Clinical Trials Register

Summary
EudraCT Number:	2015-003610-25
Sponsor's Protocol Code Number:	CT-MT001-2-2015-1
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2015-10-05
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

Germany - BfArM

A.2

EudraCT number

2015-003610-25

A.3

Full title of the trial

A randomised, double-blind, placebo-controlled, parallel-design, multi-centre study to investigate the efficacy to reduce chemotherapy-induced neutropenia (CIN), effects on the haematopoietic system, safety and pharmacokinetics of Myelo001 in patients receiving adjuvant or neoadjuvant chemotherapy for the treatment of breast cancer

A.3.1

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

A test in female patients with breast cancer to probably reduce side effects on blood cells by the planned chemotherapy and to collect data for medical research and drug development on the safety, possible benefits and distribution of an experimental drug called Myelo001 in comparison to a standard treatment.

A.3.2

Name or abbreviated title of the trial where available

MyeloConcept

A.4.1

Sponsor's protocol code number

CT-MT001-2-2015-1

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02692742

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	Myelo Therapeutics GmbH
B.1.3.4	Country	Germany
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	Myelo Therapeutics GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Myelo Therapeutics GmbH
B.5.2	Functional name of contact point	Project Management
B.5.3	Address:
B.5.3.1	Street Address	Grossenhainer Strasse 227
B.5.3.2	Town/ city	Dresden
B.5.3.3	Post code	01129
B.5.3.4	Country	Germany
B.5.4	Telephone number	+4935121927312
B.5.5	Fax number	+4935121925493
B.5.6	E-mail	clinicaltrials@myelotherapeutics.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	Dicarbamine
D.2.1.1.2	Name of the Marketing Authorisation holder	ОАО Valenta Pharmatsevtika
D.2.1.2	Country which granted the Marketing Authorisation	Russian Federation
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	Myelo001
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Imidazolyl ethanamide pentandioic acid
D.3.9.3	Other descriptive name	VITAGLUTAM
D.3.9.4	EV Substance Code	SUB179202
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Chemotherapy-induced neutropenia (CIN)

E.1.1.1

Medical condition in easily understood language

Decrease in white blood cells, particularly so called neutrophils, responsible for the human defense against bacterial infections due to cancer treatment.

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10072806
E.1.2	Term	Chemotherapy toxicity attenuation
E.1.2	System Organ Class	10042613 - Surgical and medical procedures

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the efficacy of repeated doses of Myelo001 100 mg tablets
taken once per day (QD) per os (p.o.) to reduce chemotherapy induced
neutropenia (CIN) in patients receiving chemotherapy for the
treatment of breast cancer

E.2.2

Secondary objectives of the trial

To investigate:
Efficacy to reduce chemotherapy-induced myelosuppression
White blood cell (WBC) population changes
Pharmacokinetics (PK) of Myelo001
Safety parameters

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

To investigate:
1.) Pharmacokinetics (PK) of Myelo001
2.) Surrogate, correlative and predictive markers for hematologic changes due to Myelo001

E.3

Principal inclusion criteria

Eligible patients must meet ALL of the following inclusion criteria:
1. Female patient of any racial origin having fulfilled her 18th birthday on Visit 1 (Screening)
2. Histologically confirmed invasive breast cancer scheduled for neoadjuvant CTX or adjuvant CTX (patient with primary wound healing ([R0])
3. Already selected for neoadjuvant or adjuvant standard of care poly-chemotherapy regimen containing anthracyclines in combination with cyclophosphamide (with or without follow-up treatment with taxanes) defined as EC standard regimen
4. Risk of CTX induced FN ≤20% according to ASCO guidelines (2015)
5. More than 5 days remaining before the planned initiation of the 1st CTX cycle (d1/c1) to allow initiation of IMP on d-5/c1
6. Performance status Grade 0-1 (ECOG)
7. Echocardiography: No contraindication for the scheduled CTX
8. Haematologic and chemistry thresholds at baseline:
• Absolute neutrophil count (ANC) ≥2,000 cells/ mm3 (≥2.0 x 10^9/L)
• Platelet count ≥100,000/mm3 (≥100 x 10^9/L)
• Haemoglobin ≥10 g/dL
• Total bilirubin <1.5 x, AST, ALT <2.5 x upper limit of normal (ULN)
• Serum creatinine <2.0 mg/dL
9. Able to read, understand and willing to sign the informed consent form
10. Able to undergo the investigations and to follow the Visit schedule

