Clinical Trial Results:
An exploratory, open-label, multicenter study to evaluate the safety and efficacy of a two-dose regimen of ATIR101, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment), in patients with a hematologic malignancy, who received a CD34-selected hematopoietic stem cell transplantation from a haploidentical donor
Summary
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EudraCT number |
2015-002821-20 |
Trial protocol |
BE DE PT HR |
Global end of trial date |
17 Dec 2018
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Results information
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Results version number |
v1(current) |
This version publication date |
02 Jan 2021
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First version publication date |
02 Jan 2021
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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 |
CR-AIR-008
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02500550 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Kiadis Pharma
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Sponsor organisation address |
Paasheuvelweg 25A, Amsterdam, Netherlands, 1105BP
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Public contact |
Clinical Trial Information, Kiadis Pharma Netherlands B.V., +31 203140250, clinicaltrials@kiadis.com
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Scientific contact |
Clinical Trial Information, Kiadis Pharma Netherlands B.V., +31 203140250, clinicaltrials@kiadis.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 |
25 Sep 2019
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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 |
17 Dec 2018
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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 of the study is to study the safety and efficacy of a repeat dose administration of ATIR101 in patients with a hematologic malignancy who received a T-cell depleted haploidentical HSCT.
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Protection of trial subjects |
The study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki (and all amendments thereof) and that are consistent with the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (Topic E6 [R1]) as well as the applicable regulatory requirements.
The investigator/sub-investigator was responsible for explaining the nature and purpose of the study as well as other study-related matters to patients and donors, using the written information, and for obtaining their full understanding and written consent to participate in the study at their own free will. No patient/donor was to be subjected to undue influence, such as compulsory enrollment into the study.
Informed consent had to be obtained prior to performing the first observations/examinations of the screening period (use of assessments which were performed before signing informed consent was subject of informed consent).
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
02 Sep 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 |
Canada: 7
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Country: Number of subjects enrolled |
Belgium: 5
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Country: Number of subjects enrolled |
United Kingdom: 2
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Country: Number of subjects enrolled |
Germany: 1
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Worldwide total number of subjects |
15
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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) |
15
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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 |
- | ||||||||||
Pre-assignment
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Screening details |
T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment). Two intravenous infusions with 2x10E6 viable T-cells/kg approximately 42 days apart (unless the second dose is reduced or halted for safety reasons). | ||||||||||
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 |
Not applicable
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Blinding used |
Not blinded | ||||||||||
Arms
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Arm title
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ATIR101 | ||||||||||
Arm description |
- | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
ATIR101
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection/infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
The first four ATIR101-treated patients were infused with two doses of 2.0×10e6 viable T-cells/kg (based on the patient weight at screening).
As decided in consultation with the IDMC, the next two patients were infused with a first dose 2.0×10e6 viable T-cells/kg and a second dose of 1.0×10e6 viable T-cells/kg. As recommended by the IDMC the remaining
patients were infused with a single ATIR101 dose of 2.0×10e6 viable T-cells/kg.
The first ATIR101 dose was infused at a median of 28.0 days (range 27-46) after the HSCT and the second ATIR101 dose (if given) was infused at a median of 73.5 days (range 71-77) after the HSCT.
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End points reporting groups
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Reporting group title |
ATIR101
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Reporting group description |
- |
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End point title |
incidence of grade III/IV acute GVHD up to 180 days after HSCT. [1] | ||||||
End point description |
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End point type |
Primary
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End point timeframe |
The primary endpoint was the incidence of grade III/IV acute GVHD up to 180 days after
HSCT. Therefore, the primary analysis was based on the data at 180 days after the HSCT.
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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: Overall in the study, four ATIR101-treated patients met the primary endpoint, grade III/IV acute GVHD within 180 days after HSCT: two (33.3%) in the double-dose group and two (22.2%) in the single-dose group |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Up to 1-year post HSCT
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Assessment type |
Systematic | ||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||
Dictionary version |
19
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Reporting groups
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Reporting group title |
ATIR101
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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 |