Clinical Trial Results:
Transthyretin-Associated Amyloidosis Outcomes Survey (THAOS) – Optional Blood Sample Collection Sub-study.
Summary
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EudraCT number |
2013-004090-28 |
Trial protocol |
IT |
Global end of trial date |
30 Apr 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
14 May 2016
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First version publication date |
14 May 2016
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Other versions |
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Summary report(s) |
B3461049 - Public Disclosure Synopsis |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
B3461049 (Fx-R-001-S1)
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Pfizer Inc.
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Sponsor organisation address |
235 E 42nd Street, New York, United States, NY 10017
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Public contact |
Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
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Scientific contact |
Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
15 Apr 2016
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
30 Apr 2015
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Global end of trial reached? |
Yes
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Global end of trial date |
30 Apr 2015
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The objective of this study was to collect blood samples planned to assist in the development and validation of a biomarker assay for transthyretin (TTR) amyloidoses.
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Protection of trial subjects |
The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Council on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
06 Aug 2014
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Argentina: 5
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Country: Number of subjects enrolled |
Germany: 3
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Country: Number of subjects enrolled |
Mexico: 5
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Country: Number of subjects enrolled |
Portugal: 15
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Country: Number of subjects enrolled |
United States: 2
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Country: Number of subjects enrolled |
Italy: 2
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Worldwide total number of subjects |
32
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EEA total number of subjects |
20
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
29
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From 65 to 84 years |
3
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||
Pre-assignment
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Screening details |
This sub study of THAOS was a protocol to collect blood samples from enrolled subjects who had Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, Val122Ile mutation, symptomatic ATTR disease, not treated with any medication which stabilizes or reduces the concentration of TTR,TTR monomer or oligomer in blood. | ||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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Entire Study Population | ||||||
Arm description |
Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study. | ||||||
Arm type |
Blood Sample Collection Substudy | ||||||
Investigational medicinal product name |
None
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule
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Routes of administration |
Unknown use
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Dosage and administration details |
No investigational medicinal product was administered in this study.
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Baseline characteristics reporting groups
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Reporting group title |
Overall Study
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Entire Study Population
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Reporting group description |
Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study. |
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End point title |
Number of Subjects With Their Blood Samples Collected To Assist in Development and Validation of Biomarker Assay for Transthyretin (TTR) Amyloidoses [1] | ||||||
End point description |
Blood was collected from subjects who had the following mutations in the TTR protein: Val30Met, Glu89Gln, Thr60Ala, Ser50Arg, Val20Ile, and Val122Ile, had symptomatic ATTR disease, and had not been treated with any medication or treatment which stabilizes or reduces the concentration of TTR, TTR monomer, or TTR oligomer in the blood. These blood samples were collected to assist in the development and validation of a biomarker assay for TTR amyloidosis. Entire study population was analyzed.
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End point type |
Primary
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End point timeframe |
Baseline up to 268 days
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analysis was planned or conducted for this substudy. |
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
Time of consent up to 28 days after the last visit
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Assessment type |
Systematic | ||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
No dictionary used | ||||||||||
Dictionary version |
N/A
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Reporting groups
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Reporting group title |
Entire Study Population
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Reporting group description |
Subject who had symptomatic transthyretin amyloidosis (ATTR) disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR protein and were a part of THAOS were enrolled into this sub-study. | ||||||||||
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Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||
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Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: This was a blood draw study in which no study drug was administered and there were no adverse events and no safety issues reported. |
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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30 Aug 2013 |
The protocol was updated to limit scope of blood collection for the purpose of development and validation of a biomarker assay and to remove the storage of samples for Biobanking, The amendment updated the sponsor name as FoldRx Pharmaceuticals, Inc had become a wholly-owned subsidiary of Pfizer, Inc. Pfizer protocol study number (B3461049) was inserted and protocol sections were renumbered to align with Pfizer protocol standards. Inclusion criteria and exclusion criteria were added to further clarify the substudy population. Language was added to clarify sample size determination and statistical analysis and to define study discontinuation criteria. There was a change in nomenclature whereby the term “patient” became “subject” and a change in the abbreviation ATTR-PN to TTR-FAP. |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |