Clinical Trials Register

Summary
EudraCT Number:	2011-004783-30
Sponsor's Protocol Code Number:	ARD12858-MOZ23510
National Competent Authority:	Estonia - SAM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2013-01-15
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

Estonia - SAM

A.2

EudraCT number

2011-004783-30

A.3

Full title of the trial

A pilot, exploratory, randomized, phase 2 safety study evaluating tumor cell (plasma cell) mobilization and apheresis product contamination in plerixafor plus non-pegylated G-CSF mobilized patients and in non-pegylated G-CSF alone mobilized patients

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

An Exploratory Safety Study to Investigate the Extent of Tumor Cell Mobilization (TCM) After Use of G-CSF Alone or G-CSF Plus Plerixafor in Multiple Myeloma (MM) Patients Who May be Poor Mobilizers of Stem Cells

A.4.1

Sponsor's protocol code number

ARD12858-MOZ23510

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Sanofi aventis recherche et développement
B.1.3.4	Country	France
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	Sanofi aventis recherche et développement
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	sanofi-aventis Estonia OÜ
B.5.2	Functional name of contact point	Clinical Study Unit
B.5.3	Address:
B.5.3.1	Street Address	Pärnu mnt 139E/2
B.5.3.2	Town/ city	Tallinn
B.5.3.3	Post code	11317
B.5.3.4	Country	Estonia
B.5.4	Telephone number	+372 627 3496
B.5.5	Fax number	+372 627 3491
B.5.6	E-mail	clinicaltrialsinfo_ee@sanofi-aventis.com

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	Yes
D.2.1.1.1	Trade name	Mozobil 20 mg/ml solution for injection
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/227
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Plerixafor
D.3.9.1	CAS number	110078-46-1
D.3.9.2	Current sponsor code	GZ316455
D.3.9.3	Other descriptive name	PLERIXAFOR
D.3.9.4	EV Substance Code	SUB28849
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
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	No
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

Multiple Myeloma

E.1.1.1

Medical condition in easily understood language

Multiple Myeloma

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	17.0
E.1.2	Level	LLT
E.1.2	Classification code	10028228
E.1.2	Term	Multiple myeloma
E.1.2	System Organ Class	100000004864

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

To evaluate tumor cell mobilization (TCM) with non-pegylated G-CSF alone compared with non pegylated G-CSF plus plerixafor in patients with multiple myeloma (MM) who are potentially poor mobilizers of hematopoietic stem cells (HSC).

E.2.2

Secondary objectives of the trial

To evaluate survival and disease status of G-CSF alone compared with GCSF plus plerixafor.
To evaluate the efficacy and safety of G-CSF plus plerixafor when used to mobilize stem cells for autologous transplantation.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients with a diagnosis of MM in partial or complete response, who are to undergo an autologous HSCT and could be considered potentially poor mobilizers.
A potentially poor mobilizer is defined as a person who, in the judgment of the investigator prior to study entry, may not mobilize successfully for reasons such as prior lenalidomide use.

