Clinical Trials Register

Summary
EudraCT Number:	2015-005607-92
Sponsor's Protocol Code Number:	QUIDHF_v1.0_20160106
National Competent Authority:	Norway - NOMA
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2016-03-07
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

Norway - NOMA

A.2

EudraCT number

2015-005607-92

A.3

Full title of the trial

A Phase II randomized, placebo controlled, double-blind, multi-centre study to assess safety and efficacy of incremental doses of QGC001 in patients upon discharge from hospital admission for worsening chronic heart failure with left ventricular systolic dysfunction

A.3.1

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

An European study to assess safety and efficacy of a new drug in patients with worsening chronic heart failure

A.3.2

Name or abbreviated title of the trial where available

QUID-HF

A.4.1

Sponsor's protocol code number

QUIDHF_v1.0_20160106

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	QUANTUM GENOMICS
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	QUANTUM GENOMICS
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	EDDH - Fondation Transplantation
B.5.2	Functional name of contact point	Stéphanie Grojean
B.5.3	Address:
B.5.3.1	Street Address	Centre de Médecine Préventive, 2 rue du Doyen Jacques Parisot
B.5.3.2	Town/ city	Vandoeuvre le Nancy
B.5.3.3	Post code	54500
B.5.3.4	Country	France
B.5.4	Telephone number	0033 383501921
B.5.5	Fax number	0033383501929
B.5.6	E-mail	sgrojean@eddh.fr

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	AMINO BUTANE SULFONATE DISULFURE
D.3.2	Product code	QGC001
D.3.4	Pharmaceutical form	Capsule
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	QGC001
D.3.9.1	CAS number	648927-86-0
D.3.9.2	Current sponsor code	QGC001
D.3.9.3	Other descriptive name	QGC001
D.3.9.4	EV Substance Code	SUB33157
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	50
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	AMINO BUTANE SULFONATE DISULFURE
D.3.2	Product code	QGC001
D.3.4	Pharmaceutical form	Capsule
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	QGC001
D.3.9.1	CAS number	648927-86-0
D.3.9.2	Current sponsor code	QGC001
D.3.9.3	Other descriptive name	QGC001
D.3.9.4	EV Substance Code	SUB33157
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	250
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	Capsule
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule
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

Patient with worsening chronic heart failure with left ventricular systolic dysfunction

E.1.1.1

Medical condition in easily understood language

Patient with worsening chronic heart failure

E.1.1.2

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.0
E.1.2	Level	LLT
E.1.2	Classification code	10019279
E.1.2	Term	Heart failure
E.1.2	System Organ Class	100000004849

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of the study is to investigate the safety (blood pressure changes until Day 28) and efficacy (rate of decrease in N terminal pro-hormone B-type natriuretic peptide (NT-proBNP) of more than 30% from baseline to Day 28) of QGC001 up-titrated from 50 mg twice daily to a maximum of 500 mg twice daily.

E.2.2

Secondary objectives of the trial

Further exploratory objectives of the study are
•To assess the effects of these doses on a composite endpoint of death from any cause and hospitalization for worsening heart failure (WHF) at Day 28 and after drug discontinuation up to Day 35.
•To assess the effects of the respective dose levels, and of all doses combined, on changes in NT-proBNP and BNP, from baseline to Day 7, Day 14, Day 21, Day 28, and after drug discontinuation from day 28 to day 35
•To assess the changes in quality of life Minnesota Living with Heart Failure questionnaire score from randomisation to day 28.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

