Trial Outcomes & Findings for A Clinical Study Evaluating Efficacy of Pirepemat on Falls Frequency in Patients With Parkinson's Disease (PD) (NCT NCT05258071)

Last Updated: 2026-07-09

Results Overview

Falls being recorded using a paper fall diary (Patient Reported Outcome, PRO). Change in falls is reported as relative fall rate (fall rate at evaluation period / fall rate at baseline period). The baseline period is defined as the period with complete fall diary data (for at least 4 weeks, i.e. 28 days) prior to first dose day. The evaluation period is defined as the last 28 days during which the participant was taking full dose treatment.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

104 participants

Primary outcome timeframe

Baseline to end of full dose treatment (up to Day 84).

Results posted on

2026-07-09

Participant Flow

Recruitment was open from Jun-2022 to Sep-2024 at 38 trial centers across France, Germany, Poland, Spain, Sweden and the Netherlands.

Participant milestones

Participant milestones
Measure	Pirepemat 300 mg Pirepemat tablets, 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo Placebo tablets, 2 tablets t.i.d. for 84 days.
Overall Study STARTED	35	35	34
Overall Study COMPLETED	32	28	30
Overall Study NOT COMPLETED	3	7	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Clinical Study Evaluating Efficacy of Pirepemat on Falls Frequency in Patients With Parkinson's Disease (PD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pirepemat 300 mg n=35 Participants Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=33 Participants Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=33 Participants Placebo tablets, 2 tablets t.i.d. for 84 days.	Total n=101 Participants Total of all reporting groups
Age, Continuous	71.8 year STANDARD_DEVIATION 6.97 • n=20 Participants	71.9 year STANDARD_DEVIATION 6.61 • n=20 Participants	71.2 year STANDARD_DEVIATION 7.87 • n=40 Participants	71.6 year STANDARD_DEVIATION 7.10 • n=5 Participants
Sex: Female, Male Female	16 Participants n=20 Participants	13 Participants n=20 Participants	12 Participants n=40 Participants	41 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=20 Participants	20 Participants n=20 Participants	21 Participants n=40 Participants	60 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=20 Participants	0 Participants n=20 Participants	1 Participants n=40 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=20 Participants	33 Participants n=20 Participants	32 Participants n=40 Participants	99 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to end of full dose treatment (up to Day 84).

Population: The number of participants analyzed for this endpoint differs from the numbers in the Participant Flow as not all participants were treated long enough to fulfill the evaluation period.

Outcome measures

Outcome measures
Measure	Pirepemat 300 mg n=34 Participants Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=28 Participants Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=31 Participants Placebo tablets, 2 tablets t.i.d. for 84 days.
Change in Falls Frequency as Assessed With Fall Diary From Baseline Period (4 Weeks Prior to Randomization) to the End of Treatment.	76.9 % of baseline fall rate Standard Deviation 72.05	64.5 % of baseline fall rate Standard Deviation 49.18	68.9 % of baseline fall rate Standard Deviation 90.22

SECONDARY outcome

Timeframe: Baseline to end of full dose treatment (week 11)

The total scoring range is 0-52, where a higher score indicates more severe impact on Motor Aspects of Experiences of Daily Living (M-EDL).

Outcome measures

Outcome measures
Measure	Pirepemat 300 mg n=32 Participants Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=28 Participants Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=29 Participants Placebo tablets, 2 tablets t.i.d. for 84 days.
Change in the Total Score of MDS-UPDRS Part 2 (M-EDL) From Baseline to End of Full Dose Treatment (With Pirepemat Compared to Placebo).	0.29 units on the scale Standard Error 0.95	-1.42 units on the scale Standard Error 1.03	-2.23 units on the scale Standard Error 0.98

SECONDARY outcome

Timeframe: Baseline to end of full dose treatment (week 11)

Population: A score for NPI-Apathy is reported only when the question "Has the patient lost interest in the world around him/her? Has he/she lost interest in doing things or does he/she lack motivation for starting new activities? Is he/she more difficult to engage in conversation or in doing chores? Is the patient apathetic or indifferent? " is answered Yes. If the answer is No, there is no apathy score. This is why the number of participants analyzed is so different compared to the overall analysis pop.

The total scoring range is 1-12, where a higher score indicates a higher degree of apathy/indifference. The change in NPI-Apathy total score was also a key secondary endpoint, but was not considered evaluable due to \> 50% missing data. The missing data is due to absence of apathy in some participants and thus no available score. Therefore, the statistical analysis will not be reported.

