AmyloidosisDFNS01NCT01432587
The Effect of Diflunisal on Familial Transthyretin Amyloidosis
An observational study of Amyloidosis, run by Umeå University at 3 sites in Sweden.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis
- What is being tested?
- Diflunisal
- Type of treatment
- Pill or oral medicine
- How many people
- 55 participants
- Where
- 3 sitesSweden
- Study dates
- Aug 2011to Dec 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients visiting the Units for Familal amyloidosis in Umeå, Piteå and Skellefteå
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
3 sites in 1 country.
Sweden3 sites
Dept of Clinical Medicin, Ptieå Hospital
Piteå, SE-941 28
Dept of clinical medicin, Skellefteå Hospital
Skellefteå, SE-931 86
Dept of Clinical Medicine, Umeå University Hospital
Umeå, SE-90185
About this study
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.
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.
Read the 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.
- Conditions
- Amyloidosis
- Being tested
- Diflunisal
- Keywords
- Amyloidosis, Transthyretin, Neuropathies, Diflunisal, Familial
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DiflunisalDrug
Film-coated tablet, 250 mg twice daily, orally for approximately 2 years
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Enrollment, 12 month and annual follow-up
Secondary outcomes (4)
Changes in modified body mass index (mBMI)
Changes in nutritional status measured by mBMI.Results at enrollment will be compared to results at 12 months and annual follow-ups.
Measured: Enrollment, 12 month and annual follow-up
Changes in paraneoplastic neurological disorders (PND) scale
Neurological impairment measured by the PND-score. Results at enrollment will be compared to results at 12 months and annual follow-ups.
Measured: Enrollment, 12 month and annual follow-up
Changes in cardiac function
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.
Measured: Enrollment, 1 month, 2 month, 3 month, 6 month, 9 month 12 month, 18 month and annual follow-up
Safety follow-up Blood Work
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.
Measured: 1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2011 (estimated)
- Study startAug 2011
- Primary completionDec 2014
- Study completionDec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- More trials run by Umeå UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Umeå University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ole B Suhr, Professor, MD, PhD, Umeå University
- Investigators
- Ole B Suhr, MD PhD, Principal investigator, Dept of Clinical Medicine and public Health, Umeå University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01432587
- Sponsor's protocol ID
- DFNS01
- Other IDs
- 2011-000776-34
- Acronym
- DFNS01
- Status last verified
- Aug 2015
- First submitted
- 9 Sep 2011
- Last updated
- 24 Aug 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT01432587
- Acronym / sponsor ID
- DFNS01 / DFNS01
- Conditions
- Amyloidosis
- Keywords
- Amyloidosis, Transthyretin, Neuropathies, Diflunisal, Familial
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Umeå UniversityUniversity, hospital or other
- Study start
- Aug 2011
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 13 Sep 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients visiting the Units for Familal amyloidosis in Umeå, Piteå and Skellefteå
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DiflunisalFilm-coated tablet, 250 mg twice daily, orally for approximately 2 years | Drug | – |
Primary outcome
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.
4 secondary outcomes
Changes in modified body mass index (mBMI)
Time frame: Enrollment, 12 month and annual follow-up
Changes in paraneoplastic neurological disorders (PND) scale
Time frame: Enrollment, 12 month and annual follow-up
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
Safety follow-up Blood Work
Time frame: 1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ole B Suhr, MD PhD principal investigator
Dept of Clinical Medicine and public Health, Umeå University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyloidosis, run by Umeå University at 3 sites in Sweden.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in the Kumamoto scale (time frame: Enrollment, 12 month and annual follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2015.