Clinical trial • Phase II • Neurology

fasudil hydrochloride for Idiopathic Parkinson's disease | Parkinson's disease

Phase II trial of fasudil hydrochloride for Idiopathic Parkinson's disease | Parkinson's disease.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Idiopathic Parkinson's disease | Parkinson's disease
Trial Stage
Phase II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
18-09-2024
First CTIS Authorization Date
30-09-2024

Trial design

ROCK_PD_low_dose (fasudil hydrochloride oral solution; max daily dose 44 mg, max total dose 924 mg, treatment period 22 days) vs ROCK_PD_high_dose (fasudil hydrochloride oral solution; max daily dose 88 mg, max total dose 1848 mg, treatment period 22 days) vs Placebo: Quinina Labesfal 250 mg/ml (quinine dihydrochloride; marketed product; max daily dose 2 [unit 'Other'], max total dose 42; dilution described: 0.05 ml Quinine dihydrochloride with 30 ml 40% glucose solution directly before use).-controlled Phase II trial across 13 sites in Germany.

Comparator
ROCK_PD_low_dose (fasudil hydrochloride oral solution; max daily dose 44 mg, max total dose 924 mg, treatment period 22 days) vs ROCK_PD_high_dose (fasudil hydrochloride oral solution; max daily dose 88 mg, max total dose 1848 mg, treatment period 22 days) vs Placebo: Quinina Labesfal 250 mg/ml (quinine dihydrochloride; marketed product; max daily dose 2 [unit 'Other'], max total dose 42; dilution described: 0.05 ml Quinine dihydrochloride with 30 ml 40% glucose solution directly before use).
Target Sample Size
75
Trial Duration For Participant
50

Eligibility

Recruits 75 Vulnerable population selected. Participants must be capable of thoroughly understanding all information given and giving full informed consent according to GCP. No assent or proxy consent procedures are specified in the available record..

Pregnancy Exclusion
Pregnant or breast-feeding females or females with childbearing potential, if no adequate contraceptive measures are used
Vulnerable Population
Vulnerable population selected. Participants must be capable of thoroughly understanding all information given and giving full informed consent according to GCP. No assent or proxy consent procedures are specified in the available record.

Inclusion criteria

  • {"criterion_text":"- Patients with a diagnosis of at least probable PD according to MDS criteria (Postuma et al. MovDis 2015) and\n- Hoehn & Yahr stage 1 – 3\n- must be non-fluctuating (no wearing-off, no dyskinesia) and stable on symptomatic PD medication for at least 6 weeks\n- age: 30 - 80 years\n- 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\n- Capable of thoroughly understanding all information given and giving full informed consent according to GCP"}

Exclusion criteria

  • {"criterion_text":"- 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\n- Hypersensitivity to any component of the IMP\n- 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\n- Pregnant or breast-feeding females or females with childbearing potential, if no adequate contraceptive measures are used\n- 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\n- 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\n- Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n- 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\n- Patients with an uncontrollable or unstable arterial hypertensive disease (resting blood pressure >180 mmHg systolic and/or >120 mmHg diastolic under current antihypertensive medication)\n- Known pulmonary hypertension and any medication prescribed for treatment of pulmonary hypertension\n- 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 nontransient through repeat testing\n- Renal insufficiency with a glomerular filtration rate (GFR) <60 ml/min/1,73m² (calculated by MDRD equation) and determined to be non-transient through repeat testing\n- Major psychiatric disorder, significant cognitive impairment or clinically evident dementia precluding evaluation of symptoms"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Combined occurrence of intolerance (termination of treatment because of treatment-related AE) and occurence of self-reported and pre-defined (laboratory, vital signs) treatment-related SAEs [time frame: from day 1 to day 22]","definition_or_measurement_approach":"Combined occurrence of intolerance defined as termination of treatment because of treatment-related adverse event (AE) and occurrence of self-reported and pre-defined treatment-related serious adverse events (SAEs); assessed from day 1 to day 22 with laboratory and vital signs included as pre-defined measures."}

