Clinical Trials Register

Summary
EudraCT Number:	2021-004174-64
Sponsor's Protocol Code Number:	DMX-200-301
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2021-12-23
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

France - ANSM

A.2

EudraCT number

2021-004174-64

A.3

Full title of the trial

A pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis (FSGS) who are receiving an angiotensin II receptor blocker (ARB)

Étude pivot de phase III, multicentrique, randomisée, en double aveugle, contrôlée par placebo, évaluant l’efficacité et la sécurité d’emploi du DMX-200 chez des patients atteints de glomérulosclérose segmentaire et focale (FSGS) qui reçoivent un antagoniste des récepteurs de l’angiotensine II (ARA-II)

A.3.1

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

Efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis

A.3.2

Name or abbreviated title of the trial where available

ACTION3

A.4.1

Sponsor's protocol code number

DMX-200-301

A.5.4

Other Identifiers

Name:

US FDA PIND Number:

Number:

146154

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	Dimerix Bioscience Pty Ltd
B.1.3.4	Country	Australia
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	Dimerix Bioscience Pty Ltd
B.4.2	Country	Australia
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Dimerix Bioscience Pty Ltd
B.5.2	Functional name of contact point	Clinical Trials Information
B.5.3	Address:
B.5.3.1	Street Address	425 Smith Street
B.5.3.2	Town/ city	Melbourne
B.5.3.3	Post code	3065
B.5.3.4	Country	Australia
B.5.6	E-mail	info@dimerix.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	Yes
D.2.5.1	Orphan drug designation number	EMA/OD/103/18
D.3 Description of the IMP
D.3.1	Product name	Repagermanium
D.3.2	Product code	DMX-200
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	Repagermanium
D.3.9.2	Current sponsor code	DMX-200
D.3.9.4	EV Substance Code	SUB234202
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	120
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Focal segmental glomerulosclerosis

E.1.1.1

Medical condition in easily understood language

Focal segmental glomerulosclerosis

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10067757
E.1.2	Term	Focal segmental glomerulosclerosis
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10016832
E.1.2	Term	Focal & segmental glomerulosclerosis
E.1.2	System Organ Class	10038359 - Renal and urinary 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 evaluate the efficacy of DMX-200 in terms of urine PCR and eGFR slope in patients with FSGS who are receiving an ARB

E.2.2

Secondary objectives of the trial

- To evaluate the safety and tolerability of treatment with DMX-200 in patients with FSGS who are receiving an ARB
- To evaluate the effect of DMX-200 on kidney function parameters including proteinuria in patients with FSGS who are receiving an ARB

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Key Inclusion Criteria:
- A diagnosis of FSGS confirmed by kidney biopsy. NOTE: The biopsy can have been obtained at any time. Diagnosis of FSGS should be based on light microscopy with supportive findings on either electron microscopy or immunofluorescence analysis (preferably both) and the clinical history and disease course consistent with primary FSGS, genetic FSGS, or FSGS of undetermined cause.
- Must be receiving a stable dose of an ARB (irbesartan, losartan, valsartan, candesartan, olmesartan medoxomil, or azilsartan medoxomil) at the maximal tolerated dose and ≥50% of the maximum recommended dose per the product label for 6 weeks prior to Screening, or willing to transition to this treatment during the Titration and Stabilization period.
- If taking corticosteroids, the dosage must be stable for ≥4 weeks prior to Screening and during the Stabilization period, and patients must have no plan to change their treatment regimen during the study.
- If taking aldosterone inhibitors, mineralocorticoid receptor antagonists, direct renin inhibitors, or sodium-glucose co-transporter-2 inhibitors, the dose and regimen must be stable for ≥26 weeks prior to Screening and during the Stabilization period and patients must have no plan to change their treatment regimen during the study.
- Urine protein/creatinine ratio (PCR) >1.5 g/g (>169.5 mg/mmol) or 24-hour total protein >1.5 g/day based on 24-hour urine collection during Screening.
- Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 at Screening and ≥25 mL/min/1.73 m2 at the Qualification visit (Week −1).
- Seated blood pressure ≤160/100 mm Hg (mean of 3 values) at Screening and ≤140/90 mm Hg (mean of 3 values) at the Qualification visit (Week −1).
- Body mass index ≤40 kg/m2 at Screening.

E.4

Principal exclusion criteria

Key Exclusion Criteria:
- Has FSGS secondary to another condition.
- History of type 1 diabetes mellitus, or uncontrolled type 2 diabetes mellitus (defined as glycated hemoglobin >8%), or non-fasting blood glucose >10 mmol/L at Screening.
- History of lymphoma, leukemia, or any active malignancy within the past 2 years (except for basal cell or squamous cell carcinomas of the skin or cervical carcinoma in situ that have been resected and with no evidence of metastatic disease).
- History of jaundice, active hepatitis, or known hepatobiliary disease (except asymptomatic cholelithiasis).
- Documented history of heart failure (New York Heart Association Class III/IV) or a major adverse cardiac event within 12 weeks prior to Screening.
- Serum potassium levels >5.5 mmol/L at Screening.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2 × upper limit of normal at Screening.
- Treatment with immunosuppressant biological drugs, calcineurin inhibitors, cyclophosphamide, azathioprine, or mycophenolate mofetil within 26 weeks prior to Screening
- Patients with <80.0% compliance to ARB treatment during the Stabilization Period (Weeks −6 to −1) of the study, as confirmed at the Qualification visit.

E.5 End points

E.5.1

Primary end point(s)

1.Percent change in urine PCR (based on 24-hour urine collection) following treatment with DMX-200 compared with placebo
2.Slope of eGFR following treatment with DMX-200 compared with placebo

E.5.1.1

Timepoint(s) of evaluation of this end point

1. from Baseline to Week 35
2. from Baseline to Week 104

E.5.2

Secondary end point(s)

1. Incidence and severity of AEs following treatment with DMX-200 compared with placebo
2. Incidence of clinically significant changes in the safety profile of patients treated with DMX-200 compared with placebo, as measured by changes from baseline in clinical laboratory evaluations (hematology, coagulation, clinical chemistry, and urinalysis), ECGs, vital signs, and physical examinations
3. Proportion of responders and non-responders following treatment with DMX-200 compared with placebo, defined as:
- Complete response: 24-hour urine PCR reduction to <0.3 g/g [<33.9 mg/mmol]
- Modified partial remission (FPRE): 24-hour urine PCR reduction ≥40% from Baseline and <1.5 g/g [<169.5 mg/mmol]
- No response (failure to meet any response criteria)
4. Proportion of patients on treatment with DMX 200 compared with placebo that meet a composite endpoint of worsening in kidney function, as defined by the onset of kidney failure (initiation of chronic dialysis, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), a 30% decline in eGFR from Baseline, or death from kidney or cardiovascular causes

E.5.2.1

Timepoint(s) of evaluation of this end point

1 to 4. Throughout study duration

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

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

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

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

Hong Kong

Argentina

Brazil

Denmark

Korea, Republic of

New Zealand

Spain

Taiwan

United Kingdom

United States

France

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

The end of the study is defined as the date of the last visit of the last patient in the study globally.

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:

263

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

286

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 will not receive any additional treatment or medical care from the Sponsor after completion of the study because until DMX-200 is approved for patients with FSGS, post-study care should not differ from what is normally expected for patients with FSGS. If the study progresses after the first IA, an open-label extension study may, at the Sponsor’s discretion, be offered to patients who have completed the study.

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

2022-02-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-02-11

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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