Clinical Trials Register

Summary
EudraCT Number:	2022-003126-42
Sponsor's Protocol Code Number:	DT-DEC01-DMD-CT-Phase1/2
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2023-01-16
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

Poland - Office for Medicinal Products

A.2

EudraCT number

2022-003126-42

A.3

Full title of the trial

A Phase 1/2, Open-Label, Exploratory Clinical Trial to Evaluate the Safety and Efficacy of DT-DEC01 Therapy in Patients with Duchenne Muscular Dystrophy

Badanie kliniczne fazy 1/2 (otwarte, eksploracyjne) oceniające bezpieczeństwo i skuteczność terapii DT-DEC01 u pacjentów z dystrofią mięśniową Duchenne’a.

A.3.1

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

Clinical Trial (Phase 1/2 ) to Evaluate the Safety and Efficacy of DT-DEC01 Therapy in Patients with Duchenne Muscular Dystrophy

Badanie kliniczne fazy 1/2 oceniające bezpieczeństwo i skuteczność terapii DT-DEC01 u pacjentów z dystrofią mięśniową Duchenne’a

A.3.2

Name or abbreviated title of the trial where available

DEC for DMD

A.4.1

Sponsor's protocol code number

DT-DEC01-DMD-CT-Phase1/2

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	Dystrogen Therapeutics Technology Polska Spółka z Ograniczoną Odpowiedzialnością
B.1.3.4	Country	Poland
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	Dystrogen Therapeutics Technology Polska Spółka z Ograniczoną Odpowiedzialnością
B.4.2	Country	Poland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	MedPolonia Spółka z Ograniczoną Odpowiedzialnością
B.5.2	Functional name of contact point	Head of Clinical Trials
B.5.3	Address:
B.5.3.1	Street Address	Obornicka 262
B.5.3.2	Town/ city	Poznań
B.5.3.3	Post code	60-693
B.5.3.4	Country	Poland
B.5.6	E-mail	badania.kliniczne@medpolonia.pl

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	EU/3/18/2089
D.3 Description of the IMP
D.3.1	Product name	DT-DEC01 (MB^N/MB^DMD DEC)
D.3.2	Product code	DT-DEC01
D.3.4	Pharmaceutical form	Suspension for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intraosseous use
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
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)	Yes
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	Yes
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	Yes
D.3.11.3.5.1	CAT classification and reference number	Tissue-engineered product (EMA/H0005097)
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

Duchenne muscular dystrophy (DMD) is the most common inherited neuromuscular disease that mostly affects boys. DMD is a type of X-linked disease caused by a mutation of the gene at the Xp21 locus that encodes the dystrophin protein, resulting in a complete lack of this protein in the muscles of affected boys. DMD is characterized by skeletal and cardiac muscle degeneration. Most DMD-affected boys lose their ability to walk by the age of 10-14.

E.1.1.1

Medical condition in easily understood language

Duchenne muscular dystrophy (DMD) is the most common inherited neuromuscular disease that mostly affects boys. DMD is characterized by skeletal and cardiac muscle degeneration.

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

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

Phase 1:
Primary objectives include evaluation of safety by clinical observation of the incidence and severity of all treatment-related adverse events: Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESI) to assess the potential risks.
Phase 2:
Primary objectives include evaluation of safety by clinical observation of the incidence and severity of all treatment-related adverse events: AEs, SAEs and AESI and to assess the potential risks; and evaluation of the efficacy, based on the functional assessments of muscle strength and function adjusted to the stage of the disease including:
(a) for ambulatory patients: 6MWT; timed functions of NSAA: supine to rise and 10-meter walk/run)
(b) for non-ambulatory patients: PUL 2.0,
(c) for both, ambulatory and non-ambulatory patients: EMG assessment of duration of the MUP of the selected muscles.

