Trial · NCT00630864
The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
NCT00630864, Phase 2, Completed, Pfizer; results first posted 2012-12-17
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis. Trials, record NCT00630864. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00630864
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
- NCT number
- NCT00630864
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 21 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Transthyretin-associated Amyloidosis With Polyneuropathy
- Interventions
- Fx-1006A
- First posted
- 2008-03-07
- Last updated
- 2013-01-18
- Results first posted
- 2012-12-17
Source ClinicalTrials.gov
Registered outcomes34
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Week 6 | Week 6 | TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI. |
| Secondary | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Month 6 and 12 | Month 6, Month 12 | TTR tetramer was assessed using a validated immunoturbidimetric assay. The FOI is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI. |
| Other | Number of Participants With Treatment-Emergent Adverse Events (AEs) | Baseline up to 30 days after the last dose | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state. |
| Other | Number of Participants With Greater Than or Equal to Grade 3 Treatment-Emergent Adverse Events | Baseline up to 30 days after the last dose | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state. On the basis of intensity, grade 3 was referred as severe, grade 4 as life-threatening and grade 5 as death. |
| Other | Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Day 1 up to Month 12 | ECHO: investigator assessed test to assess cardiac function. ECHO abnormality criteria: any abnormality, valvular abnormality, pericardial effusion, abnormal regional wall motion, inferior vena cava respiratory variation, posterior (P) left ventricular (LV) wall/septal (S) thickness, right ventricular thickness, ejection fraction, ratio of early (E) diastolic transmitral flow and atrial(A) contraction velocity (E/A), ratio of 'E'to lateral/septal mitral annular velocity (e') (E/e'prime lateral, E/e'prime septal), E deceleration time (DT), isovolumic relaxation time (IVRT). |
| Other | Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings | Day 1 up to Month 12 | ECG: investigator assessed test to assess cardiac function. ECG abnormality criteria: any abnormality, arrhythmia, rhythm, conduction, morphology, myocardial infarction, ST segment, T waves and abnormal U waves. |
| Other | Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings | Day 1 up to Month 12 | Holter monitoring recorded heart rhythm. Holter monitoring abnormality criteria: any abnormality, atrial fibrillation/flutter, atrial tachycardia, non-sustained ventricular tachycardia (VT), sustained VT and sinus pause. |
| Other | Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events | Baseline up to Month 12 | |
| Other | Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 6, Month 12 | Baseline, Month 6, Month 12 | NIS assessed cranial nerves(nerve 3,6; facial, palate and tongue weakness),muscle weakness (respiratory; neck, elbow(E), wrist(W), finger(F), hip, knee(K) flexion; shoulder, thumb abduction; brachioradialis; E, W, hip, K extension; F spread; toe, dorsal and plantar ankle flexors; toe extensors); score: 0-4, higher score=more weakness, reflexes(biceps and triceps brachii; brachioradialis; quadriceps femoris; triceps surae), index F and great toe sensation(touch pressure, pin-prick, vibration, joint position)score:0=normal,1=decreased or 2=absent. Total score=0-244, higher score=more impairment. |
| Other | Change From Baseline in the Neuropathy Impairment Score-Lower Limb (NIS-LL) at Month 6, Month 12 | Baseline, Month 6, Month 12 | NIS-LL: assessed muscle weakness, reflexes and sensation; scored separately for left and right limbs. Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) are scored on 0 to 4 scale, higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) were scored 0 = normal, 1= decreased, or 2 = absent. Total possible NIS-LL score range 0-88, higher score=greater impairment. |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 registered outcomesOutcomes34
