Ignite Creation Date:
2025-12-24 @ 4:07 PM
Ignite Modification Date:
2025-12-25 @ 2:08 PM
Study NCT ID:
NCT03994666
Status:
UNKNOWN
Last Update Posted:
2019-06-21
First Post:
2019-06-04
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Cell Therapy in Critical Limb Ischemia
Sponsor:
CHU de Reims
Organization:
Study Overview
Official Title:
Cell Therapy in Critical Limb Ischemia by Implantation of Allogeneic Umbilical Cord-derived Mesenchymal Stem Cells
Status:
UNKNOWN
Status Verified Date:
2019-06
Last Known Status:
NOT_YET_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CLI
Brief Summary:
Study drug and dosage form : Umbilical cord-derived mesenchymal stem cells (HB-MSC1)
Dose and route of administration : 60 × 106 cells or 120 x 106 cells to be injected as 30 individual intramuscular injections, once at V0 within 48 hours to 2 weeks maximum after the revascularization procedure.
Comparator, dose and route of administration : Placebo, injected as 30 individual intramuscular injections, once at V2 within 48 hours to 2 weeks maximum after the revascularization procedure.
Study centers : 3 centers in France
Study objectives :
Primary: Evaluation of the feasibility and systemic and local tolerance of an implantation, via intramuscular route, of allogenic HB-MSC1, associated with a revascularization procedure, in patients suffering from critical limb ischemia (CLI).
Secondary: Preliminary evaluation of efficacy and dose effect relationship of the MSC implantation in hemodynamic, anatomical and functional terms.
Exploratory: Constitution of a serum bank of the patients included in the study for inflammation and auto immunity biomarkers analysis
Study design : This will be a multicenter Phase IIa study, consisting in a first, open-label, ascending dose feasibility and safety stage followed by a randomized placebo-controlled feasibility, safety and preliminary efficacy stage.
Dose and route of administration : 60 × 106 cells or 120 x 106 cells to be injected as 30 individual intramuscular injections, once at V0 within 48 hours to 2 weeks maximum after the revascularization procedure.
Comparator, dose and route of administration : Placebo, injected as 30 individual intramuscular injections, once at V2 within 48 hours to 2 weeks maximum after the revascularization procedure.
Study centers : 3 centers in France
Study objectives :
Primary: Evaluation of the feasibility and systemic and local tolerance of an implantation, via intramuscular route, of allogenic HB-MSC1, associated with a revascularization procedure, in patients suffering from critical limb ischemia (CLI).
Secondary: Preliminary evaluation of efficacy and dose effect relationship of the MSC implantation in hemodynamic, anatomical and functional terms.
Exploratory: Constitution of a serum bank of the patients included in the study for inflammation and auto immunity biomarkers analysis
Study design : This will be a multicenter Phase IIa study, consisting in a first, open-label, ascending dose feasibility and safety stage followed by a randomized placebo-controlled feasibility, safety and preliminary efficacy stage.
Detailed Description:
Demographics, baseline and follow-up characteristics: descriptive statistics. Primary analysis: Descriptive statistics for the rate of patients with high grade AEs (grade 2 and more), related to the cell implantation will be computed in each arm, with its two-sided 95% CIs.
Each dose arm will be compared to the control arm by computing the absolute difference and the relative risk between the active dose arm and the control arm, together with their 95% two-sided CIs.
The p-values for the comparisons of the difference between the observed absolute rate to 0 and of the relative risk to 1 will be computed, but it is not expected that the study will have sufficient power to detect a significant difference on either of the two statistics (absolute difference or relative risk).
Primary efficacy analysis: The primary efficacy criterion (TcPO2) will be compared between each dose group versus the control group to test for a superiority of MSC over control.
The tested hypotheses are:
* Null hypothesis: mean (MSC dose i) ≤ mean (Control)
* Alternative hypothesis: mean (MSC dose i) \> mean (Control) A mixed - model for repeated measures will be used to model the evolution of TcPO2 over time. The significance of the time by treatment interaction will be used to test the two hypotheses. Model adjusted means (LS means) and their differences between treatment arms will be computed at each timepoint.
The LS-Means for change from baseline to 180 days will be used as the primary efficacy endpoint.
Other efficacy analyses: the same analysis model (mixed - model for repeated measures) will be used for all the other continuous efficacy endpoints.
Each dose arm will be compared to the control arm by computing the absolute difference and the relative risk between the active dose arm and the control arm, together with their 95% two-sided CIs.
The p-values for the comparisons of the difference between the observed absolute rate to 0 and of the relative risk to 1 will be computed, but it is not expected that the study will have sufficient power to detect a significant difference on either of the two statistics (absolute difference or relative risk).
Primary efficacy analysis: The primary efficacy criterion (TcPO2) will be compared between each dose group versus the control group to test for a superiority of MSC over control.
The tested hypotheses are:
* Null hypothesis: mean (MSC dose i) ≤ mean (Control)
* Alternative hypothesis: mean (MSC dose i) \> mean (Control) A mixed - model for repeated measures will be used to model the evolution of TcPO2 over time. The significance of the time by treatment interaction will be used to test the two hypotheses. Model adjusted means (LS means) and their differences between treatment arms will be computed at each timepoint.
The LS-Means for change from baseline to 180 days will be used as the primary efficacy endpoint.
Other efficacy analyses: the same analysis model (mixed - model for repeated measures) will be used for all the other continuous efficacy endpoints.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: