NCT01432587

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
55

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2011

Typical duration for all trials

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2011

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 9, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 13, 2011

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
Last Updated

August 24, 2015

Status Verified

August 1, 2015

Enrollment Period

3.3 years

First QC Date

September 9, 2011

Last Update Submit

August 21, 2015

Conditions

Amyloidosis

Keywords

AmyloidosisTransthyretinNeuropathiesDiflunisalFamilial

Outcome Measures

Primary Outcomes (1)

  • Changes in the Kumamoto scale

    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.

    Enrollment, 12 month and annual follow-up

Secondary Outcomes (4)

  • Changes in modified body mass index (mBMI)

    Enrollment, 12 month and annual follow-up

  • Changes in paraneoplastic neurological disorders (PND) scale

    Enrollment, 12 month and annual follow-up

  • Changes in cardiac function

    Enrollment, 1 month, 2 month, 3 month, 6 month, 9 month 12 month, 18 month and annual follow-up

  • Safety follow-up Blood Work

    1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months

Interventions

DiflunisalDRUG

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

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Patients visiting the Units for Familal amyloidosis in Umeå, Piteå and Skellefteå

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Dept of Clinical Medicin, Ptieå Hospital

Piteå, SE-941 28, Sweden

Location

Dept of clinical medicin, Skellefteå Hospital

Skellefteå, SE-931 86, Sweden

Location

Dept of Clinical Medicine, Umeå University Hospital

Umeå, SE-90185, Sweden

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum Whole blood Urine

MeSH Terms

Conditions

Amyloidosis

Interventions

Diflunisal

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

SalicylatesHydroxybenzoatesPhenolsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic Chemicals

Study Officials

  • Ole B Suhr, MD PhD

    Dept of Clinical Medicine and public Health, Umeå University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor MD PhD

Study Record Dates

First Submitted

September 9, 2011

First Posted

September 13, 2011

Study Start

August 1, 2011

Primary Completion

December 1, 2014

Study Completion

December 1, 2014

Last Updated

August 24, 2015

Record last verified: 2015-08

Locations

SE(3)