Clinical Trial Results:
A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)
Summary
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EudraCT number |
2014-004856-68 |
Trial protocol |
NO NL FR |
Global end of trial date |
15 Feb 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
26 Mar 2017
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First version publication date |
26 Mar 2017
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Other versions |
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Summary report(s) |
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 |
CB8025-21427
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02472535 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
CymaBay Therapeutics, Inc
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Sponsor organisation address |
7999 Gateway Blvd, suite 130, Newark, CA, United States, 94560
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Public contact |
Pol Boudes, CymaBay Therapeutics, Inc., 001 5102938815, pboudes@cymabay.com
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Scientific contact |
Pol Boudes, CymaBay Therapeutics, Inc., 001 5102938815, pboudes@cymabay.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 Feb 2016
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
15 Feb 2016
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Global end of trial reached? |
Yes
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Global end of trial date |
15 Feb 2016
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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 |
Primary:
To evaluate the effect of MBX-8025 on Low Density Lipoprotein Cholesterol (LDL-C).
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Protection of trial subjects |
If AEs were severe and drug related, the subject stopped treatment and entered the follow-up period.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
29 Apr 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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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 |
Norway: 2
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Country: Number of subjects enrolled |
Canada: 5
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Country: Number of subjects enrolled |
Netherlands: 2
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Country: Number of subjects enrolled |
France: 4
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Worldwide total number of subjects |
13
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EEA total number of subjects |
8
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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) |
11
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From 65 to 84 years |
2
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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 |
The screening period was a maximum of 2 weeks. All subjects had to confirm eligibility on Visit 2 (Week 0) prior to entering the run-in period | ||||||
Period 1
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Period 1 title |
2 weeks screening period
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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single-arm | ||||||
Arm description |
- | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
No investigational medicinal product assigned in this arm
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Period 2
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Period 2 title |
Run-in period
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Is this the baseline period? |
Yes [1] | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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2 weeks treatment with placebo | ||||||
Arm description |
2 weeks treatment with placebo | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
Placebo
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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 |
Oral use
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Dosage and administration details |
The placebo treatment was administered PO once daily and only during the run-in period. The placebo was a gelatin capsule, containing all ingredients in the investigational product, except for the active pharmaceutical ingredient, MBX-8025. Only 1 batch of placebo was used: batch number 14G058.
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Notes [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period. Justification: Period 2 is the Baseline period |
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Period 3
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Period 3 title |
Treatment Phase 1
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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4 weeks treatment with 50 mg daily | ||||||
Arm description |
4 weeks treatment with 50 mg daily | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
MBX-8025
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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 |
Oral use
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Dosage and administration details |
The test product was MBX-8025 capsules (50 mg). Dosing was oral (PO), once daily, in doses of 50 mg (first 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059.
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Period 4
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Period 4 title |
Treatment Phase 2
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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4 weeks treatment with 100 mg daily | ||||||
Arm description |
4 weeks treatment with 100 mg daily | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
MBX-8025
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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 |
Oral use
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Dosage and administration details |
The test product was MBX-8025 capsules (50 or 100 mg). Dosing was oral (PO), once daily, in doses of 100 or 50 mg (second 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059, and the 100 mg MBX-8025 test product was batch number 14G060.
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Period 5
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Period 5 title |
Treatment Phase 3
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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4 weeks treatment with 200 mg daily | ||||||
Arm description |
4 weeks treatment with 200 mg daily | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
MBX-8025
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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 |
Oral use
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Dosage and administration details |
The test product was MBX-8025 capsules (50 or 100 mg). Dosing was oral (PO), once daily, in doses of 200, 100, or 50 (third 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059, and the 100 mg MBX-8025 test product was batch number 14G060.
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Period 6
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Period 6 title |
Follow-up period
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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2 weeks follow-up | ||||||
Arm description |
2 weeks follow-up | ||||||
Arm type |
Cohort | ||||||
Investigational medicinal product name |
No investigational medicinal product assigned in this arm
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Baseline characteristics reporting groups
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Reporting group title |
Run-in period
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
single-arm
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Reporting group description |
- | ||
Reporting group title |
2 weeks treatment with placebo
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Reporting group description |
2 weeks treatment with placebo | ||
Reporting group title |
4 weeks treatment with 50 mg daily
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Reporting group description |
4 weeks treatment with 50 mg daily | ||
Reporting group title |
4 weeks treatment with 100 mg daily
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Reporting group description |
4 weeks treatment with 100 mg daily | ||
Reporting group title |
4 weeks treatment with 200 mg daily
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Reporting group description |
4 weeks treatment with 200 mg daily | ||
Reporting group title |
2 weeks follow-up
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Reporting group description |
2 weeks follow-up |
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End point title |
Safety [1] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
4 weeks
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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: Please see attached synopsis |
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No statistical analyses for this end point |
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End point title |
Efficacy [2] | ||||||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
4 weeks
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Notes [2] - 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: Please see attached synopsis |
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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 |
From baseline through week 16
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Assessment type |
Systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
18.0
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Frequency threshold for reporting non-serious adverse events: 5% | |||
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: Please see attached synopsis |
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
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 |