Secondary endpoints

  • {"endpoint_text":"- Occurrence of intolerance (termination of treatment because of treatment-related AE) [time frame: from day 1 to day 22].","definition_or_measurement_approach":"Occurrence of intolerance defined as termination of treatment due to treatment-related AE; assessed from day 1 to day 22."}
  • {"endpoint_text":"- Occurrence of self-reported and pre-defined (laboratory, vital signs) treatment-related SAEs [time frame: from day 1 to day 22, and day 1 to day 50].","definition_or_measurement_approach":"Occurrence of self-reported and pre-defined treatment-related SAEs based on laboratory and vital signs; assessed from day 1 to day 22 and day 1 to day 50."}
  • {"endpoint_text":"- the change of MDS-Unified PD Rating Scale (MDS-UPDRS) each part I to IV [time frame: 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]","definition_or_measurement_approach":"Change from baseline in MDS-UPDRS parts I–IV at specified time frames (part I & II: day 1 to day 22 and day 1 to day 50; part III & IV: day 1 to day 10, day 1 to day 22, and day 1 to day 50)."}
  • {"endpoint_text":"- the change of MDS-Unified PD Rating Scale (MDS-UPDRS) score part I to IV","definition_or_measurement_approach":"Change in total and part scores of MDS-UPDRS (parts I–IV) over specified assessment visits."}
  • {"endpoint_text":"- the change of 8-item PD Quality of Life Scale (PDQ-8) [time frame: from day 1 to day 22, and day 1 to day 50]","definition_or_measurement_approach":"Change from baseline in PDQ-8 scores assessed from day 1 to day 22 and day 1 to day 50."}
  • {"endpoint_text":"- the change of PD Non-Motor Symptom Questionnaire (NMSQuest) [time frame: from day 1 to day 10, day 1 to day 22, and day 1 to day 50]","definition_or_measurement_approach":"Change from baseline in NMSQuest scores at day 10, day 22 and day 50."}
  • {"endpoint_text":"- the change of Montreal Cognitive Assessment (MoCA) [time frame: from day 1 to day 22, and day 1 to day 50]","definition_or_measurement_approach":"Change from baseline in MoCA cognitive assessment at day 22 and day 50."}
  • {"endpoint_text":"- the change of Becks depression inventory-II (BDI-II) [time frame: from day 1 to day 22, and day 1 to day 50],","definition_or_measurement_approach":"Change from baseline in BDI-II depression scores at day 22 and day 50."}
  • {"endpoint_text":"- the Global Impression of Improvement score (CGI-I [clinician], PGI-I [patient]) [time point: at day 10, 22 and day 50].","definition_or_measurement_approach":"Clinician Global Impression of Improvement (CGI-I) and Patient Global Impression of Improvement (PGI-I) assessed at day 10, day 22 and day 50."}

Recruitment

Planned Sample Size
75
Recruitment Window Months
41
Consent Approach
Participants must be capable of thoroughly understanding all information given and giving full informed consent according to GCP. Subject information and informed consent forms are listed in the documents (L1_SIS_and_ICF variants). No assent, proxy consent procedures, age-specific consent documents or languages are specified in the available record.

Geography

Total Number Of Sites
13
Total Number Of Participants
75

Germany

Earliest CTIS Part Ii Submission Date
20-09-2024
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
544
Number Of Sites
13
Number Of Participants
75

Sites

Site Name
Universitaet Muenster
Department Name
Klinik für Neurologie
Contact Person Name
Inga Claus
Contact Person Email
inga.claus@ukmuenster.de
Site Name
Katholisches Klinikum Bochum gGmbH
Department Name
Neurologische Klinik
Contact Person Name
Siegfried Muhlack
Contact Person Email
siegfried.muhlack@rub.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Neurologie
Contact Person Name
Thomas Gasser
Site Name
Technische Universitaet Dresden
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Björn Falkenburger
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Neurologische Klinik und Poliklinik
Contact Person Name
Jens Volkmann
Contact Person Email
volkmann_j@ukw.de
Site Name
Klinikum rechts der Isar der TU Muenchen AöR
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Paul Lingor
Contact Person Email
paul.lingor@tum.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik für Neurologie
Contact Person Name
Christoph van Riesen
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
Institut für Klinische Neurowissenschaften und Medizinische Psychologie
Contact Person Name
Alfons Schnitzler
Site Name
Universitaet Leipzig
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Joseph Claßen
Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Klinikum für Neurologie
Contact Person Name
Daniela Berg
Contact Person Email
daniela.berg@uksh.de
Site Name
Philipps-Universitaet Marburg
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Karla Eggert
Contact Person Email
eggert@med.uni-marburg.de
Site Name
Universitaetsklinikum Erlangen AöR
Department Name
Molekulare Neurologie, Bewegungsambulanz
Contact Person Name
Martin Regensburger
Site Name
Medical Center - University Of Freiburg
Department Name
Klinik für Neurologie und Neurophysiologie
Contact Person Name
Michel Rijntjes

Sponsor

Primary sponsor

Full Name
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Germany

Investigational products

Investigational Product Name
ROCK_PD_low_dose
Active Substance
fasudil hydrochloride
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Authorisation Status
Investigational product (no marketing authorisation listed)
Dose Levels
max daily dose 44 mg; max total dose 924 mg; max treatment period 22 days
Maximum Dose
44 mg per day (max daily dose); max total 924 mg
Investigational Product Name
ROCK_PD_high_dose
Active Substance
fasudil hydrochloride
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Authorisation Status
Investigational product (no marketing authorisation listed)
Dose Levels
max daily dose 88 mg; max total dose 1848 mg; max treatment period 22 days
Maximum Dose
88 mg per day (max daily dose); max total 1848 mg
Investigational Product Name
Quinina Labesfal 250 mg/ml Solução injetável
Active Substance
quinine dihydrochloride
Modality
Small molecule
Routes Of Administration
ORAL
Route
oral
Authorisation Status
Authorised (marketing authorisation in PT: marketingAuthNumber 5506795)
Dose Levels
max daily dose 2 (unit 'Other'); max total dose 42; product change description: Dilution (0.05 ml Quinine dihydrochloride with 30 ml 40% glucose solution, directly before use)
Maximum Dose
2 (daily unit 'Other'); max total 42

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