Clinical Trials Register

Summary
EudraCT Number:	2019-002627-16
Sponsor's Protocol Code Number:	IRL752C003
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-01-11
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

2019-002627-16

A.3

Full title of the trial

A randomised, placebo-controlled, multicentre phase IIb study evaluating the efficacy of pirepemat on falls frequency in patients with Parkinson’s disease

Randomizowane, kontrolowane placebo, wieloośrodkowe badanie fazy IIb oceniające skuteczność pirepematu w odniesieniu do częstości upadków pacjentów z chorobą Parkinsona

A.3.1

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

A study for evaluation of the efficacy of pirepemat on falls frequency in patients with Parkinson’s disease

Badanie oceniające skuteczność pirepematu na częstość upadków u
pacjentów z chorobą Parkinsona

A.4.1

Sponsor's protocol code number

IRL752C003

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	Integrative Research Laboratories Sweden AB (IRLAB)
B.1.3.4	Country	Sweden
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	Integrative Research Laboratories Sweden AB (IRLAB)
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Integrative Research Laboratories Sweden AB (IRLAB)
B.5.2	Functional name of contact point	Clinical Trial Information Desk
B.5.3	Address:
B.5.3.1	Street Address	Arvid Wallgrens Backe 20
B.5.3.2	Town/ city	Göteborg
B.5.3.3	Post code	413 46
B.5.3.4	Country	Sweden
B.5.4	Telephone number	+46 31 757 38 00
B.5.6	E-mail	info@irlab.se

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	Pirepemat, 50 mg
D.3.2	Product code	IRL752
D.3.4	Pharmaceutical form	Film-coated tablet
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	Pirepemat
D.3.9.2	Current sponsor code	IRL752
D.3.9.3	Other descriptive name	Pirepemat x fumarate
D.3.9.4	EV Substance Code	SUB217567
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	Pirepemat, 100 mg
D.3.2	Product code	IRL752
D.3.4	Pharmaceutical form	Film-coated tablet
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	Pirepemat
D.3.9.2	Current sponsor code	IRL752
D.3.9.3	Other descriptive name	Pirepemat x fumarate
D.3.9.4	EV Substance Code	SUB217567
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	100
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	Film-coated tablet
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

Parkinson’s disease

E.1.1.1

Medical condition in easily understood language

Parkinson’s disease

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

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	10061536
E.1.2	Term	Parkinson's disease
E.1.2	System Organ Class	10029205 - Nervous 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 evaluate the effects of pirepemat on falls frequency as compared to placebo.

E.2.2

Secondary objectives of the trial

To evaluate the effects of pirepemat on cognitive function as compared to placebo.
To evaluate the effects of pirepemat on postural dysfunction compared to placebo.
To evaluate the effects of pirepemat on Parkinson's disease motor symptoms as compared to placebo.
To evaluate the effects of pirepemat on apathy as compared to placebo.

Other objectives:
To evaluate the effects of pirepemat on Parkinson's disease symptoms and severity as compared to placebo.
To evaluate the effects of pirepemat on postural dysfunction as compared to placebo.
To evaluate the safety and tolerability of pirepemat

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female 55-85 years of age, inclusive.
2. Diagnosis of idiopathic Parkinson's disease, according to the UK Parkinson's disease Society Brain Bank criteria.
3. Montreal Cognitive Assessment (MoCA) score of ≥10 and <26 at screening.
4. A modified Hoehn & Yahr score of ≥2.5 in “on”.
5. Having experienced recurrent falls during the past 3 months (based on interview with the patient and/or caregiver) and at least 2 falls during the past 4 weeks before baseline.
6. On a stable regimen of anti-Parkinson's medications for at least 30 days prior to baseline, and willing to continue the same doses and regimens during study participation.
7. Able to cooperate and participate in study related procedures. This includes the ability to accurately complete a falls diary. The falls diary may also be completed by a responsible caregiver. For patients meeting DSM-IV TR criteria for Parkinson’s disease dementia, the falls diary should be completed by the caregiver.
8. Availability of a responsible caregiver at least five days per week at least 2 hours per day. For patients meeting DSM-IV TR criteria for Parkinson’s disease dementia, availability of a responsible live-in caregiver is required.
9. Female patients must be of non-childbearing potential (defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or post-menopausal females defined as 12 months of amenorrhoea [in questionable cases a blood sample with simultaneous follicle stimulation hormone (FSH) 25-140 IE/L and oestradiol <200 pmol/L is confirmatory]).
10. Fertile male patients must be willing to use condom and refrain from donating sperm during the study and until 3 months after last dosing of IMP and ensure that their fertile female partners are using contraceptive methods to prevent pregnancy1.
11. Written informed consent for participation in the study given by the patient and the responsible caregiver.

