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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
Sponsor:
Information provided by (Responsible Party):
Ole B Suhr, Professor, MD, PhD, Umeå University

Study Description
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Brief Summary:

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

Detailed Description:
Duration of treatment in this study is dependent of the results from the ongoing IND 68092-study, which are planned to be presented 2013.
Study Design
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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Diflunisal

Groups and Cohorts
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Intervention Details:
  • Drug: Diflunisal
    Film-coated tablet, 250 mg twice daily, orally for approximately 2 years

Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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
Serum Whole blood Urine

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients visiting the Units for Familal amyloidosis in Umeå, Piteå and Skellefteå
Criteria

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.
Contacts and Locations
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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): NCT01432587


Locations
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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
Sponsors and Collaborators
Umeå University
Investigators
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Principal Investigator: Ole B Suhr, MD PhD Dept of Clinical Medicine and public Health, Umeå University
More Information
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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
Keywords provided by Ole B Suhr, Professor, MD, PhD, Umeå University:
Amyloidosis
Transthyretin
Neuropathies
Diflunisal
Familial
Additional relevant MeSH terms:
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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