Clinical Trials Register

Summary
EudraCT Number:	2010-023790-19
Sponsor's Protocol Code Number:	Monofer-ISS-Blooddonor-01
National Competent Authority:	Denmark - DHMA
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2011-08-02
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

Denmark - DHMA

A.2

EudraCT number

2010-023790-19

A.3

Full title of the trial

Et randomiseret, prospektivt, dobbeltblindt, komparativt, placebokontrolleret forsøg med intravenøs indgift af jernisomaltosid 1000 (Monofer®) via infusioner til bloddonorer med jernmangel

A.3.1

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

Sammenligneligt placebokontrolleret forsøg med jernisomaltosid (Monofer) til bloddonorer med jernmangel

A.3.2

Name or abbreviated title of the trial where available

Monofer-ISS-Blooddonor-01

A.4.1

Sponsor's protocol code number

Monofer-ISS-Blooddonor-01

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	Rigshospitalet, afd. 2034
B.1.3.4	Country	Denmark
B.3.1 and B.3.2	Status of the sponsor	Non-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	Pharmacosmos CRO
B.5.2	Functional name of contact point	Lars Lykke Thomsen
B.5.3	Address:
B.5.3.1	Street Address	Rørvangsvej 30
B.5.3.2	Town/ city	Holbæk
B.5.3.3	Post code	4300
B.5.3.4	Country	Denmark
B.5.4	Telephone number	+4559485935
B.5.5	Fax number	+4559485960
B.5.6	E-mail	llt@pharmacosmos.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	Monofer
D.2.1.1.2	Name of the Marketing Authorisation holder	Pharmacosmos A/S
D.2.1.2	Country which granted the Marketing Authorisation	Denmark
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	Monofer
D.3.4	Pharmaceutical form	Solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
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	Solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Forsøg med intravenøs indgift af jernisomaltosid 1000 (Monofer®) via infusioner til bloddonorer med jernmangel. Dobbelblindet, placebokontrolleret forsøg.

E.1.1.1

Medical condition in easily understood language

I dette forsøg ønsker man at undersøge, hvordan jern, der i stedet for at blive givet som tabletter, gives direkte i blodbanen, virker på bloddonorer.

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.0
E.1.2	Level	LLT
E.1.2	Classification code	10022974
E.1.2	Term	Iron deficiency anemia
E.1.2	System Organ Class	10005329 - Blood and lymphatic system disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

• At vurdere effekten af intravenøs jernisomaltosid 1000 (Monofer®) sammenlignet med placebo til kvindelige førstegangsdonorer med P-ferritin under 30 µg/l.

E.2.2

Secondary objectives of the trial

• At sammenligne ændringerne i Hb-koncentrationer fra baseline til lige inden anden donation
• At sammenligne antallet af forsøgspersoner, som ikke kan tåle 3 donationer på grund af cHb < LA, mellem de to grupper
• At sammenligne jernrelaterede parametre (Hb, serumjern, serumferritin, TfS og retikulocytter) mellem de to grupper
• Livskvalitet (spørgeskema, herunder Fatique Visual Numeric Scale og 5 valgte spørgsmål fra Fatique Severity Scale (FSS))
• Motionstolerance (to-punkts test på cykel)
• At sammenligne antallet af uønskede hændelser relateret til forsøgslægemidlet (AE'er/SAE'er/SUSAR'er) mellem forsøgspersoner, som får jernisomaltosid 1000 (Monofer®)-infusion, og forsøgspersoner, som får placebo-infusion.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Kvindelige førstegangsdonorer
2. Over 18 år
3. P-ferritin < 30 microgram/l
4. Personen er villig til at deltage efter informeret samtykke.

E.4

Principal exclusion criteria

1. Jernoverbelastning eller forstyrrelser i jernudnyttelsen (f.eks. hæmokromatose og hæmosiderose)
2. Kendt overfølsomhed over for et eller flere af hjælpestofferne i forsøgslægemidlet.
3. Forsøgspersoner med allergi i anamnesen.
4. Forsøgspersoner med aktiv astma inden for de sidste 5 år.
5. Dekompenseret levercirrhose og hepatitis (alaninaminotranferase (ALT) > 3 gange den øvre normalgrænse).
6. Aktive akutte eller kroniske infektioner (vurderet ved klinisk skøn), understøttet af leukocyttal (WBC) og C-reaktivt protein (CRP).
7. Rheumatoid arthritis med symptomer eller tegn på aktiv inflammation.
8. Forsøgspersoner, som er gravide eller ammer (For at udelukke graviditet skal kvinder være postmenopausale (mindst 12 måneder siden sidste menstruation) eller være steriliseret eller, for reproduktionsdygtige kvinder, anvende en af følgende præventionsmetoder i hele forsøgsperioden og i mindst 5 biologiske plasmahalveringstider for forsøgslægemidlet efter forsøgets afslutning: P-piller, spiral, injektionspræparat af gestagen med depotvirkning, subdermalt implantat, vaginalring og depotplastre.)
9. Deltagelse i et andet klinisk forsøg mindre end 5 halveringstider for det relevante forsøgslægemiddel inden screening.
10. Ubehandlet B12-vitamin- eller folatmangel.
11. Anden i.v. eller peroral jernbehandling inden for 4 uger før screeningsbesøget.
12. Behandling med erytropoietin inden for 4 uger før screeningsbesøget.

E.5 End points

E.5.1

Primary end point(s)

Ændring i Hb-koncentrationer fra baseline (inden første donation) til lige inden tredje donation.

E.5.1.1

Timepoint(s) of evaluation of this end point

Ca. 3 mdr.

E.5.2

Secondary end point(s)

• Ændring i Hb-koncentrationer fra baseline til lige inden anden donation
• Antallet af forsøgspersoner, som ikke kan tåle 3 donationer på grund af cHb < LA
• Ændring fra baseline i koncentrationer af serumferritin, serumjern, transferrinmætning (TfS), retikulocytter
• Livskvalitet (spørgeskema, herunder Fatique Visual Numeric Scale og 5 valgte spørgsmål fra Fatique Severity Scale (FSS))
• Motionstolerance (to-punkts test på cykel)
• Antallet af forsøgspersoner i hver randomiseringsgruppe, som oplever enhver uventet og alvorlig formodet bivirkning (SUSAR).

E.5.2.1

Timepoint(s) of evaluation of this end point

3-6 mdr.

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

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)

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

Yes

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

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

Yes

E.8.4

The trial involves multiple sites in the 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

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

Sidste forsøgspersons sidste besøg (Last Subject Last Visit) er den seneste af følgende datoer: enten datoen for sidste besøg af den sidste forsøgsperson, der gennemfører forsøget, eller datoen for modtagelse af det sidste datapunkt for den sidste forsøgsperson, der er nødvendigt til den statistiske analyse (dvs. de resultater vedrørende sikkerhed og effekt, der er grundlaget for lægelige afgørelser).

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	No
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)	No
F.1.2	Adults (18-64 years)	Yes
F.1.2.1	Number of subjects for this age range:	95
F.1.3	Elderly (>=65 years)	Yes
F.1.3.1	Number of subjects for this age range:	5
F.2 Gender
F.2.1	Female	Yes
F.2.2	Male	No
F.3 Group of trial subjects
F.3.1	Healthy volunteers	Yes
F.3.2	Patients	No
F.3.3	Specific vulnerable populations	No
F.3.3.1	Women of childbearing potential not using contraception	No
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	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

2011-08-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-08-24

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2012-03-19

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