E.2.2

Secondary objectives of the trial

Phase 1:
Secondary outcomes include evaluation of efficacy:
(a) for ambulatory patients: Six-Minute Walk Test (6MWT); NorthStar Ambulatory Assessment (NSAA): supine to rise and 10-meter walk/run), Performance of Upper Limb (PUL 2.0), assessment of grip strength;
(b) for non- ambulatory patients: PUL 2.0, assessment of grip strength;
(c) assessments for both ambulatory and non-ambulatory patients: electromyography (EMG) assessment of duration of motor unit potential (MUP) of the selected muscles, quality of life (QoL) assessment by Pediatric Outcomes Data Collection Instrument (PODCI) and overall treatment effect (OTE) assessment.
Phase 2:
Secondary outcomes include:
(a) for ambulatory patients: PUL 2.0, assessment of grip strength,
(b) for non-ambulatory patients: assessment of grip strength;
(c) for both, ambulatory and non-ambulatory patients: evaluation of efficacy, quality of life (QoL) assessment by PODCI and overall treatment effect (OTE) assessment.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Any subject meeting all of the following inclusion criteria and verified by the Investigator during the screening visit V0a will be considered acceptable for study inclusion:
1) Subject and his legal representative/parent(s)/legal guardian have understood and accepted to participate in the study according to all study procedures by signing the approved informed consent.
2) Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing.
3) Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to the biopsy of muscle tissue.
4) Subjects with progressive, symmetrical proximal muscle weakness of arms and legs.
5) Willingness and ability to comply with scheduled visits, tissue biopsy procedure under anesthesia, drug administration plan, laboratory tests, study restrictions, study procedures, and functional testing adapted to the stage of the disease.
6) Patients must be cleared by anesthesiologist for tissue biopsy and DT-DEC01 intraosseous injection procedures which will be performed under anesthesia (local anesthesia / general anesthesia / analgosedation).

E.4

Principal exclusion criteria

Any subject meeting any of the following exclusion criteria verified by the Investigator
during the screening visit V0a will be excluded from enrolment into the study:
1) Subject was exposed to any experimental therapy with the use of Advanced Therapy Medicinal Product (ATMP) or any other cell-based product within the last 12 months prior to screening and has known history of immune reaction to administered drug or Graft versus Host Disease (GvHD).
2) Subjects with known cognitive impairment or behavioral issues that would impede the ability to follow instructions.
3) Subject is positive for human immunodeficiency virus (HIV) 1 or 2, hepatitis B (HBV) or C (HCV*), cytomegalovirus (CMV*), toxoplasmosis* or syphilis at screening visit (V0a). *In case of positive or doubtful result of anti-CMV IgG and anti-Toxo IgG, anti-HCV total the final qualification decision can be made after confirming the negative Nucleic Acid Test (NAT) results in cell culture.
4) Subject has a history of any autoimmune disease.
5) Ongoing participation in any other therapeutic clinical trial.
6) Presence of pre-existing antibodies in the subject’s serum against the donor lymphocytes.
7) Subject has undergone an organ or bone marrow transplantation.
8) Change in systemic corticosteroid therapy (e.g. initiation of treatment; cessation of treatment; change in dose, schedule, or type of steroid) within 3 months prior to administration of the investigational product.
9) Any injury or procedure which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months should pass from injury date.
10) Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary and cardiac disease.
11) History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of the investigational product.
12) Ongoing chronic use of any agents with an immunomodulating (activating or suppressing) effect, such as, but not limited to, immunosuppressants or drugs related to immunotherapy (e.g. based on antibodies), chemotherapy or similar therapy affecting cell proliferation.
13) Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for inclusion into this study.

E.5 End points

E.5.1

Primary end point(s)

Phase 1: Safety Endpoints
1. The frequency of all adverse events related to the DT-DEC01 therapy including: the AEs (from skeletal muscle tissue collection visit (V0b) through Month 6 post DT-DEC01 administration timepoint), the SAEs from skeletal muscle tissue collection visit (V0b) through Month 12 post DT-DEC01 administration timepoint) and the AESI (by Month 1 post DT-DEC01 administration).
Phase 2: Safety Endpoints
1. The frequency of all adverse events related to the DT-DEC01 therapy including: the AEs (from skeletal muscle tissue collection visit (V0b) through Month 6 post DT-DEC01 administration timepoint), the SAEs from skeletal muscle tissue collection visit (V0b) through Month 12 post DT-DEC01 administration timepoint) and the AESI (by Month 1 post DT-DEC01 administration).
Phase 2: Efficacy Endpoints
1. Mean changes from baseline recorded in the functional assessments adjusted to the stage of the disease:
a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run,
b. for non-ambulatory patients: PUL 2.0
at month 1, 3, 6 and 12.
2. Mean changes from baseline in the EMG assessment of MUP duration of the selected muscles in both ambulatory and non-ambulatory patients at month 3, 6 and 12.

