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The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)

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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

Tracking Information
First Submitted Date September 9, 2011
First Posted Date September 13, 2011
Last Update Posted Date August 24, 2015
Study Start Date August 2011
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 26, 2013)
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.
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures
 (submitted: June 26, 2013)
  • 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.
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title The Effect of Diflunisal on Familial Transthyretin Amyloidosis
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.
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.

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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples Without DNA
Description:
Serum Whole blood Urine
Sampling Method Probability Sample
Study Population Patients visiting the Units for Familal amyloidosis in Umeå, Piteå and Skellefteå
Condition Amyloidosis
Intervention Drug: Diflunisal
Film-coated tablet, 250 mg twice daily, orally for approximately 2 years
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: August 21, 2015)
55
Original Estimated Enrollment
 (submitted: September 12, 2011)
40
Actual Study Completion Date December 2014
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Sweden
Removed Location Countries  
 
Administrative Information
NCT Number NCT01432587
Other Study ID Numbers DFNS01
2011-000776-34 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Ole B Suhr, Professor, MD, PhD, Umeå University
Study Sponsor Umeå University
Collaborators Not Provided
Investigators
Principal Investigator: Ole B Suhr, MD PhD Dept of Clinical Medicine and public Health, Umeå University
PRS Account Umeå University
Verification Date August 2015