Trial · NCT00294671
The Effect of Diflunisal on Familial Amyloidosis
NCT00294671, Phase 2, Phase 3, Completed, Boston University; results first posted 2017-03-17
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. The Effect of Diflunisal on Familial Amyloidosis. Trials, record NCT00294671. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00294671
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The Effect of Diflunisal on Familial Amyloidosis
- NCT number
- NCT00294671
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 130 participants, actual
- Lead sponsor
- Boston University
- Conditions
- Familial Amyloid Polyneuropathy, Familial Amyloidosis
- Interventions
- diflunisal, placebo
- First posted
- 2006-02-22
- Last updated
- 2017-03-17
- Results first posted
- 2017-03-17
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Neurologic Impairment Score + 7 (NIS+7) | Baseline, 1 and 2 years | The primary endpoint, the difference in polyneuropathy progression between treatments, was measured by the Neuropathy Impairment Score plus 7 nerve tests (NIS+7) which ranges from 0 (no neurologic deficits) to 270 points (no detectable peripheral nerve function). |
| Secondary | Kumamoto Neurologic Scale; | Baseline, 1 and 2 years | Change from baseline of the Kumamoto Score (0-102 points, increasing with disease severity), a clinical neurologic scale of motor, sensory, and autonomic function combined with heart and kidney end organ measures developed to track disease progression in Familial Amyloid Polyneuropathy (ATTR-FAP) |
| Secondary | Modified Body Mass Index (mBMI); | Baseline, 1 and 2 years | The product of body mass index (BMI) and serum albumin level (g/L) \[kg/M2xg/L\]. |
| Secondary | Quality of Life Questionnaire: SF-36 Physical Component Score | Baseline, 1 and 2 years | The 36 item short-form health survey (SF-36) was used to assess the difference between treatment groups for change of physical component scores over 2 years treatment. Range 0-100; lower scores reflect lower quality-of-life. |
| Secondary | Quality of Life Questionnaire: SF-36 Mental Component Score | Baseline, 1 and 2 years | The 36 item short-form health survey (SF-36) was used to assess the difference between treatment groups for change of mental component scores over 2 years treatment. Range 0-100; lower scores reflect lower quality-of-life. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Neurologic Impairment Score + 7 (NIS+7) | Baseline, 1 and 2 years | Longitudinal analysis examined data from all 130 participants using intention-to-treat principles. | units on a scale | MEAN |
| SECONDARY | Kumamoto Neurologic Scale; | Baseline, 1 and 2 years | units on a scale | MEAN | |
| SECONDARY | Modified Body Mass Index (mBMI); | Baseline, 1 and 2 years | kg/M2xg/L | MEAN | |
| SECONDARY | Quality of Life Questionnaire: SF-36 Physical Component Score | Baseline, 1 and 2 years | Longitudinal analysis examined data from all 130 participants using intention-to-treat principles. | units on a scale | MEAN |
| SECONDARY | Quality of Life Questionnaire: SF-36 Mental Component Score | Baseline, 1 and 2 years | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Neurologic Impairment Score + 7 (NIS+7) | Diflunisal | Change from baseline to 2 years | 8.2 | 95% Confidence Interval 2.9 to 13.6 | units on a scale |
| Neurologic Impairment Score + 7 (NIS+7) | Placebo | Change from baseline to 2 years | 26.3 | 95% Confidence Interval 20.2 to 32.4 | units on a scale |
| Neurologic Impairment Score + 7 (NIS+7) | Diflunisal | Change from baseline to 1 year | 6.2 | 95% Confidence Interval 2.8 to 9.6 | units on a scale |
| Neurologic Impairment Score + 7 (NIS+7) | Placebo | Change from baseline to 1 year | 12.5 | 95% Confidence Interval 8.6 to 16.4 | units on a scale |
| Kumamoto Neurologic Scale; | Diflunisal | Change from baseline to 2 years | 3.1 | 95% Confidence Interval 1.1 to 5.1 | units on a scale |
| Kumamoto Neurologic Scale; | Placebo | Change from baseline to 2 years | 8.0 | 95% Confidence Interval 5.8 to 10.3 | units on a scale |
| Kumamoto Neurologic Scale; | Diflunisal | Change from baseline to 1 year | 1.9 | 95% Confidence Interval 0.1 to 3.7 | units on a scale |
| Kumamoto Neurologic Scale; | Placebo | Change from baseline to 1 year | 4.1 | 95% Confidence Interval 2.1 to 6.2 | units on a scale |
| Modified Body Mass Index (mBMI); | Diflunisal | Change from baseline to 2 years | -33.7 | 95% Confidence Interval -69.3 to 1.8 | kg/M2xg/L |
| Modified Body Mass Index (mBMI); | Placebo | Change from baseline to 2 years | -67.9 | 95% Confidence Interval -108.1 to -27.7 | kg/M2xg/L |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Diflunisal | 64 | |
| Overall Study | STARTED | Placebo | 66 | |
| Overall Study | COMPLETED | Diflunisal | 37 | |
| Overall Study | COMPLETED | Placebo | 26 | |
| Overall Study | NOT COMPLETED | Diflunisal | 27 | |
| Overall Study | NOT COMPLETED | Placebo | 40 |
Source ClinicalTrials.gov
Adverse events56
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Infections | Infections and infestations | Diflunisal | 24 | 64 | |
| other | Infections | Infections and infestations | Placebo | 27 | 66 | |
| other | GI disorders | Gastrointestinal disorders | Diflunisal | 23 | 64 | |
| other | GI disorders | Gastrointestinal disorders | Placebo | 25 | 66 | |
| other | Nervous system disorders | Nervous system disorders | Diflunisal | 23 | 64 | |
| other | Nervous system disorders | Nervous system disorders | Placebo | 20 | 66 | |
| other | Musculoskeletal and connective tissue disorders | Musculoskeletal and connective tissue disorders | Diflunisal | 19 | 64 | |
| other | Musculoskeletal and connective tissue disorders | Musculoskeletal and connective tissue disorders | Placebo | 8 | 66 | |
| other | General disorders | General disorders | Diflunisal | 19 | 64 | |
| other | General disorders | General disorders | Placebo | 7 | 66 |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Diflunisal | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Diflunisal | 29 | 64 |
| Total, other adverse events | Placebo | 27 | 66 |
| Total, serious adverse events | Diflunisal | 3 | 64 |
| Total, serious adverse events | Placebo | 4 | 66 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 24368466 | DERIVED |
Source PubMed
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01NS051306 | Effect of diflunisal (IND68092) on familial amyloidosis | BERK, JOHN L | BOSTON UNIVERSITY MEDICAL CAMPUS | NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE | 2005 to 2009 |
Source NIH RePORTER
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Diflunisal vs Placebo: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00294671
GET https://conductscience.org/api/trials/v1/NCT00294671/events?page=2