Safety, Tolerability and Symptomatic Efficacy of the ROCK-Inhibitor Fasudil in Patients With Parkinson's Disease

NCT05931575 | Recruiting Phase 2

• 2 other identifiers: ROCK-PD-0000-LIN-0075-I01EN2005
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this phase Ila trial is to provide evidence on safety, tolerability and symptomatic efficacy of the ROCK-inhibitor Fasudil in patients with early Parkinson's disease (PD). Fasudil has shown neuroprotective and pro-regenerative effects, modulated microglial activity and attenuated alpha-synuclein aggregation in PD models in vitro and in vivo. It has been licensed in Japan since 1995 for the treatment of vasospasms and has a beneficial safety profile arguing for its repurposing. Up to 15 trial centers in Germany will recruit patients. Blinded trial medication will be prepared and shipped by the University Pharmacy Leipzig. Fasudil in two dosages or placebo will be administered orally twice daily to 75 early PD patients for a total of 3 weeks. Safety, tolerability and symptomatic efficacy endpoints will be assessed up to 4 weeks after end of treatment. Its well-known safety profile and the lack of disease-modifying treatments for PD justifies its use in patients with early Parkinson's disease. ROCK-PD is a prerequisite for subsequent long-term clinical trials assessing disease-modification in PD in addition to symptomatic efficacy.

Conditions

Idiopathic Parkinson´s Disease

Outcome Measures

Primary Outcomes (1)

• Combined occurrence of intolerance and/or occurrence of self-reported and pre-defined treatment-related SAEs

  Combined occurrence of intolerance (termination of treatment because of treatment-related AE) and/or occurrence of self-reported and pre-defined (laboratory, vital signs) treatment-related SAEs

  from day 1 to day 22

Secondary Outcomes (9)

• Occurrence of intolerance

  from day 1 to day 22

• Occurrence of self-reported and pre-defined treatment-related SAEs

  from day 1 to day 22, and day 1 to day 50

• Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

  part I and II from day 1 to day 22, and day 1 to day 50; time frame: part III and IV from day 1 to day 10, day 1 to day 22, and day 1 to day 50

• MDS-Unified PD Rating Scale (MDS-UPDRS) score

  from day 1 to day 22, and day 1 to day 50

• Parkinson's Disease Questionnaire (PDQ-8)

  from day 1 to day 22, and day 1 to day 50

Study Arms (3)

intervention arm (low dose)

EXPERIMENTAL

oral Fasudil solution 88 mg/day (2 x 44 mg)

Drug: Fasudil hydrochloride

intervention arm (high dose)

EXPERIMENTAL

oral Fasudil solution 44 mg/day (2 x 22 mg)

Drug: Fasudil hydrochloride

Control intervention arm (placebo)

PLACEBO COMPARATOR

oral placebo solution 2x/day.

Drug: Placebo

Interventions

Fasudil hydrochloride DRUG

Duration of intervention per patient: 22 days; Application scheme: one dose on day 1, two doses on days 2 - 21, one dose on day 22.

Also known as: Fasudil hydrochloride (Fasudil)

intervention arm (high dose); intervention arm (low dose)

Placebo DRUG

0.05 ml Quinine dihydrochloride solution (from Quinina Labesfal) in screw flask supplemented with 30 ml Glucose 40% solution from miniplasco directly before use

Also known as: Quinine dihydrochloride solution

Control intervention arm (placebo)

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with a diagnosis of at least probable PD according to MDS criteria (Postuma et al. MovDis 2015) and
• Hoehn \& Yahr stage 1 - 3
• must be non-fluctuating (no wearing-off, no dyskinesia) and stable on symptomatic PD medication for at least 6 weeks
• age: 30 - 80 years
• Women of childbearing potential must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. Acceptable methods of birth control with a low failure rate (i.e. less than 1% per year) when used consistently and correct are for example implants, injectables, combined oral contraceptives, hormonal intrauterine devices (IUDs), sexual abstinence or vasectomized partner
• Capable of thoroughly understanding all information given and giving full informed consent according to GCP

You may not qualify if:

• Atypical, secondary Parkinsonian syndromes, PD mimics, or any other medical condition known to have an association with Parkinsonian syndromes, which might confound or obscure the diagnosis of PD
• Patients with a history of intracranial bleeding, known intracerebral aneurysms or Moyamoya disease, or positive family history for the above. If only family history positive, MR- or x-ray-based cranial imaging not older than 24 months must confirm absence of bleeding, aneurysms, or Moyamoya
• Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment
• Patients with known arterial hypotension (resting blood pressure \<90/60 mmHg) or previous hypotensive episodes or requiring treatment for increasing of blood pressure, such as fludrocortisone, midodrine, etilefrine, cafedrine, or theodrenaline
• Patients with an uncontrollable or unstable arterial hypertensive disease (resting blood pressure \>180 mmHg systolic and/or \>120 mmHg diastolic under current antihypertensive medication)
• Known pulmonary hypertension and any medication prescribed for treatment of pulmonary hypertension
• Confirmed hepatic insufficiency or abnormal liver function (stable ASAT and/or ALAT greater than 3 times the upper limit of the normal range) and determined to be non-transient through repeat testing
• Renal insufficiency with a glomerular filtration rate (GFR) \<60 ml/min/1,73m² (calculated by MDRD equation or byCKD-EPI equation) and determined to be non-transient through repeat testing
• Major psychiatric disorder, significant cognitive impairment or clinically evident dementia precluding evaluation of symptoms
• Hypersensitivity to any component of the IMP
• Liable to be not cooperative or comply with the trial requirements (as assessed by the investigator), or unable to be reached in the case of emergency
• Pregnant or breast-feeding females or females with childbearing potential, if no adequate contraceptive measures are used
• Previous participation in another clinical study involving trial medication within the preceding 12 weeks or five terminal half times of the longest to be eliminated trial medications (whichever is longer) or previous participation in this trial

Sponsors & Collaborators

• Technical University of Munich: lead

Study Sites (1)

Technische Universität München, Klinikum rechts der Isar, Klinik und Poliklinik für Neurologie

Munich, 81675, Germany

RECRUITING

Related Publications (1)

• Wolff AW, Bidner H, Remane Y, Zimmer J, Aarsland D, Rascol O, Wyse RK, Hapfelmeier A, Lingor P. Protocol for a randomized, placebo-controlled, double-blind phase IIa study of the safety, tolerability, and symptomatic efficacy of the ROCK-inhibitor Fasudil in patients with Parkinson's disease (ROCK-PD). Front Aging Neurosci. 2024 Feb 14;16:1308577. doi: 10.3389/fnagi.2024.1308577. eCollection 2024.

  PMID: 38419648 DERIVED

MeSH Terms

Interventions

fasudil

Study Officials

• Paul Lingor, MD

  Technische Universität München, Klinikum rechts der Isar, Klinik und Poliklinik für Neurologie

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Paul Lingor, MD

CONTACT

+49 89 4140 8257; paul.lingor@tum.de

Andreas Wolff, MD

CONTACT

+49 89 4140 8237; andreas.wolff@tum.de

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2023
• First Posted: July 5, 2023
• Study Start: September 11, 2023
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)