E.4

Principal exclusion criteria

1. Any of the following potential hepatic conditions:
a. known history of alcohol abuse, chronic liver or biliary disease, with the exception of Gilbert’s syndrome
b. total bilirubin greater than the upper limit of the normal range (unless associated with isolated instances of suspected Gilbert’s syndrome)
c. alkaline phosphatase (ALP) greater than 1.5 times the upper limit of the normal range
d. aspartate aminotransferase (AST) or alanine aminotransferase (ALT) greater than 2 times the upper limit of the normal range
e. history of repeated unexplained upper right quadrant abdominal pain and/or nausea, or jaundice
2. A positive Hepatitis B surface antigen or a positive Hepatitis C antibody result.
3. A score of 5 (wheelchair bound or bedridden) in the "on"-state on the modified Hoehn & Yahr scale.
4. Uncontrolled symptomatic orthostatic hypotension.
5. Clinically significant polyneuropathy.
6. Weight <55 kg at Screening.
7. Patients with current or past treatment with deep brain stimulation (DBS) or patients with previous history of stereotaxic brain surgery for PD.
8. A current diagnosis of any primary neurodegenerative disorder other than idiopathic PD.
9. A current diagnosis of any treatable dementia (hypothyroidism, syphilis, vitamin B12 or folate deficiency) that is verified by the investigator to be the cause of dementia.
10. A current diagnosis of a major depressive episode according to DSM-IV criteria.
11. Patient has delirium.
12. Any history of a heart condition, including prolonged QTc (>450 ms for males and > 470 ms for females, QTcF and/or QTcB), cardiac arrhythmias, any repolarisation deficits or any other clinically significant abnormal ECG as judged by the Investigator.
13. Severe or ongoing unstable medical condition including a history of poorly controlled diabetes; obesity associated with metabolic syndrome; uncontrolled hypertension; cerebrovascular disease, or any form of clinically significant cardiac disease; renal failure, history of abnormal renal function.
14. History of seizures within two years of screening.
15. History of cancer within five years prior to screening, with the following exceptions: adequately treated non-melanomatous skin cancers, localised bladder cancer, non-metastatic prostate cancer or in situ cervical cancer.
16. History of severe allergy/hypersensitivity or on-going allergy/hypersensitivity, as judged by the Investigator, or history of hypersensitivity to drugs with a similar chemical structure or class to pirepemat.
17. Creatinine clearance <30 mL/min (calculated according to the Cockroft-Gault formula).
18. Treatment with Warfarin within three months before study treatment.
19. Treatment with Amantadine within 6 weeks before study treatment.
20. Treatment with Selegiline within 6 weeks before study treatment.
21. Administration of another new chemical entity (defined as a compound which has not been approved for marketing) or has participated in any other clinical study that included drug treatment with less than three months between administration of last dose and first dose of IMP in this study.
22. Current or history of drugs of abuse according to DSM-IV criteria.
23. Any planned major surgery within the duration of the study.
24. Any other condition or symptoms preventing the patient from entering the study, according to the Investigator’s judgement.
Where the clinical significance of an abnormal Screening test result (lab or any other tests) is considered uncertain, the test may be repeated once, at the discretion of the Investigator, as long as the repeat test result is available within the 6 weeks screening period to determine eligibility.

E.5 End points

E.5.1

Primary end point(s)

Change in falls frequency from baseline period (4 weeks prior to randomisation) to the end of treatment visit as assessed by falls diary.

E.5.1.1

Timepoint(s) of evaluation of this end point

at every visit during the treatment period

E.5.2

Secondary end point(s)

Change in total MoCA score from baseline to Week 11.
Change in MDS-UPDRS item 3.12 (postural stability) from baseline to Week 11.
Change in the total score of MDS-UPDRS part 2 (M-EDL) from baseline to Week 11.
Change in total score (Frequency*Severity) and Caregiver distress of NPI Item G (Apathy/Indifference) from baseline to Week 11.

Safety objective:
As measured by subject incident of treatment-emergent adverse events, clinically significant changes in vital signs and physical examination clinical laboratory safety tests, and ECGs.

E.5.2.1

Timepoint(s) of evaluation of this end point

From baseline to Week 11:
MoCA - V1, V2, V5, V8
MDS-UPDRS - V1, V2, V5, V8
NPI - V2, V5, V8

Safety objective timepoints: every visit

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

E.8.1.4

Double blind

Yes

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

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

The end of the clinical part of the study is defined as the last visit of the last patient participating in the study.

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:

115

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

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

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

165

F.4.2.2

In the whole clinical trial

165

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)

The patients will continue on the standard of care for this indication.

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

2022-01-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-02-21

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

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