Double-blind, randomised, prospective, placebo controlled parallel group phase II study to investigate the effect of glycerol phenylbutyrate (GPB) on neurofilament light chain (NfL) levels in patients with corticobasal syndrome (CBS)
- Conditions
- MedDRA version: 20.0Level: PTClassification code 10078208Term: Corticobasal degenerationSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]Corticobasal Syndrome (CBS)
- Registration Number
- EUCTR2022-002988-30-DE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 32
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
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method