E.4

Principal exclusion criteria

The patient does not have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
The patient has a history of any acute or chronic leukemia (including myelodysplastic syndrome).
The patient had prior allogeneic or autologous transplantation.
The patient is less than 3 to 6 weeks since last anti-cancer therapy.
Chemotherapy for mobilization is not allowed. Note: Treatment with thalidomide, dexamethasone, lenalidomide (Revlimid®), and/or bortezomib (Velcade®) is allowed up to 7 days prior to the first dose
of G-CSF.
The patient has bone marrow involvement >10% assessed based on the most recent bone marrow aspirate or biopsy performed prior to first dose of G-CSF.
The patient was treated with G-CSF or other cytokine within 14 days prior to the first dose of GCSF
for mobilization.
The patient has previously received plerixafor.
The patient is known to be HIV positive.
The patient has active hepatitis B or hepatitis C.
The patient has an acute infection (febrile, ie, temperature >38°C) within 24 hours prior to dosing or antibiotic therapy within 7 days prior to the first dose of G-CSF.
The patient has hypercalcaemia as evidenced by >1 mg/dL above ULN.
The patient previously received investigational therapy within 4 weeks of screening in this protocol or currently enrolled in another investigational protocol during the mobilization phase.
The patient has central nervous system involvement including brain metastases or leptomeningeal disease.
The patient has an electrocardiogram (ECG) or study result (exercise study, scan) indicative of cardiac ischemia or a history of clinically significant rhythm disturbance (arrhythmias), or other
conduction abnormality in the last year that in the opinion of the investigator warrants exclusion of the subject from the trial.
The patient has co-morbid condition(s), which in the opinion of the investigator, renders the patient at high risk from treatment complications or impairs his/her ability to comply with the study treatment and protocol.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study
The patient has a white blood cell (WBC) count <2.5 x _109/L.
The patient has an absolute neutrophil count (ANC) <1.5 x _109/L.
The patient has a platelet count <100 x 109/L.
The patient has an estimated creatine clearance ≤50 mL/min.
The patient has aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT), and total bilirubin ≥2.5 x _upper limit of normal (ULN).
The patient does not have adequate cardiac, and pulmonary function sufficient to undergo apheresis and transplantation, ie, eligible by institutional standards for autologous stem cell
transplant.
Pregnant or breastfeeding women
The patient does not agree to use a highly effective method of contraception while on study treatment and for at least 3 months following study treatment (including both female patients of childbearing potential and male patients with partners of child-bearing potential). Effective birth control includes:
a) birth control pills, depot progesterone, or an intrauterine device plus one barrier method, or
b) two barrier methods. Effective barrier methods are: male and female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm). For patients using a hormonal contraceptive method, information about any interaction of plerixafor with hormonal contraceptives is not known.

E.5 End points

E.5.1

Primary end point(s)

Peripheral blood parameters:
1. The percentage of myeloma tumor cells/CD34+ cells
2. The percentage of myeloma tumor cells/plerixafor cumulative dose/kg body weights
3. The percentage of myeloma tumor cells/G-CSF cumulative dose/kg body weight
4.The change in TCM in the peripheral blood
Apheresis product parameters:
5. The number/volume of myeloma tumor cells per patient at each apheresis
6. The proportion of patients who mobilize at least 4.5x105 myeloma tumor cells/kg body weight as measured in each apheresis product

E.5.1.1

Timepoint(s) of evaluation of this end point

Peripheral blood parameters:

Variable Day 1 to Day 8 of the Apheresis/Treatment Period :
1. The percentage of myeloma tumor cells/CD34+ cells
5. The number/volume of myeloma tumor cells per patient at each apheresis

Variable Day 5 to Day 8 of the Apheresis/Treatment Period :
2. The percentage of myeloma tumor cells/plerixafor cumulative dose/kg body weights
3. The percentage of myeloma tumor cells/G-CSF cumulative dose/kg body weight
6. The proportion of patients who mobilize at least 4.5x105 myeloma tumor cells/kg body weight as measured in each apheresis product

Up to Day 5 pre-G-CSF Apheresis product parameters:
4.The change in TCM in the peripheral blood

E.5.2

Secondary end point(s)

- CD34+ stem cell yield in the apheresis product
- The proportion of patients that proceed to transplantation
- Overall survival

E.5.2.1

Timepoint(s) of evaluation of this end point

- CD34+ stem cell yield in the apheresis product : Variable Day 1 to Day 8 of the Apheresis/Treatment Period
- The proportion of patients that proceed to transplantation : Up to two months after final apheresis
- Overall survival : At day 100 post transplant and up to 2 years post first G-CSF dose

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

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

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

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

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

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the 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

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

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

LVLS

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

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

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

2013-02-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-08-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2016-09-28

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