Clinical Trials Register

Summary
EudraCT Number:	2019-003140-71
Sponsor's Protocol Code Number:	RMFPC-22
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:	2020-04-20
Trial 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

2019-003140-71

A.3

Full title of the trial

Hemoglobin maintenance in pediatric ESRD patients by ferric pyrophosphate citrate (FPC)

Hämoglobinerhalt bei pädiatrischen ESRD-Patienten durch Eisenpyrophosphatcitrat (FPC).

A.3.1

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

Iron maintenance during hemodialysis in pediatric patients

Eisenerhalt bei pädiatrischen Patienten während der Hämodialyse

A.4.1

Sponsor's protocol code number

RMFPC-22

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/263/2018

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Rockwell Medical, Inc.
B.1.3.4	Country	United States
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	Rockwell Medical, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Rockwell Medical, Inc.
B.5.2	Functional name of contact point	Raymond D. Pratt
B.5.3	Address:
B.5.3.1	Street Address	30142 S. Wixom Rd
B.5.3.2	Town/ city	Wixom
B.5.3.3	Post code	MI 48393
B.5.3.4	Country	United States
B.5.4	Telephone number	+1 248-960-9000
B.5.6	E-mail	rpratt@rockwellmed.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	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	Triferic
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Ferric pyrophosphate citrate
D.3.9.1	CAS number	1802359-96-1
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	Iron(III) pyrophosphate citrate; soluble FePPi; ferric pyrophosphate, soluble; 1,2,3-Propanetricarboxylic acid, 2-hydroxy-, iron (3+), diphosphate, sodium salt
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	1.5
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.IMP: 2
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	Triferic
D.2.1.1.2	Name of the Marketing Authorisation holder	Rockwell Medical, Inc.
D.2.1.2	Country which granted the Marketing Authorisation	United States
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.3.4	Pharmaceutical form	Solution for haemodialysis
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Haemodialysis
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Ferric pyrophosphate citrate
D.3.9.1	CAS number	1802359-96-1
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	Iron (III) pyrophosphate citrate; soluble FePPi; ferric pyrophosphate, soluble; 1,2,3-Propanetricarboxylic acid, 2-hydroxy-, iron (3+), diphosphate, sodium salt
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	5.44
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

Iron deficiency anaemia

E.1.1.1

Medical condition in easily understood language

Anemia caused by iron deficiency

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10022972
E.1.2	Term	Iron deficiency anaemia
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

To assess the safety of Triferic administration via dialysate and IV in pediatric CKD-5HD patients

E.2.2

Secondary objectives of the trial

1. To assess the ability of Triferic to maintain haemoglobin in pediatric CKD-5HD patients
2. To explore the effect of FPC treatment on ESA dose and ferritin concentration
3. To explore the effect of FPC treatment to reduce the need for IV iron supplementation for development of iron deficiency anaemia

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients must meet all of the following criteria to be eligible for inclusion in the study:
1. Parents/legal guardians of the patient have the ability to understand the requirements of the study and have demonstrated a willingness to have their child comply with all study procedures by signing an institutional review board-approved informed consent form. Where applicable, assent of the patient has also been obtained for all study procedures prior to any study-related activities.
2. Patient is between 6 months and <18 years of age at screening.
3. Patient has chronic kidney disease receiving in-center hemodialysis or hemodiafiltration (HDF) at least twice weekly for at least 3 months prior to screening.
4. Patient is receiving adequate hemodialysis as assessed by the investigator and based on a single pool Kt/V measurement >1.2.
5. Patient has a vascular access (tunneled catheter, AV fistula or AV graft) suitable to support blood flows for hemodialysis treatment.
6. Patient has a body mass of ≥11 lbs (5 kg).
7. Patient is iron-replete as measured by a TSAT ≥ 20% and a ferritin >100 μg/L at screening.
8. Patient has a whole blood Hgb concentration of ≥ 9.5 g/dL at. screening.
9. If the patient is female, she must be pre-pubertal, have had documented surgical sterilization prior to Baseline admission, or be practicing adequate birth control. All female patients who have reached menarche must have a negative serum pregnancy test during screening. It is the investigator’s responsibility to determine whether the patient has adequate birth control for study participation.
10. Patients who have experienced a previous adverse event with IV iron products are eligible to participate in this study if the agent that caused the event is not administered during the Baseline period.

E.4

Principal exclusion criteria

A patient will not be eligible for inclusion in the study if any of the following criteria apply:
1. Patient is positive for human immunodeficiency virus (HIV) or hepatitis B by history.
2. Patient is receiving intravenous or oral antibiotics or antifungals for any infectious process. (Prophylactic antibiotics administered on a regular basis are allowed. Patients may enter the study once the infection has cleared.)
3. Patient has evidence of an ongoing active inflammatory process (e.g., systemic lupus erythematosus, acute or chronic active hepatitis, etc.) requiring treatment.
4. Patient has been dosed in an investigational drug study within the 30 days prior to Baseline.
5. Administration of iron containing phosphate binder ferric citrate (Aurixia®) or sucroferric oxyhydide (Velphoro®) within 2 weeks prior to Baseline. (Patient is only eligible if iron based binders are stopped at least 2 weeks prior to Baseline).

E.5 End points

E.5.1

Primary end point(s)

Incidence and severity of adverse events compared to Baseline.

E.5.1.1

Timepoint(s) of evaluation of this end point

Primary endpoint will be evaluated from Baseline to week 47.

E.5.2

Secondary end point(s)

1. Change from Baseline in Hgb concentration.
2. Change from Baseline in reticulocyte hemoglobin content (CHr).
3. Proportion of patients maintaining hemoglobin between 10.5 – 12.0 g/dL compared to Baseline.
4. Proportion of patients with stable or a reduction in erythropoietin dose compared to Baseline.
5. Proportion of patients requiring intravenous iron compared to Baseline.
6. The change from Baseline in iron parameters.
7. Safety parameters.

E.5.2.1

Timepoint(s) of evaluation of this end point

Secondary endpoints will be evaluated from base line to week 47.

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

historical controls

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

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

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

France

Germany

Italy

Poland

United Kingdom

United States

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

150

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

124

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

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 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 2020-04-20.

Yes

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

<18 year old participants

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

150

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 – Investigator should resume standard of care treatment as deemed necessary and appropriate.

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

2020-08-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-08-25

P. End of Trial
P.	End of Trial Status	Completed

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