Clinical Trial Results:
Phase II study of nilotinib efficacy in Pigmented Villo-Nodular Synovitis / Tenosynovial Giant Cell Tumor (PVNS / TGCT)
Summary
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EudraCT number |
2010-018869-29 |
Trial protocol |
FR IT NL GB |
Global end of trial date |
04 Oct 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
20 Oct 2019
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First version publication date |
20 Oct 2019
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Other versions |
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Summary report(s) |
PVNS |
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 |
ET2009-095
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01261429 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Centre Léon Bérard
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Sponsor organisation address |
28 rue Laennec , LYON Cedex 08, France,
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Public contact |
Centre Léon Bérard
S.GUILLEMAUT
, Centre Léon Bérard
S.GUILLEMAUT
, + 4 78 78 28 28, DRCIreglementaire@lyon.unicancer.fr
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Scientific contact |
Centre Léon Bérard
J.Y BLAY
, Centre Léon Bérard
J.Y BLAY
, +33 4 78 78 28 28, DRCIreglementaire@lyon.unicancer.fr
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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 2014
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
04 Oct 2013
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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 primary objective of the study will be to determine the efficacy of 12 weeks (3 months) of nilotinib treatment as measured by the non progression rate (Complete response + Partial Response + Stable disease according to Response Evaluation Criteria In Solid Tumours - RECIST version 1.1) in patients with progressive or relapsing PVNS/TGCT who cannot be treated by surgery.
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Protection of trial subjects |
Several follow-up (consultation with physician)
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
15 Dec 2010
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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 |
Australia: 7
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Country: Number of subjects enrolled |
Netherlands: 16
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Country: Number of subjects enrolled |
France: 25
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Country: Number of subjects enrolled |
Italy: 8
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Worldwide total number of subjects |
56
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EEA total number of subjects |
49
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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) |
56
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
The inclusion will take during a follow-up consultation of the patient by her oncologist. The investigator will verify the eligibility of the patient, inform him about the study and collect him consent to participation. | ||||||
Pre-assignment
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Screening details |
Patients will be selected among those contacting the study centre for the treatment of PVNS/TGCT according to the inclusion and non-inclusion criteria described above. After being informed of the study and having asked all their questions to the investigator, they will have enough time to decide whether or not they want to be included in the study. | ||||||
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 |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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Treatment with Nilotinib | ||||||
Arm description |
- | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Nilotinib
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Coated tablet
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Routes of administration |
Oral use
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Dosage and administration details |
400 mg twice a day
Oral administration
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Baseline characteristics reporting groups
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Reporting group title |
overall trial
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
final statistical analysis
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Subject analysis set type |
Intention-to-treat | |||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All included patients were analyzed for baseline characteristics and efficacy data. Since all these patients have received at least one dose of the study drug, they were also all analyzed for safety data.
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End points reporting groups
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Reporting group title |
Treatment with Nilotinib
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Reporting group description |
- | ||
Subject analysis set title |
final statistical analysis
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Subject analysis set type |
Intention-to-treat | ||
Subject analysis set description |
All included patients were analyzed for baseline characteristics and efficacy data. Since all these patients have received at least one dose of the study drug, they were also all analyzed for safety data.
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End point title |
Endpoint analysis [1] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
The primary endpoint of the study was the 12-week progression-free rate (12w-PFR).
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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: There is only one arm, so it is not possible to have a statistical analysis. |
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No statistical analyses for this end point |
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End point title |
Endpoint analysis | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Secondary endpoints of the study were: 24w-PFR, best overall response (BOR), objective response rate (ORR), duration of response (Drep), progression-free survival (PFS), time to progression (TTP), time to treatment failure (TTF).
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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 |
The investigator immediately informs the sponsor of any serious adverse events occurring during the study in a written report, whether or not they are attributable to the research.
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Assessment type |
Systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
21.0
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Frequency threshold for reporting non-serious adverse events: 1% | |||
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: Adverse events concerned 55/56 patients (98.2%); 54 patients (96.4%) experienced treatment-related adverse events and 6 patients (10.7%) experienced grade 3-4 treatmentrelated adverse events. Serious Adverse Events (SAEs) concerned 3 patients among the 56 included in the study, including one patient not directly concerned (particular case, the wife of the patient was pregnant). Only 1 AE was considered as potentially related to the drug according to the sponsor. No death was 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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01 Apr 2011 |
- to prolong the inclusion period by one year ;
- to add the calcium dosage for each biological test.
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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 |