Clinical Trial Results:
Mesenchymal stem cells for radiation-induced xerostomia (MESRIX) in previous HPV-positive oropharyngeal head and neck cancer patients
Summary
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EudraCT number |
2014-004349-29 |
Trial protocol |
DK |
Global end of trial date |
03 Jun 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
20 Aug 2021
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First version publication date |
20 Aug 2021
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Other versions |
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Summary report(s) |
Paper |
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 |
01-10-2014
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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 |
Rigshospitalet
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Sponsor organisation address |
Blegdamsvej 9, Copenhagen, Denmark,
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Public contact |
Rigshospitalet, Dept. of Otolaryngology, Head and Neck surgery, 0045 35452071, christian.von.buchwald@regionh.dk
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Scientific contact |
Rigshospitalet, Dept. of Otolaryngology, Head and Neck surgery, 0045 35452071, christian.von.buchwald@regionh.dk
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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 Jun 2017
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
01 Jun 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
03 Jun 2017
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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 is to examine whether enrichment of the submandibular gland with injection of autologous ASC will improve the result of salivary function in radiation-induced gland hypofunction.
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Protection of trial subjects |
None
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
01 Jan 2015
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Long term follow-up planned |
Yes
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Long term follow-up rationale |
Safety, Efficacy, Ethical reason, Regulatory reason, Scientific research | ||
Long term follow-up duration |
2 Years | ||
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 |
Denmark: 33
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Worldwide total number of subjects |
33
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EEA total number of subjects |
33
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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) |
23
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From 65 to 84 years |
10
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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 |
Eligibility criteria included previous radio- therapy, with or without concomitant chemotherapy, for a human papilloma virus-positive, T1-T2, and N0, N1, or N2A oropharyngeal squamous cell carcinoma; (12) two years of follow-up without disease progression; an unstimulated whole saliva flow rate in the range of 0.05 to 0.20 ml/min, correspon | ||||||||||||||||||
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 |
Double blind | ||||||||||||||||||
Roles blinded |
Subject, Investigator | ||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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ASC arm | ||||||||||||||||||
Arm description |
Intervention | ||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||
Investigational medicinal product name |
Mesenchymal stem cells
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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 |
injection of 2.8 million ASCs/cm3 pr gland
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Arm title
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Placebo | ||||||||||||||||||
Arm description |
- | ||||||||||||||||||
Arm type |
Placebo | ||||||||||||||||||
Investigational medicinal product name |
saline with 1% human albumin
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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 |
2 ml in each submandibular gland
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Baseline characteristics reporting groups
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Reporting group title |
ASC arm
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Reporting group description |
Intervention | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Experimental arm
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Subject analysis set type |
Per protocol | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Subjects randomized to experimental arm
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Subject analysis set title |
Placebo
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Subject analysis set type |
Per protocol | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Subjects randomized to placebo arm
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End points reporting groups
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Reporting group title |
ASC arm
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Reporting group description |
Intervention | ||
Reporting group title |
Placebo
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Reporting group description |
- | ||
Subject analysis set title |
Experimental arm
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
Subjects randomized to experimental arm
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Subject analysis set title |
Placebo
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
Subjects randomized to placebo arm
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End point title |
Safety | |||||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
4 months
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Statistical analysis title |
Primary endpoints | |||||||||||||||
Statistical analysis description |
For salivary flow rates, patient-reported outcome measures,
and tissue type analysis we evaluated the changes from
baseline to the one- and four-month follow-up visits.
Within-group comparisons were performed with the Wil-
coxon signed-rank test, and between-group comparisons
were performed with the Mann-Whitney U test. We chose
to use non parametric statistics as the data were not nor-
mally distributed, evaluated by Shapiro-Wilks tests.
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Comparison groups |
ASC arm v Placebo
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Number of subjects included in analysis |
30
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Analysis specification |
Pre-specified
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Analysis type |
superiority | |||||||||||||||
P-value |
< 0.05 | |||||||||||||||
Method |
Wilcoxon (Mann-Whitney) | |||||||||||||||
Confidence interval |
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End point title |
Unstimulated whole saliva flow | ||||||||||||
End point description |
Unstimulated whole salivary flow rates significantly increased in the ASC-arm at one (33%; P Z .048) and four months (50%; P Z .003), but not in the placebo-arm (P Z .6 and P Z .8), compared to baseline.
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End point type |
Secondary
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End point timeframe |
4 months
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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 |
4 months
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Assessment type |
Systematic | ||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||
Dictionary version |
10
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Reporting groups
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Reporting group title |
All patients
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Reporting group description |
- | ||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||
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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: There were no. |
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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 |