Clinical Trial Page
Clinical Trial Results:
An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)
| Summary | |
EudraCT number | 2017-003757-41 |
Trial protocol | GB AT NL DE FR |
Global end of trial date | 17 Apr 2020 |
| Results information | |
Results version number | v1(current) |
This version publication date | 17 Oct 2020 |
First version publication date | 17 Oct 2020 |
Other versions | |
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 | |||
Sponsor protocol code | CCP-020-302 | ||
| Additional study identifiers | |||
ISRCTN number | - | ||
US NCT number | NCT03389308 | ||
WHO universal trial number (UTN) | - | ||
Other trial identifiers | IND: 131 384 | ||
| Sponsors | |||
Sponsor organisation name | Castle Creek Pharmaceuticals, LLC | ||
Sponsor organisation address | 233 Mt. Airy Road, Basking Ridge, United States, 07920 | ||
Public contact | Dr. Mary Spellman, Castle Creek Pharmaceuticals, LLC, +001 8622860400, mspellman@castlecreekpharma.com | ||
Scientific contact | Dr. Mary Spellman, Castle Creek Pharmaceuticals, LLC, +001 8622860400, mspellman@castlecreekpharma.com | ||
| Paediatric regulatory details | |||
Is trial part of an agreed paediatric investigation plan (PIP) | No | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? | No | ||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? | No | ||
| Results analysis stage | |||
Analysis stage | Final | ||
Date of interim/final analysis | 08 Jul 2020 | ||
Is this the analysis of the primary completion data? | Yes | ||
Primary completion date | 17 Apr 2020 | ||
Global end of trial reached? | Yes | ||
Global end of trial date | 17 Apr 2020 | ||
Was the trial ended prematurely? | No | ||
| General information about the trial | |||
Main objective of the trial | The primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101. | ||
Protection of trial subjects | The study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and are consistent with International Council for Harmonisation (ICH)/Good Clinical Practice (GCP), applicable regulatory requirements, and the Castle Creek Policy on Bioethics. | ||
Background therapy | - | ||
Evidence for comparator | - | ||
Actual start date of recruitment | 15 Nov 2017 | ||
Long term follow-up planned | No | ||
Independent data monitoring committee (IDMC) involvement? | No | ||
| Population of trial subjects | |||
Number of subjects enrolled per country | |||
Country: Number of subjects enrolled | Netherlands: 3 | ||
Country: Number of subjects enrolled | United Kingdom: 7 | ||
Country: Number of subjects enrolled | Austria: 1 | ||
Country: Number of subjects enrolled | France: 9 | ||
Country: Number of subjects enrolled | Germany: 2 | ||
Country: Number of subjects enrolled | Australia: 2 | ||
Country: Number of subjects enrolled | Israel: 5 | ||
Country: Number of subjects enrolled | United States: 22 | ||
Worldwide total number of subjects | 51 | ||
EEA total number of subjects | 22 | ||
Number of subjects enrolled per age group | |||
In utero | 0 | ||
Preterm newborn - gestational age | 0 | ||
Newborns (0-27 days) | 0 | ||
Infants and toddlers (28 days-23 months) | 0 | ||
Children (2-11 years) | 33 | ||
Adolescents (12-17 years) | 7 | ||
Adults (18-64 years) | 11 | ||
From 65 to 84 years | 0 | ||
85 years and over | 0 | ||
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| Recruitment | |||||||||||||||||
Recruitment details | Subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101 were recruited. | ||||||||||||||||
| Pre-assignment | |||||||||||||||||
Screening details | At baseline, EBS subjects who participated in the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 pharmacokinetic study (feeder studies) and who met all of the inclusion/exclusion criteria were eligible to enroll and complete up to 2 treatment cycles of diacerein 1% ointment in this study. | ||||||||||||||||
| Period 1 | |||||||||||||||||
Period 1 title | Overall Trial (overall period) | ||||||||||||||||
Is this the baseline period? | Yes | ||||||||||||||||
Allocation method | Non-randomised - controlled | ||||||||||||||||
Blinding used | Not blinded | ||||||||||||||||
| Arms | |||||||||||||||||
| Arm title | Open Label | ||||||||||||||||
Arm description | Each treatment cycle consisted of 8 weeks on treatment (once-daily, at-home study drug applications) followed by 8 weeks off treatment (only Investigator-approved, bland, non-medicated emollient/moisturizer, routine cleansing products, and sunscreens were allowed off treatment), with a maximum of 2 treatment cycles allowed for up to 52 weeks. | ||||||||||||||||
Arm type | Experimental | ||||||||||||||||
Investigational medicinal product name | Diacerein | ||||||||||||||||
Investigational medicinal product code | CCP-020 | ||||||||||||||||
Other name | |||||||||||||||||
Pharmaceutical forms | Ointment | ||||||||||||||||
Routes of administration | Topical use | ||||||||||||||||
Dosage and administration details | During the treatment period of each cycle, subjects/caregivers applied a thin layer of the assigned study drug, sufficient to cover the subject’s EBS lesions and approximately ¾ inch (2 cm) of surrounding uninvolved skin, and gently rubbed in the study drug. Subjects/caregivers applied the assigned study drug to all EBS lesions, including any new EBS lesions that developed (up to 30% BSA), once daily, every evening until the lesions resolved, for 8 weeks. | ||||||||||||||||
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| Baseline characteristics reporting groups | ||||||||||||||||||||||||||||||||||||||||
Reporting group title | Overall Trial | |||||||||||||||||||||||||||||||||||||||
Reporting group description | - | |||||||||||||||||||||||||||||||||||||||
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| End points reporting groups | |||
Reporting group title | Open Label | ||
Reporting group description | Each treatment cycle consisted of 8 weeks on treatment (once-daily, at-home study drug applications) followed by 8 weeks off treatment (only Investigator-approved, bland, non-medicated emollient/moisturizer, routine cleansing products, and sunscreens were allowed off treatment), with a maximum of 2 treatment cycles allowed for up to 52 weeks. | ||
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End point title | Treatment-Emergent Adverse Events [1] | ||||||||
End point description | Number and percentage of participants with any treatment-emergent adverse event | ||||||||
End point type | Primary | ||||||||
End point timeframe | Up to 52 weeks | ||||||||
| 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: The primary objective of this safety study was to describe treatment-emergent adverse events. | |||||||||
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| No statistical analyses for this end point | |||||||||
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| Adverse events information | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events | Up to 52 weeks | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type | Non-systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Dictionary used for adverse event reporting | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name | MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version | 19.0 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Reporting groups | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title | All study participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description | Safety population | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) | |||
| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) | |||
| Were there any global interruptions to the trial? No | |||
Limitations and caveats | |||
| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||