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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)
EU CTIS ID: 2024-516897-31-00
What this study is testing
To assess the efficacy of glycerol phenylbutyrate vs. placebo in reducing the levels of neurofilament light chain (NfL) during 26 weeks of exposure to glycerol phenylbutyrate as well as safety and tolerability of glycerol phenylbutyrate in patients with CBS.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Age: ≥ 18 years
- „clinical possible“ or „clinical probable“ CBS (Armstrong et al., Neurology, 2013 80;496-503) and patients with Progressive Supranuclear Palsy-CBS according to Höglinger et al. (Mov Disord. 2017 Jun;32(6):853-864)
- No regular consumption of glycerol phenylbutyrate within the last 6 months prior to V1
- Capable of thoroughly understanding all information given and giving full informed consent according to GCP
- Capability and willingness to comply with the procedures of the clinical trial
- Women of childbearing age must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. In case of using a hormonal contraception, the method must be supplemented 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 correct 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 vasectomized partner. Unacceptable birth control methods are: periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only and lactational amenorrhoea method (LAM). Female condom and male condom should not be used together
You likely can't join if
- Neurodegenerative diseases other than CBS
- Underlying Alzheimer’s pathology as defined by positive β-amyloid- PET or reduced Aβ 1-42 in CSF
- Participation in another clinical trial involving administration of an investigational medicinal product within 1 month or 5 half-lives of the investigational medicinal product, whichever is longer, prior to V1
- 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
- Treatment with valproic acid, haloperidol or probenecid
- A physical or psychiatric condition (e.g. frontal lobe syndrome, psychotic disorder or major depression), which at the investigator’s discretion may put the subject at risk, may confound the trial results or may interfere with the subject’s participation in this clinical trial
See the full eligibility criteria
Who can join
- Age: ≥ 18 years
- „clinical possible“ or „clinical probable“ CBS (Armstrong et al., Neurology, 2013 80;496-503) and patients with Progressive Supranuclear Palsy-CBS according to Höglinger et al. (Mov Disord. 2017 Jun;32(6):853-864)
- No regular consumption of glycerol phenylbutyrate within the last 6 months prior to V1
- Capable of thoroughly understanding all information given and giving full informed consent according to GCP
- Capability and willingness to comply with the procedures of the clinical trial
- Women of childbearing age must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. In case of using a hormonal contraception, the method must be supplemented 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 correct 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 vasectomized partner. Unacceptable birth control methods are: periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only and lactational amenorrhoea method (LAM). Female condom and male condom should not be used together
- 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, Dopamine- Agonists, Amantadine and MAO-B-Inhibitors) b.) other CNS-active substances including e.g. antidepressants and antidementia drugs
What rules you out
- Neurodegenerative diseases other than CBS
- Underlying Alzheimer’s pathology as defined by positive β-amyloid- PET or reduced Aβ 1-42 in CSF
- Participation in another clinical trial involving administration of an investigational medicinal product within 1 month or 5 half-lives of the investigational medicinal product, whichever is longer, prior to V1
- 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
- Treatment with valproic acid, haloperidol or probenecid
- A physical or psychiatric condition (e.g. frontal lobe syndrome, psychotic disorder or major depression), which at the investigator’s discretion may put the subject at risk, may confound the trial results or may interfere with the subject’s participation in this clinical trial
- Persistent abuse of medication, drugs or alcohol
- Current or planned pregnancy or breast-feeding in females
- Other severe medical conditions upon the discretion of the investigator
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.