E.5.1.1

Timepoint(s) of evaluation of this end point

Study Evaluation Timepoints: V0a – screening visit, V0b – muscle biopsy visit, V1 – DT-DEC01 administration visit, V2 – hospital discharge, V3 – week 1 post administration visit, V4 – month 1 post administration visit, V5 – month 3 post administration visit, V6 – month 6 post administration visit, V7 – month 12 post administration visit
Phase 1 and Phase 2: Safety Endpoints
1. Adverse events: the AEs on visits V0b, V1, V2, V3, V4, V5, V6, the SAEs on visits V0b, V1, V2, V3, V4, V5, V6,V7 and the AESI on visits V1, V2, V3, V4.
Phase 2: Efficacy Endpoints
1. Functional assessments: on visits V0a, V4, V5, V6 and V7.
2. EMG assessment on visits V0a, V5, V6 and V7.

E.5.2

Secondary end point(s)

Phase 1: Efficacy Endpoints
1. Mean changes from baseline recorded in the functional assessments adjusted to the
stage of the disease:
a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, PUL 2.0, assessment of grip strength (by dynamometer).
b. for non-ambulatory patients: PUL 2.0, assessment of grip strength (by dynamometer).
at month 1, 3, 6 and 12.
2. Mean changes from baseline in the EMG assessment of MUP duration of the selected muscles in both ambulatory and non-ambulatory patients at month 3, 6 and 12.
3. Mean changes from baseline in QoL assessed by PODCI at month 1, 3, 6 and 12.
4. Evaluation of Overall Treatment Effect at month 1, 3, 6 and 12.
Phase 2: Efficacy Endpoints
1. Mean changes from baseline recorded in functional assessments (adjusted to the stage of the disease): a. for ambulatory patients: PUL 2.0, assessment of grip strength (by dynamometer),
b. for non-ambulatory patients: assessment of grip strength (by dynamometer).
at month 1, 3, 6 and 12.
2. Mean changes from baseline in QoL assessed by PODCI at month 1, 3, 6 and 12.
3. Evaluation of Overall Treatment Effect at month 1, 3, 6 and 12.

E.5.2.1

Timepoint(s) of evaluation of this end point

Study Evaluation Timepoints: V0a – screening visit, V0b – muscle biopsy visit, V1 – DT-DEC01 administration visit, V2 – hospital discharge, V3 – week 1 post administration visit, V4 – month 1 post administration visit, V5 – month 3 post administration visit, V6 – month 6 post administration visit, V7 – month 12 post administration visit

Phase 1:
Efficacy Endpoints
1. Functional assessments on visits V0a, V4, V5, V6 and V7.
2. EMG assessment on visits V0a, V5, V6 and V7.
3. QoL assessed on visits V0a, V4, V5, V6 and V7.
4. Overall Treatment Effect on visits V4, V5, V6 and V7.
Phase 2:
Efficacy Endpoints
1. Functional assessments on visits V0a, V4, V5, V6 and V7.
2. QoL assessed on visits V0a, V4, V5, V6 and V7.
3. Overall Treatment Effect on visits V4, V5, V6 and V7.

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

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)

Yes

E.7.1.1

First administration to humans

Yes

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

Evaluation of the initial safety and tolerance of the DT-DEC01

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

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

E.8.2.3

Other

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

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

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:

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)

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:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

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

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

Patients under 13 years of age.

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)

After completion of the clinical trial, patients will be subject to standard medical care.

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

2023-12-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-12-14

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

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