disease.
- Anticoagulation therapy, low dose ASA permitted.
- Non-steroidal or aspirin allergy/hypersensitivity
- Thrombocytopenia (< 100,000 platelets/mm3)
- Inability or unwillingness of subject to give written informed consent
- By the investigator regarded as unable to follow the study guidelines and scheduled controls.

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): NCT01432587
Sweden | |
Dept of Clinical Medicin, Ptieå Hospital | |
Piteå, Sweden, SE-941 28 | |
Dept of clinical medicin, Skellefteå Hospital | |
Skellefteå, Sweden, SE-931 86 | |
Dept of Clinical Medicine, Umeå University Hospital | |
Umeå, Sweden, SE-90185 |
Principal Investigator: | Ole B Suhr, MD PhD | Dept of Clinical Medicine and public Health, Umeå University |
Responsible Party: | Ole B Suhr, Professor, MD, PhD, Professor MD PhD, Umeå University |
ClinicalTrials.gov Identifier: | NCT01432587 |
Other Study ID Numbers: |
DFNS01 2011-000776-34 ( EudraCT Number ) |
First Posted: | September 13, 2011 Key Record Dates |
Last Update Posted: | August 24, 2015 |
Last Verified: | August 2015 |
Amyloidosis Transthyretin Neuropathies Diflunisal Familial |
Amyloidosis Proteostasis Deficiencies Metabolic Diseases Diflunisal Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Analgesics Sensory System Agents |
Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents Antirheumatic Agents Cyclooxygenase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |