Clinical Trial Results:
Pilot study of vulval lichen sclerosus treatment by adipose tissue associated with autologous platelet-rich plasma
Summary
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EudraCT number |
2016-003952-63 |
Trial protocol |
ES |
Global end of trial date |
12 Dec 2020
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Results information
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Results version number |
v1(current) |
This version publication date |
12 Feb 2022
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First version publication date |
12 Feb 2022
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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
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Sponsor protocol code |
LIQUENIA
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
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WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Instituto de Investigación Sanitaria La Fe de Valencia
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Sponsor organisation address |
Avenida Fernando Abril Martorell, Torre 106 A 7planta, 46026 València, , Valencia, Spain,
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Public contact |
Jose Maria Millan Salvador, Instituto de Investigación Sanitaria La Fe, investigacion_clinica@iislafe.es
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Scientific contact |
Jose Maria Millan Salvador, Instituto de Investigación Sanitaria La Fe, investigacion_clinica@iislafe.es
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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 |
02 Dec 2020
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
02 Dec 2020
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Global end of trial reached? |
Yes
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Global end of trial date |
12 Dec 2020
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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 |
Demonstrate the improvement in the elasticity of the fibrosis plaques of patients affected by vulval lichen sclerosus after two infiltrations of fat tissue and autologous platelet-rich plasma.
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Protection of trial subjects |
The reference study was conducted in Spain under the legal framework of Royal Decree 1090/2015. It has been performed in accordance with the Declaration of Helsinki on Ethical Principles for Medical Research Involving Human Subjects, adopted by the General Assembly of the World Medical Association (1996). In addition, the study has been conducted in accordance with the protocol, good clinical practice (GCP) in accordance with the guidelines of the international conference on harmonization (ICH) and regulatory requirements for participating institutions.
An appropriately performed informed consent has been used, in compliance with GCP according to ICH guidelines and approved by the CEIm of the Hospital Universitario y Politécnico La Fe. Prior to inclusion of subjects in the study, a copy of the CEIm-approved informed consent has been reviewed with the prospective participant, signed and dated. The investigator has provided a copy of each subject's signed informed consent form and has retained a copy in the subject's study file.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
06 Sep 2017
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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 |
Spain: 20
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Worldwide total number of subjects |
20
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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) |
19
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
• Start of patient inclusion: September 2017 • Completion of inclusion of patients: March 2018 • Patient follow-up time: 12 months. • Expected completion date of the last patient included: March 2019. | |||||||||||||||
Pre-assignment
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Screening details |
• Adult women between 18 and 70 years of age. • Patients with a clear clinical and/or histological diagnosis of lichen sclerosus (LE). • Moderate or severe involvement of the disease at the genital level. • Patients who have received topical treatment for at least three months with 0.05% clobetasol propionate. • Prior signed informed consent | |||||||||||||||
Period 1
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Period 1 title |
Overall Trial (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Single blind | |||||||||||||||
Roles blinded |
Subject | |||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Control | |||||||||||||||
Arm description |
maintenance treatment of topical corticosteroid therapy (clobetasol 0.05%) to be administered as per usual clinical practice. | |||||||||||||||
Arm type |
Normal treatment | |||||||||||||||
Investigational medicinal product name |
clobetasol
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Cream
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Routes of administration |
Topical use
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Dosage and administration details |
Reference treatment, dose and mode of administration: Clobetasol propionate 0.05%.
Route of administration: topical
Dosage: 1 application per day, twice a week.
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Arm title
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Treatment group | |||||||||||||||
Arm description |
2 infiltrations separated by three months will be applied by intra and subdermal injection in each vulvar half with identical amount of treatment composed of autologous fat tissue (10cc) enriched with autologous platelet-rich plasma (2cc). | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
Autologous adipose tissue
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Injection
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Routes of administration |
Injection
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Dosage and administration details |
Intra and subdermal injection in each vulvar half of 20cc of autologous fatty tissue + 4cc of autologous platelet-rich plasma.
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Baseline characteristics reporting groups
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Reporting group title |
Control
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Reporting group description |
maintenance treatment of topical corticosteroid therapy (clobetasol 0.05%) to be administered as per usual clinical practice. | ||||||||||||||||||||||||||||||||||||
Reporting group title |
Treatment group
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Reporting group description |
2 infiltrations separated by three months will be applied by intra and subdermal injection in each vulvar half with identical amount of treatment composed of autologous fat tissue (10cc) enriched with autologous platelet-rich plasma (2cc). | ||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Control
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Reporting group description |
maintenance treatment of topical corticosteroid therapy (clobetasol 0.05%) to be administered as per usual clinical practice. | ||
Reporting group title |
Treatment group
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Reporting group description |
2 infiltrations separated by three months will be applied by intra and subdermal injection in each vulvar half with identical amount of treatment composed of autologous fat tissue (10cc) enriched with autologous platelet-rich plasma (2cc). |
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End point title |
Elasticity of the fibrotic plaques in the affected vulvar area | ||||||||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
12 moths
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Statistical analysis title |
T-Test | ||||||||||||||||||
Statistical analysis description |
Categorical variables were described as absolute counts and percentages, while continuous variables were described as means and standard deviations. The efficacy analysis was based on the per-protocol principle—defined as all the randomized patients without major protocol violations that met the inclusion criteria who received the scheduled study medication and completed the efficacy measurements both at baseline and at 12 months. The baseline characteristics of both groups were compared,
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Comparison groups |
Control v Treatment group
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
superiority [1] | ||||||||||||||||||
P-value |
< 0.05 | ||||||||||||||||||
Method |
Wilcoxon (Mann-Whitney) | ||||||||||||||||||
Parameter type |
Mean difference (final values) | ||||||||||||||||||
Point estimate |
0.86
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Confidence interval |
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level |
95% | ||||||||||||||||||
sides |
1-sided
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lower limit |
0 | ||||||||||||||||||
upper limit |
- | ||||||||||||||||||
Variability estimate |
Standard deviation
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Notes [1] - For continuous variables, Student’s t tests were used for normal data or Mann–Whitney U tests were employed otherwise. Data symmetry was analyzed using the Shapiro–Wilk normality test. Mann–Whitney U tests were used for group comparisons. Histological changes were evaluated by using Wilcoxon matched-pairs signed-rank tests. All the statistical tests used to evaluate the treatment effects were 1-sided and were considered statistically significant when the P values were\0.05. All the stat |
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End point title |
Pain | ||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
12 moths
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Statistical analysis title |
t-Student | ||||||||||||||||||
Comparison groups |
Control v Treatment group
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
superiority | ||||||||||||||||||
P-value |
< 0.05 | ||||||||||||||||||
Method |
Chi-squared | ||||||||||||||||||
Parameter type |
Mean difference (final values) | ||||||||||||||||||
Point estimate |
0.077
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Confidence interval |
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level |
95% | ||||||||||||||||||
sides |
1-sided
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lower limit |
0 | ||||||||||||||||||
upper limit |
- |
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Adverse events information
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Timeframe for reporting adverse events |
The investigator recorded in the CRD all the adverse events that occurred in the patients who participated in the clinical trial. AEs were followed up by the investigator and documented on the CRF up to 28 days after the end of the treatment period.
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Adverse event reporting additional description |
The SAEs were notified by the investigator to the sponsor when he became aware of it
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Assessment type |
Systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
24.1
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Reporting groups
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Reporting group title |
Control
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Reporting group description |
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Reporting group title |
Treatment
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Reporting group description |
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Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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 | |||
Online references |
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http://www.ncbi.nlm.nih.gov/pubmed/35048150 |