Clinical Trials Register

Summary
EudraCT Number:	2017-003598-33
Sponsor's Protocol Code Number:	LJ401-HH01
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-12-20
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

UK - MHRA

A.2

EudraCT number

2017-003598-33

A.3

Full title of the trial

A Phase 2, Multi-center, Randomized, Placebo-controlled, Single-blind Study with LJPC-401 for the Treatment of Iron Overload in Adult Patients with Hereditary Hemochromatosis

A.3.1

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

A multi-center clinical study with LJPC-401 for the treatment of iron overload in adult patients with hereditary hemochromatosis

A.4.1

Sponsor's protocol code number

LJ401-HH01

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03395704

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	La Jolla Pharmaceutical Company
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	La Jolla Pharmaceutical Company
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	La Jolla Pharmaceutical Company
B.5.2	Functional name of contact point	Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	4550 Towne Centre Ct.
B.5.3.2	Town/ city	San Diego
B.5.3.3	Post code	92121
B.5.3.4	Country	United States
B.5.4	Telephone number	+1 858 433 6908
B.5.6	E-mail	lajollaregulatoryaffairs@ljpc.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	LJPC-401
D.3.2	Product code	LJPC-401
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	Hepcidin-25 (human)
D.3.9.1	CAS number	342809-17-0
D.3.9.2	Current sponsor code	hepcidin-25 acetate, Synthetic human hepcidin
D.3.9.3	Other descriptive name	HEPCIDIN-25
D.3.9.4	EV Substance Code	SUB192682
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
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	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	LJPC-401
D.3.2	Product code	LJPC-401
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	Hepcidin-25 (human)
D.3.9.1	CAS number	342809-17-0
D.3.9.2	Current sponsor code	hepcidin-25 acetate, Synthetic human hepcidin
D.3.9.3	Other descriptive name	HEPCIDIN-25
D.3.9.4	EV Substance Code	SUB192682
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	10
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 injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Hereditary Hemochromatosis

E.1.1.1

Medical condition in easily understood language

Iron overload caused by the body making lower than normal amounts of the hormone hepcidin

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10027433
E.1.2	Term	Metabolism and nutrition disorders
E.1.2	System Organ Class	10027433 - Metabolism and nutrition disorders

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 compare the effect of weekly dosing of LJPC-401 versus placebo on Transferrin saturation (TSAT) in adult patients with hereditary hemochromatosis

E.2.2

Secondary objectives of the trial

- To compare the effect of LJPC-401 versus placebo on phlebotomy requirements
- To establish the safety and tolerability of LJPC-401 versus placebo in adult patients with hereditary hemochromatosis
- To compare the effect of LJPC-401 versus placebo on serum ferritin

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Patients with a clinical diagnosis of hereditary hemochromatosis.
2. Patients who are prescribed therapeutic phlebotomy for the treatment of hereditary hemochromatosis.
3. Patients ≥ 18 years of age.
4. Patients with a serum ferritin level from >100 ng/mL.
5. Patients with a TSAT level > 45%.
6. Female patients must be of non-childbearing potential, or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. Refer to Protocol Section 10.1.6.4.1
for the list of highly effective methods of contraception that can be used in this study.
7. Female patients of child bearing potential must have a negative serum pregnancy test at Screening (within 30 days prior to the first dose of study drug) and negative urine pregnancy test at Day 1 (ie, prior to initial dosing of study drug).
8. Male patients must be surgically sterile (vasectomy), or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. Refer to Protocol Section 10.1.6.4.1 for the list of highly effective methods of contraception that can be used in this study.
9. Patient must be willing and able to provide written informed consent.

E.4

Principal exclusion criteria

1. Patients receiving iron chelation therapy within 7 days prior to the
first dose of study drug.
2. Patients recently diagnosed, who have initiated phlebotomy treatments less than 3 months prior to the first dose of study drug.
3. Patients with a concomitant disease, disability or condition, including laboratory abnormality and ECG findings, which may interfere with the conduct of the study, or which would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this study, including, but not limited to clinically significant arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease. Patients with depression are eligible if receiving a
stable dose of medication for at least 90 days prior to the first dose of
study drug.
4. Pregnant or lactating women.
5. Patients taking an immunosuppressive agent that has not been
approved for use by La Jolla (excluding topical over-the-counter steroids, and nonsteroidal anti-inflammatory drugs). Refer to Section 7.1 for the list of prohibited medications and procedures.
6. Patients participating in an unapproved investigational drug or investigational therapeutic device study within 30 days prior to study drug dosing, ie, there must be at least 30 days in between the last dose on a prior study and the first dose administration on this trial.
7. Patients who are unwilling or unable to comply with the study protocol requirements.
8. Patients with type 1 diabetes or type 2 diabetes with haemoglobin Alc greater than 8% within 2 months prior to randomisation.
9. Patients with uncontrolled active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.
10. Patients with Child Pugh class C cirrhosis or liver failure.
11. Patients with severe congestive heart failure (NYHA = Class 4).
12. Patients who have advanced renal failure with an estimated glomerular filtration rate (eGFR) < 15 mL/min/1.73m2.
13. Patients with a history of allergic reaction to hepcidin or excipients.
14. Patients who have planned surgery (excluding dental surgery or simple dermatologic procedures) during participation in this study.

E.5 End points

E.5.1

Primary end point(s)

Change in TSAT defined as follows:
− For patients with 1 phlebotomy (the standard of care therapeutic
phlebotomy on Day 1 [predose]), change in TSAT from baseline to Week
16 (24 [± 4] hours postdose).
− For patients undergoing 2 or more phlebotomies, change in TSAT from baseline to the most recent postdose fasting TSAT observed prior to the second phlebotomy.

E.5.1.1

Timepoint(s) of evaluation of this end point

At screening, weekly from Week 1 to Week 16

E.5.2

Secondary end point(s)

• Number of phlebotomies Day 2 to End of Study (EOS)/30 (+ 3) days
after the last dose of study drug.
• Change in serum ferritin defined as follows:
− For patients with 1 phlebotomy (the standard of care therapeutic
phlebotomy on Day 1 [predose]), change in serum ferritin from baseline
to Week 16 (24 [± 4] hours postdose).
− For patients undergoing 2 or more phlebotomies, change in serum
ferritin from baseline to the most recent postdose serum ferritin
observed prior to the second phlebotomy.
• Adverse events (AEs) from consent to EOS
• Changes in clinical laboratory evaluations including serum iron parameters from baseline to EOS
• Changes in vital signs, electrocardiograms (ECGs), use of concomitant medications, and physical examinations from baseline to EOS
• Immunogenicity (anti-drug antibody)/pharmacokinetics (PK) from baseline to EOS

E.5.2.1

Timepoint(s) of evaluation of this end point

At screening, weekly from Week 1 to Week 16 and at EOS

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

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

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

Yes

E.8.1.2

Open

E.8.1.3

Single blind

Yes

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

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

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

Australia

France

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

Last Patient Last Visit

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

2018-02-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-04-03

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2019-10-28

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