Clinical Trials Register

Summary
EudraCT Number:	2006-005159-14
Sponsor's Protocol Code Number:	GC P#02.01.001
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2008-08-04
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2006-005159-14

A.3

Full title of the trial

: A Multi-Center, Multi-National, Historical Cohort Controlled Study to Evaluate Efficacy and Safety of Transplantation of StemEx, Umbilical Cord Blood (UCB) Stem and Progenitor Cells Expanded Ex Vivo, in Subjects with Hematologic Malignancies following Myeloablative Therapy

A.3.2

Name or abbreviated title of the trial where available

ExCELL

A.4.1

Sponsor's protocol code number

GC P#02.01.001

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

A.7

Trial is part of a Paediatric Investigation Plan

Information not present in EudraCT

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	GamidaCell-Teva Joint Venture Ltd.
B.1.3.4	Country	Israel
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support
B.4.2	Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation
B.5.2	Functional name of contact point

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Stemex
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.3	Other descriptive name	Cellule allogeniche da cordone ombelicale espanse exvivo
D.3.10	Strength
D.3.10.2	Concentration type	equal
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	Information not present in EudraCT
D.3.11.3.1	Somatic cell therapy medicinal product	Yes
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Information not present in EudraCT
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Information not present in EudraCT
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Hight-risk hemathological malignacies ( including AML,ALL,CML,NHL,HD,and MDS)with an indication for allogenic hematopoietic stem cell transplantation, to support hematopoietic ricostitution following myeloablative therapy

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	HLT
E.1.2	Classification code	10024291
E.1.2	Term	Leukaemias acute myeloid

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	HLGT
E.1.2	Classification code	10024324
E.1.2	Term	Leukaemias

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	HLT
E.1.2	Classification code	10024296
E.1.2	Term	Leukaemias chronic myeloid

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	HLGT
E.1.2	Classification code	10025322
E.1.2	Term	Lymphomas non-Hodgkin's unspecified histology

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Asses the effect of Stemex transplantation on overall 100 day mortality

E.2.2

Secondary objectives of the trial

Time from infusion of CB cells to overall mortality at 100 and 180 days. % of overall mortality at 180 days. % of subjects how developed acute GvHD grades III-IV. % of subjects with graft failure.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

a.Subject is a candidate for allogeneic SCT for treatment of one of the following hematologic malignancies.b. Age >12 and <55.
c. Availability of CBU that meets the following requirements:
c.1. Matched at 4, 5, 6/6 HLA class I and II (high resolution).
c.2. The CBU will have undergone volume reduction prior to cryopreservation and will have been preserved in two fractions, of which the larger (or equal) fraction contains a minimum of 1.0x107 TNC/kg (pre-thaw).
c.3. CBUs should be obtained from public CBBs where they are tested for infectious agents in compliance with the applicable local requirements and regulations.
d. Patients’ Performance score ≥70% by Karnofsky (age ≥13) or a Lansky Play-Performance scale of ≥70% (age < 13).
e. Patient has sufficient physiologic reserves including:
e.1. Left ventricular ejection fraction ≥40%.
e.2. Pulmonary function test demonstrating a corrected CO2 diffusion capacity ≥50% predicted.
e.3. Serum Creatinine <1.6 mg/dl.
e.4. Serum Bilirubin < 2.0 x upper limit of normal range.
e.5. SGPT (ALT) ≤ 2.0 x upper limit of normal range.
f. Patient must have at least one of the following back-up stem cell sources in case of engraftment failure:
f.1. Patient is willing to undergo BM harvest or peripheral blood progenitor cells (PBPC) collection for use in case of engraftment failure (for HD or NHL patients only).
f.2. Patient has a second CBU as a possible back up.
f.3. Patient’s haploidentical family member has been identified and agreed (by signing a written informed consent) to donate hematopoietic stem cells in case of engraftment failure.
g. Patient or guardian signs the written informed consent after being aware of the nature of the patients’ disease and willingly consents to the treatment program after being informed of alternative treatments, potential risks, benefits, and discomforts

E.4

Principal exclusion criteria

a. Less than twenty-one days have elapsed since the patient's last radiation or chemotherapy prior to screening (except Hydroxyurea).
b. HIV positive.
c. Pregnancy or lactation.
d. Uncontrolled bacterial, fungal or viral infection.
e. Patients with signs and symptoms of active central nervous system (CNS) disease.
f. Availability of appropriate related and willing stem cell donor, who is HLA-matched at 5 or 6/6 antigens.
g. Prior allogeneic cell transplant.
h. Allergy to bovine or to any product, which may interfere with the treatment.
i. Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor.

E.5 End points

E.5.1

Primary end point(s)

Overall 100 day mortality

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Gruppo storico

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

corte storica

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects
F.1 Age Range
F.1.1	Trial has subjects under 18	Yes
F.1.1.1	In Utero	No
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	No
F.1.1.3	Newborns (0-27 days)	No
F.1.1.4	Infants and toddlers (28 days-23 months)	No
F.1.1.5	Children (2-11years)	No
F.1.1.6	Adolescents (12-17 years)	Yes
F.1.2	Adults (18-64 years)	Yes
F.1.3	Elderly (>=65 years)	No
F.2 Gender
F.2.1	Female	Yes
F.2.2	Male	Yes
F.3 Group of trial subjects
F.3.1	Healthy volunteers	No
F.3.2	Patients	Yes
F.3.3	Specific vulnerable populations	Yes
F.3.3.1	Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2008-08-04.	Yes
F.3.3.2	Women of child-bearing potential using contraception	Yes
F.3.3.3	Pregnant women	No
F.3.3.4	Nursing women	No
F.3.3.5	Emergency situation	No
F.3.3.6	Subjects incapable of giving consent personally	No
F.3.3.7	Others	No
F.4 Planned number of subjects to be included
F.4.1	In the member state	6
F.4.2	For a multinational trial
F.4.2.1	In the EEA	21
F.4.2.2	In the whole clinical trial	100

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2008-04-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2008-05-21

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-01-13

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