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Week 6 | Week 6 | Intent-to-Treat (ITT) population included all participants who received at least 1 dose of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Month 6 and 12 | Month 6, Month 12 | ITT population included all participants who received at least 1 dose of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure. | percentage of participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants With Treatment-Emergent Adverse Events (AEs) | Baseline up to 30 days after the last dose | ITT population included all participants who received at least 1 dose of study medication. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants With Greater Than or Equal to Grade 3 Treatment-Emergent Adverse Events | Baseline up to 30 days after the last dose | ITT population included all participants who received at least 1 dose of study medication. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Day 1 up to Month 12 | ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings | Day 1 up to Month 12 | ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings | Day 1 up to Month 12 | ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events | Baseline up to Month 12 | ITT population included all participants who received at least 1 dose of study medication. | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 6, Month 12 | Baseline, Month 6, Month 12 | ITT population included all participants who received at least 1 dose of study medication. Here, 'n' signifies those participants who were analyzed for the specific category. | units on a scale | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in the Neuropathy Impairment Score-Lower Limb (NIS-LL) at Month 6, Month 12 | Baseline, Month 6, Month 12 | ITT population included all participants who received at least 1 dose of study medication. Here, 'n' signifies those participants who were analyzed for the specific category. | units on a scale | MEAN |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 outcomesResults by group201
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Week 6 | Tafamidis | 94.7 | 95% Confidence Interval 74.0 to 99.9 | percentage of participants | |
| Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Month 6 and 12 | Tafamidis | Month 6 (n= 18) | 100.0 | 95% Confidence Interval 81.5 to 100.0 | percentage of participants |
| Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Month 6 and 12 | Tafamidis | Month 12 (n= 17) | 100.0 | 95% Confidence Interval 80.5 to 100.0 | percentage of participants |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Tafamidis | 17 | participants | ||
| Number of Participants With Greater Than or Equal to Grade 3 Treatment-Emergent Adverse Events | Tafamidis | 3 | participants | ||
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Tafamidis | Any ECHO abnormalities (n= 19) | 12 | participants | |
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Tafamidis | Pericardial effusion (n= 19) | 2 | participants | |
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Tafamidis | Valvular abnormalities- thickening (n= 5) | 3 | participants | |
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Tafamidis | Valvular abnormalities - regurgitation (n= 9) | 2 | participants | |
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | Tafamidis | Abnormal regional wall motion (n= 11) | 2 | participants |
1 to 10 of 201Source ClinicalTrials.gov
See all 201 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 (up to Week 6) | STARTED | Tafamidis | 21 | |
| Part 1 (up to Week 6) | COMPLETED | Tafamidis | 20 | |
| Part 1 (up to Week 6) | NOT COMPLETED | Tafamidis | 1 | |
| Part 2 (After Week 6 up to Month 12) | STARTED | Tafamidis | 20 | |
| Part 2 (After Week 6 up to Month 12) | COMPLETED | Tafamidis | 18 | |
| Part 2 (After Week 6 up to Month 12) | NOT COMPLETED | Tafamidis | 2 |
Source ClinicalTrials.gov
Adverse events32
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 | Diarrhoea | Gastrointestinal disorders | Tafamidis | 5 | 21 | |
| other | Vomiting | Gastrointestinal disorders | Tafamidis | 3 | 21 | |
| other | Constipation | Gastrointestinal disorders | Tafamidis | 3 | 21 | |
| other | Nausea | Gastrointestinal disorders | Tafamidis | 2 | 21 | |
| other | Fatigue | General disorders | Tafamidis | 2 | 21 | |
| other | Oedema peripheral | General disorders | Tafamidis | 2 | 21 | |
| other | Laryngitis | Infections and infestations | Tafamidis | 2 | 21 | |
| other | Sinusitis | Infections and infestations | Tafamidis | 2 | 21 | |
| other | Fall | Injury, poisoning and procedural complications | Tafamidis | 4 | 21 | |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Tafamidis | 4 | 21 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tafamidis | ||
| Total, other adverse events | Tafamidis | 13 | 21 |
| Total, serious adverse events | Tafamidis | 8 | 21 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
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.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00630864
GET https://conductscience.org/api/trials/v1/NCT00630864/registered?page=2