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Completed

NCT Number: NCT01432587

The Effect of Diflunisal on Familial Transthyretin Amyloidosis

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.

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Key information

Conditions

Amyloidosis Metabolic Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Dept of Clinical Medicin, Ptieå Hospital, Piteå, Sweden

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

diflunisal

Drug

Film-coated tablet, 250 mg twice daily, orally for approximately 2 years

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Umeå University

Other

Registry information

Official study 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.

Acronym: DFNS01

Important dates

Study start
2011
Primary completion
2014
Study completion
2014
First posted
Sep 13, 2011
Registry last updated
Aug 24, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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