Corticobasal Syndrome (CBS) MedDRA version: 20.0 Level: PT Classification code 10078208 Term: Corticobasal degeneration System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: RAVICTI
Pharmaceutical Form: Oral solution in bottle
INN or Proposed INN: Glycerol phenylbutyrate
CAS Number: 611168-24-2
Concentration unit: g/ml gram(s)/millilitre
Concentration type: eq
Sponsors
Klinikum rechts der Isar der Technischen Universität München
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age: = 18 years 2. „clinical possible“ or „clinical probable“ CBS (Armstrong et al., Neurol-ogy, 2013 80;496-503) and patients with Progressive Supranuclear Palsy-CBS according to Höglinger et al. (Mov Disord. 2017 Jun;32(6):853-864) 3. No regular consumption of glycerol phenylbutyrate within the last 6 months prior to V1 4. Capable of thoroughly understanding all information given and giving full informed consent according to GCP 5. Capability and willingness to comply with the procedures of the clini-cal trial 6. Women of childbearing age must be non-lactating and surgically ster-ile or using a highly effective method of birth control and have a nega-tive pregnancy test. In case of using a hormonal contraception, the method must be sup-plemented with a barrier method (preferably male condom). Acceptable methods of birth control with a low failure rate i.e. less than 1% per year when used consistently and cor-rect are such as implants, injectables, combined oral contraceptives, hormonal intrauterine devices (IUDs), sexual abstinence (defined as refraining from heterosexual intercourse during the clinical trial) or vasecto-mized partner. Unacceptable birth control methods are: periodic abstinence (calen-dar, symptothermal, post-ovulation methods), withdrawal (coitus inter-ruptus), spermicides only and lactational amenorrhoea method (LAM). Female condom and male condom should not be used together. 7. A stable regimen for at least 1 month prior to V1 and no foreseeable need to change the regimen throughout the 26 week treatment period for a. drugs acting against Parkinsonism (e.g. Levodopa, Dopa-mine-Agonists, Amantadine and MAO-B-Inhibitors) b. other CNS-active substances including e.g. antidepressants and antidementia drugs Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 26
Exclusion criteria
Exclusion criteria: 1. Neurodegenerative diseases other than CBS 2. Underlying Alzheimer’s pathology as defined by positive ß-amyloid-PET or reduced Aß 1-42 in CSF 3. Participation in another clinical trial involving administration of an in-vestigational medicinal product within 1 month or 5 half-lives of the investigational medicinal product, whichever is longer, prior to V1 4. Known hypersensitivity to glycerol phenylbutyrate or its further components, or to drugs with a similar chemical structure or to any of the components of the placebo 5. Treatment with valproic acid, haloperidol or probenecid 6. A physical or psychiatric condition (e.g. frontal lobe syndrome, psychotic disorder or major depression), which at the investigator’s discre-tion may put the subject at risk, may confound the trial results or may interfere with the subject’s participation in this clinical trial 7. Persistent abuse of medication, drugs or alcohol 8. Current or planned pregnancy or breast-feeding in females 9. Other severe medical conditions upon the discretion of the investiga-tor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of glycerol phenylbutyrate vs. placebo in reducing the levels of neurofilament light chain (NfL) during 26 weeks of exposure to glyc-erol phenylbutyrate as well as safety and tolerability of glycerol phenylbutyrate in patients with CBS.;Secondary Objective: To assess longitudinal changes in clinical scales (MDS-UPDRS Part III, PSP-RS, PSP-CDS, CBFS, DATE, MoCA, SEADL, CGI-s, PSP-QoL) between V1 and V3 comparing placebo- vs. verum-treated patients;Primary end point(s): Primary efficacy endpoint: The primary outcome measure will be the longitudinal change of NfL in plasma between V1 and V3 comparing GPB vs. Placebo-treated pa-tients. Primary Endpoints for Safety and Tolerability: Safety • Incidence of specific AEs • Safety laboratory values (basic clinical chemistry) • Vital signs (blood pressure, heart rate, temperature) • Physical and neurological examination • Survival time and survival rate during the study period Tolerability • Number of subjects (and % of the intention-to-treat population), who discontinue the clinical trial due to adverse reactions ;Timepoint(s) of evaluation of this end point: between V1 and V3 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Longitudinal changes in clinical scales (MDS-UPDRS Part III, PSP-RS, PSP-CDS, CBFS, DATE, MoCA, SEADL, CGI-s, PSP-QoL) between V1 and V3 comparing placebo- vs. verum-treated patients;Timepoint(s) of evaluation of this end point: between V1 and V3 | — |
Countries
Germany
Contacts
Public ContactHelen Bidner
Technische Universität München, Fakultät für Medizin, Münchner Studienzentrum
Outcome results
None listed