E.4

Principal exclusion criteria

A patient will be excluded from participation in the trial, if one or more of the following exclusion criteria are identified:
1. Suspected allergy to Myelo001 or its excipients
2. Prior chemotherapy
3. Prior or concomitant treatment with radiotherapy
4. Currently on or scheduled for other immunomodulatory or immunosuppressive therapies (e.g. TNF inhibitors) for the first CTX cycle
5. Currently on or scheduled for other immunostimulatory or hematopoietic active therapies (e.g.G-CSF, GM-CSF) for the first CTX cycle
6. Currently on or scheduled for primary prophylaxis with antibiotics for the first CTX cycle
7. History of bone marrow transplantation or stem cell transplant
8. Administration of another IMP / medical device within 30 days prior to screening. Participation in non-interventional, national or international cancer registries is allowed
9. Already confirmed HIV, HBV or HCV infection
10. History of somatic disease/condition that may interfere with the
objectives of the study
11. Any other medical disease or clinical laboratory parameter outside the normal range and of clinical significance according to the investigator
12. Serious uncontrolled comorbidities
13. Pregnant or breast-feeding subject
14. Woman considered to be of childbearing potential who do not use highly effective birth control methods during the study

E.5 End points

E.5.1

Primary end point(s)

Primary variables (Proof of Concept):

1 Primary endpoint: Threshold area over the curve (AOC) of ANC: Area below the threshold line (ANC 2.0x10^9/L classified as grade 1 neutropenia) and above the individual ANC trajectory, in the study period of d1/c1 to d22/c1

2 Co-primary endpoint:
Threshold area over the curve (AOC3) of ANC: Area below the threshold line (ANC 1.0x10^9/L classified as grade 3 neutropenia) and above the individual ANC trajectory, in the study period of d1/c1 to d22/c1

3 Co-primary endpoint:
Duration of ANC < 1.0x10^9/L classified as grade 3 neutropenia in the study period of d1/c1 to d22/c1

E.5.1.1

Timepoint(s) of evaluation of this end point

Frequent blood counts in the study period between day1/cycle1 to day22/cycle1

E.5.2

Secondary end point(s)

1 Secondary variables Treatment success rate will fulfill all of the following criteria:
a) Neutropenia Grade ≤ 3
b) No need for G-CSF / GM-CSF rescue therapy
c) No early withdrawal (drop-out)
Evaluation: d1/c1 to d22/c1 or drop-out or until one day post administration of rescue medication, whichever occurs first. Treatment failures are subjects not reaching any of the three criteria
2 Threshold AOC of ANC: Area below the threshold line (ANC 0.5x10^9/L classified as grade 4 neutropenia) and above the individual ANC trajectory during study period of d1/c1 to d22/c1
3 Threshold AOC of lymphocytes: Area below the threshold line (classified as grade 1) and above the individual absolute lymphocyte count trajectory during study period of d1/c1-d22/c1
4 Threshold AOC of leukopenia: Area below the threshold line (classified as grade 1) and above the individual absolute leukocyte count trajectory during the study period of d1/c1 to d22/c1
5 Threshold AOC of thrombocytes: Area below the threshold line (classified as grade 1) and above the individual absolute thrombocyte count trajectory during study period d1/c1 to d22/c1
6 Rate of neutropenia grade 1 and higher; 3 and higher, 4, and ANC ≤0.1x 10^9/L
7 Duration of neutropenia grade 1 and higher; 3 and higher, 4, and ANC ≤0.1x 10^9/L
8 ANC at nadir
9 Time to ANC nadir (from start of chemotherapy)
10 Time to ANC recovery from grade 3 neutropenia, i. e. time from onset to time of reaching neutropenia grade ≤2 (ANC ≥ 1.5x10^9/L)
11 Time to ANC recovery from grade 4 neutropenia, i. e. time from onset to time of reaching neutropenia grade ≤2 (ANC ≥ 1.5x10^9/L)
12 Time to ANC recovery from profound neutropenia (ANC ≥ 0.1x10^9/L), i. e. time from onset to time of reaching neutropenia grade ≤2 (ANC ≥ 1.5x10^9/L)
13 Rate of patients with rescue therapy during study period d1/c1 to d22/c1
14 Rate of patients developing febrile neutropenia (body temperature ≥38.3°C by single tympanic or oral measurement) and ANC ≤0.5x 10^9/L (Grade 4) during study period d1/c1 to d22/c1
15 Rate of patients with CTX dose reduction and/or delay of CTX cycle 2

E.5.2.1

Timepoint(s) of evaluation of this end point

Various timepoints:
In the study period of d1/c1 to d22/c1
During entire study until EOS

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

Yes

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

Yes

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

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

multi-centre

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

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

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

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

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

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

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:

110

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

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

130

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. Continuation of standard medical care. However, the data and safety monitoring board (DSMB) may decide for a longer follow-up of patients if "non-unresolved" adverse events persist.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	Arbeitsgemeinschaft Gynäkologische Onkologie e.V. (AGO-B) - Sektion Brusterkrankungen
G.4.3.4	Network Country	Germany
G.4 Investigator Network to be involved in the Trial: 2
G.4.1	Name of Organisation	CESAR Central European Society for Anticancer Drug Research – EWIV
G.4.3.4	Network Country	Austria

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2015-12-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-12-09

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2017-11-20

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