The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)
This study is currently recruiting participants.
Verified September 2011 by Umeå University
Sponsor:
Umeå University
Information provided by (Responsible Party):
Ole B Suhr, Umeå University
ClinicalTrials.gov Identifier:
NCT01432587
First received: September 9, 2011
Last updated: September 12, 2011
Last verified: September 2011
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Purpose
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 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 | Intervention |
|---|---|
|
Amyloidosis |
Drug: Diflunisal |
| Study Type: | Observational |
| Study Design: | 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. |
Resource links provided by NLM:
Genetics Home Reference related topics:
transthyretin amyloidosis
MedlinePlus related topics:
Amyloidosis
Drug Information available for:
Diflunisal
U.S. FDA Resources
Further study details as provided by Umeå University:
Biospecimen Retention: Samples Without DNA
Serum Whole blood Urine
| Estimated Enrollment: | 40 |
| Study Start Date: | August 2011 |
| Estimated Study Completion Date: | August 2013 |
| Estimated Primary Completion Date: | August 2013 (Final data collection date for primary outcome measure) |
Intervention Details:
Detailed Description:
-
Drug: Diflunisal
Film-coated tablet, 250 mg twice daily, orally for approximately 2 years
Duration of treatment in this study is dependent of the result from the ongoing IND 68092-study, which are planned to be presented 2013.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| 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 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01432587
Contacts
| Contact: Ole B Suhr, MD PhD | +46 90 785 0000 ext 3959 | ole.suhr@medicin.umu.se |
Locations
| Sweden | |
| Dept of Clinical Medicin, Ptieå Hospital | Recruiting |
| Piteå, Sweden, SE-941 28 | |
| Contact: Katarzyna Liszewska, MD 0911 - 750 00 ext 232 Katarzyna.Liszewska@nll.se | |
| Principal Investigator: Katarzyna Liszewska, MD | |
| Dept of clinical medicin, Skellefteå Hospital | Recruiting |
| Skellefteå, Sweden, SE-931 86 | |
| Contact: Lars Wikström, MD +46 910-7710 00 ext 1667 lars.wikstrom@vll.se | |
| Principal Investigator: Lars Wikström, MD | |
| Dept of Clinical Medicine, Umeå University Hospital | Recruiting |
| Umeå, Sweden, SE-90185 | |
| Contact: Ole B Suhr, MD PhD +46 90 785 0000 ext 3959 ole.suhr@medicin.umu.se | |
| Contact: Christina Frykholm, Nurse +46 90 785 0000 ext 3959 fapteam.medicin@vll.se | |
| Principal Investigator: Ole B Suhr, MD PhD | |
Sponsors and Collaborators
Umeå University
Investigators
| Principal Investigator: | Ole B Suhr, MD PhD | Dept of Clinical Medicine and public Health, Umeå University |
More Information
No publications provided
| Responsible Party: | Ole B Suhr, M.D. Ph.D., Umeå University |
| ClinicalTrials.gov Identifier: | NCT01432587 History of Changes |
| Other Study ID Numbers: | DFNS01, 2011-000776-34 |
| Study First Received: | September 9, 2011 |
| Last Updated: | September 12, 2011 |
| Health Authority: | Sweden: Medical Products Agency |
Keywords provided by Umeå University:
|
Amyloidosis Transthyretin Neuropathies Diflunisal Familial |
Additional relevant MeSH terms:
|
Amyloidosis Proteostasis Deficiencies Metabolic Diseases Diflunisal Cyclooxygenase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Anti-Inflammatory Agents, Non-Steroidal |
Analgesics, Non-Narcotic Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents Therapeutic Uses Antirheumatic Agents Central Nervous System Agents |
ClinicalTrials.gov processed this record on May 23, 2013