Outcome measures

Outcome measures
Measure	Pirepemat 300 mg n=12 Participants Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=14 Participants Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=9 Participants Placebo tablets, 2 tablets t.i.d. for 84 days.
Change in Total Score (Frequency*Severity) of NPI Item G (Apathy/Indifference) From Baseline to End of Full Dose Treatment (With Pirepemat Compared to Placebo).	-0.418 units of NPI-G score Standard Error 0.78	-0.524 units of NPI-G score Standard Error 0.67	-3.00 units of NPI-G score Standard Error 0.87

SECONDARY outcome

Timeframe: Baseline to end of full dose treatment (week 11)

The scoring range is 0-5, where a higher score indicates a more severe caregiver distress. The change in NPI-Apathy total score was also a key secondary endpoint, but was not considered evaluable due to \> 50% missing data. The missing data is due to absence of apathy and thus no available score. Therefore, the statistical analysis is not reported.

Outcome measures

Outcome measures
Measure	Pirepemat 300 mg n=12 Participants Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=14 Participants Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=9 Participants Placebo tablets, 2 tablets t.i.d. for 84 days.
Change in Caregiver Distress of NPI Item G (Apathy/Indifference) From Baseline to End of Full Dose Treatment (With Pirepemat Compared to Placebo).	0.18 units on a scale Standard Error 0.3	-0.09 units on a scale Standard Error 0.26	-0.53 units on a scale Standard Error 0.35

Adverse Events

Pirepemat 300 mg

Serious events: 4 serious events

Other events: 26 other events

Deaths: 0 deaths

Pirepemat 600 mg

Serious events: 4 serious events

Other events: 27 other events

Deaths: 0 deaths

Placebo

Serious events: 3 serious events

Other events: 18 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Pirepemat 300 mg n=35 participants at risk Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=35 participants at risk Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=34 participants at risk Placebo tablets, 2 tablets t.i.d. for 84 days.
Hepatobiliary disorders Hepatitis cholestatic	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Hepatobiliary disorders Hepatitis toxic	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Bradykinesia	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Neurodenerative disorder	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Cardiac disorders Cardiac Failure	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Gastrointestinal disorders Duodenal Ulcer	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
General disorders Death	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Infections and infestations Covid-19	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Investigations Cystatin C Increased	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Investigations Glomerular Filtration Rate Decreased	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Papillary Thyroid Cancer	2.9% 1/35 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Psychiatric disorders Delusion	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Vascular disorders Haematoma	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Number of events 1 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).

Other adverse events

Additional Information

Joakim Tedroff, Chief Medical Officer

Integrative Research Laboratories Sweden AB

Phone: +46 31 757 38 00

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER

Measure	Pirepemat 300 mg n=35 participants at risk Pirepemat tablets, dose 50 mg, 2 tablets t.i.d. for 84 days.	Pirepemat 600 mg n=35 participants at risk Pirepemat tablets, dose 100 mg, 2 tablets t.i.d. for 84 days.	Placebo n=34 participants at risk Placebo tablets, 2 tablets t.i.d. for 84 days.
Nervous system disorders Head Discomfort	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Psychiatric disorders Hallucination, visual	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Surgical and medical procedures Cataract Operation	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.9% 2/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Dizziness	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Freezing Phenomenon	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Somnolence	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Gastrointestinal disorders Diarrhoea	11.4% 4/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Gastrointestinal disorders Nausea	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.8% 3/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Musculoskeletal and connective tissue disorders Arthralgia	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.8% 3/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Musculoskeletal and connective tissue disorders Pain In Extremity	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Musculoskeletal and connective tissue disorders Musculoskeletal Chest Pain	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Infections and infestations Urinary Tract Infection	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Infections and infestations Covid-19	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Infections and infestations Nasopharyngitis	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
General disorders Asthenia	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
General disorders Fatigue	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Vascular disorders Hypertension	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Surgical and medical procedures Dental Implantation	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.9% 2/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Endocrine disorders Hypothyroidism	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Rib Fracture	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.9% 2/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Contusion	11.4% 4/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	11.8% 4/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Head Injury	11.4% 4/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Joint Injury	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.9% 2/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Fall	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Injury, poisoning and procedural complications Skin Abrasion	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Investigations Hepatic Enzyme Increased	20.0% 7/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	11.4% 4/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Investigations Blood Creatine Phosphokinase Increased	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Investigations Electrocardiogram QT prolonged	0.00% 0/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	5.7% 2/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	0.00% 0/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).
Nervous system disorders Headache	8.6% 3/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/35 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).	2.9% 1/34 • Adverse events were collected from signature of ICF to completion of trial (20-22 weeks).