•Written informed consent will be obtained prior to any study procedure
•Adult male subjects and female subjects without childbearing potential ≤ 80 years
•Clinical diagnosis of WHF who were New York Heart Association (NYHA) class II III at least 3 months prior to emergency presentation to hospital.
•Eligible subjects must be enrolled short before discharge or within 8 days after discharge from an episode of decompensated HF (index event) defined as
o(a) an overnight stay in a hospital, emergency department, or medical observation facility with the capability of treating with intravenous medications and observing HF patients, or
o(b) an unscheduled outpatient visit to a HF management centre, where parenteral therapy is required for HF stabilization.
•Documented left ventricular ejection fraction (LVEF) ≤ 40% measured by any modality within the previous 6 months in the subject’s medical history
•Subjects must also have at least one local measurement of BNP level ≥ 400 pg/mL or NT-proBNP level ≥1600 pg/mL (preferred assay, local lab) at any time during the index hospitalization or before randomization.
•eGFR ≥ 60 mL/min/1.73 m2 (MDRD) at screening
•No decrease in eGFR greater than 20% from admission to the index hospitalisation and up to the randomisation day.
•Serum potassium ≤ 5.0 mmol/L at screening
•Systolic blood pressure ≥ 120 mmHg (average of 3 consecutive measurements)
•Prescribed to optimal pharmacologic therapy per “ESC guidelines for the diagnosis and treatment of acute and chronic heart failure 2012”, or based on the updated current clinical practice, unless contra-indicated or not-tolerated, and on a stable dose (e.g., recommended not more than double or decrease in dosage) for at least 30 days prior to enrolment.
•Taking oral loop diuretics at doses ≤ 250 mg furosemide daily (or equivalent)

E.4

Principal exclusion criteria

• Age > 80
• BMI > 35kg.m-2
• Patients who require the use of HF IV therapy or oral furosemide > 250 mg (or equivalent) at any time during the 48 hours immediately before randomisation.
• Patients with unstable angina, myocardial infarction, PTCA, coronary artery bypass graft, cerebral vascular accident, or transient ischemic attack within previous 3 months (90 days) before enrolment.
• Patients whose primary cause of heart failure is mitral or aortic valve disease or congenital heart disease or hypertrophic obstructive cardiomyopathy or infiltrative cardiomyopathy (e.g. amyloidosis, sarcoidosis) or myocarditis.
• Patients with persistent or permanent atrial fibrillation (AF) (per “ACC/AHA/ESC 2006 guidelines for the management of patients with AF”) within 3 months (90days) prior to enrolment
• Patients scheduled for Pacemaker (including ICD, CRT), Angioplasty, CABG or LVAD within the next 3 months
• Patients with documented chronic obstructive lung disease.
• eGFR < 60 mL/min/1.73 m2 (MDRD) at screening
• Decrease in eGFR greater than 20% from admission to the index hospitalisation and up to the randomisation day
•Serum potassium > 5.0 mmol/L at screening
•Systolic blood pressure < 120 mmHg or with signs or symptoms of hypotension
•symptomatic hypotension or orthostatic hypotension defined by a decrease of more than 30 mm Hg in the standing vs. sitting position at screening and at T0 of the D0 (before having taken the study medication)
•Insulin-requiring diabetic patients (including type 1 Diabetes)
•HbA1c ≥ 10% at screening visit
•History of angioneurotic edema
•Patients with a life expectancy of less than 12 months per physician judgment
•Patients involved in any concurrent clinical investigation
•Patients who take an investigational or non approved treatment
•Women of childbearing potential
•Patients with a prior cardiac transplant or patients currently on the list for cardiac transplantation

E.5 End points

E.5.1

Primary end point(s)

The Efficacy Primary endpoint will be the percentage of subjects with a relative decrease in NT proBNP (Central Lab) of more than 30% from baseline to Day 28.

The Safety Primary endpoint will be the blood pressure changes at each visit, compared to the baseline measure

E.5.1.1

Timepoint(s) of evaluation of this end point

for efficacy endpoint : at day 28 compared to Baseline
for safety endpoint : at each visit, compared to the baseline

E.5.2

Secondary end point(s)

The Efficacy Secondary endpoints will be

•Blood biochemistry, electrolytes, urinary osmolarity at Day 7, Day 14, Day 21, Day 28, Day 35
•Change in central lab values of NT-proBNP and BNP, at Day 7, Day 14, Day 21, Day 28, Day 35
•Changes in central lab values from baseline in selected biomarker levels (copeptin, apelin, PRA and others TBD) at Day 7, Day 14, Day 21, Day 28
•Death from any cause or readmission for worsening heart failure at Day 28 and Day 35
•Quality of life Minnesota Living with Heart Failure Score and D0 and Day 28.

E.5.2.1

Timepoint(s) of evaluation of this end point

at each visit, compared to the baseline

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

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

E.8.1.4

Double blind

Yes

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

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

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)

Patients completing the randomized trial must stop the study medication and will follow the current standard treatment for heart failure

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

2016-05-03

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-04-04

P. End of Trial
P.	End of Trial Status	Ongoing

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