Clinical Trial Results:
Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
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Summary
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EudraCT number |
2014-003572-23 |
Trial protocol |
FR |
Global end of trial date |
20 Nov 2018
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Results information
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Results version number |
v1(current) |
This version publication date |
27 Nov 2019
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First version publication date |
27 Nov 2019
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Other versions |
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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 |
GCP#01.01.030
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02504619 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Gamida Cell
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Sponsor organisation address |
5 Nahum Hafzadi, Givat Shaul, Jerusalem, Israel, 9548401
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Public contact |
Clinical Trial department, Gamida Cell Ltd, 972 26595666, kelly@gamida-cell.com
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Scientific contact |
Clinical Trial department, Gamida Cell Ltd, 026595631 26595666, kelly@gamida-cell.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 |
15 Apr 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 |
20 Nov 2018
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
Assess the acute toxicities associated with the infusion of Omidubicel, formerly known as CordIn, within 24 hours post-infusion
Assess the proportion of patients with donor-derived engraftment at 42 days following transplantation
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Protection of trial subjects |
The protocol was reviewed by institutional review boards at the study sites and approved by relevant health authorities. Patients were enrolled after a formal informed consent process. The study was conducted within the principles of good clinical practice. An independent data monitoring committee was in place in order to review toxicity data in individual patients.
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Background therapy |
The Preparative Phase consisted of: Hydroxyurea: 30mg/kg/day orally on days -35 to -13 The conditioning regimen consisted of: Busulfan: 1mg/kg/dose IV q 6h on days -12 to -9 for 16 doses Thiotepa: 5mg/kg/day on days -8 and -7 Fludarabine: 40mg/m2/day on days -6, -5, -4, and -3 The GvHD prophylaxis regimen was: Mycophenolate Mofetil (MMF) beginning day –3 for at least 90 days and Cyclosporine: beginning day -3 to at least nine months post transplant | ||
Evidence for comparator |
N/A as single arm study | ||
Actual start date of recruitment |
04 Apr 2016
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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 |
United States: 1
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Worldwide total number of subjects |
1
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EEA total number of subjects |
0
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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) |
1
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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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 |
France was open for recruitment in June 2015 followed by the US where one patient was recruited in April 2016. | ||||||
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Pre-assignment
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Screening details |
2 subjects were assessed for eligibility. One subject was excluded for clinically significant iron overload. | ||||||
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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 | ||||||
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Arms
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Arm title
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CordIn | ||||||
Arm description |
Underwent hematopoietic stem cell transplantation with CordIn graft | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
CordIn
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Dispersion for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Dosage is administered once
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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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End points reporting groups
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Reporting group title |
CordIn
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Reporting group description |
Underwent hematopoietic stem cell transplantation with CordIn graft | ||
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End point title |
proportion of patients with donor-derived engraftment [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
42 days post transplant
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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: only 1 patient was evaluated. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of patients with acute toxicities associated with the infusion of CordIn [2] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
24 hours post transplant
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| Notes [2] - 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: only 1 patient was evaluated. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Cumulative incidence of transplant-related mortality | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
day 100 post-transplant
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| No statistical analyses for this end point | |||||||||
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End point title |
Event-free survival | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
day 100 post-transplant
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| No statistical analyses for this end point | |||||||||
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End point title |
Event-free survival | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
One year post-transplant
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| No statistical analyses for this end point | |||||||||
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End point title |
Overall survival | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
One year post-transplant
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of treatment free HbS ≤ 30% | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
One year post-transplant
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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-transplant
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Adverse event reporting additional description |
Grade 2/3 Infections & GvHD reported to 1 year post-transplant.
All common events post-transplant collected to day 42 post-transplant. Grade 3-4 non-serious AEs reported to 1 year post-transplant.
The list below includes grade ≥3 AEs only. Occurrences not collected (only highest grade over each specified period). Default number entered.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
21
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Reporting groups
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Reporting group title |
General
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Reporting group description |
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| Frequency threshold for reporting non-serious adverse events: 3% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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08 Dec 2014 |
Clarification on supportive care therapy for inclusion criteria |
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27 Mar 2015 |
Update of CBU selection and product release criteria. Removal of Conditioning regimen B
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24 Jun 2015 |
- Addition of SCD eligibility committee approval requirement
- Additional options for back-up stem cell source
- Additional eligibility criteria
- Clarification of infection monitoring, GvHD prophylaxis, eligibility criteria |
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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 | |||
