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A randomized, placebo-controlled trial to assess the efficacy, tolerability, and pharmacokinetics of clemastine in children and adults with Pitt-Hopkins syndrome
EU CTIS ID: 2025-524195-29-00
What this study is testing
To assess the overall treatment effects of clemastine in PTHS, according to caretakers
- 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
- A molecularly confirmed diagnosis of PTHS according to the criteria described in the 2019 consensus statement.(Zollino et al. 2019)
- An age of at least 1.0 years old at the start of the trial.
- A bodyweight of at least 12.0 kilograms at the start of the trial.
- Ability of the parents or legally designated representative of the subject to understand the information about trial procedures and requirements.
- Ability and willingness of the parents or legally designated representative to provide written informed consent for trial participation.
You likely can't join if
- Comorbidity with any other relevant disease or condition, including a second neurodevelopmental disorder in addition to PTHS, that would impair assessment of treatment effects (judged on a case by case basis by the principal investigator).
- Simultaneous involvement in another study protocol without prior approval
- Non-incidental use of clemastine in the 6 months prior to the start of the trial
- Non-incidental use of medication currently being investigated in remyelination trials, including but not limited to domperidone, liothyronine, quetiapine, testosterone and bazedoxifene, in the 6 months prior to the start of the trial.
- Any change in use or dosage of psychotropic medications during the 4 weeks prior to the start of the trial.
- Contraindications for use of clemastine, such as known porphyria or hypersensitivity to clemastine or any of the excipients.
See the full eligibility criteria
- A molecularly confirmed diagnosis of PTHS according to the criteria described in the 2019 consensus statement.(Zollino et al. 2019)
- An age of at least 1.0 years old at the start of the trial.
- A bodyweight of at least 12.0 kilograms at the start of the trial.
- Ability of the parents or legally designated representative of the subject to understand the information about trial procedures and requirements.
- Ability and willingness of the parents or legally designated representative to provide written informed consent for trial participation.
- Comorbidity with any other relevant disease or condition, including a second neurodevelopmental disorder in addition to PTHS, that would impair assessment of treatment effects (judged on a case by case basis by the principal investigator).
- Simultaneous involvement in another study protocol without prior approval
- Non-incidental use of clemastine in the 6 months prior to the start of the trial
- Non-incidental use of medication currently being investigated in remyelination trials, including but not limited to domperidone, liothyronine, quetiapine, testosterone and bazedoxifene, in the 6 months prior to the start of the trial.
- Any change in use or dosage of psychotropic medications during the 4 weeks prior to the start of the trial.
- Contraindications for use of clemastine, such as known porphyria or hypersensitivity to clemastine or any of the excipients.
- Non-incidental use and planned continuation of CNS depressants including but not limited to hypnotics, anxiolytics, monoamine-oxidase inhibitors, tricyclic antidepressants, opioid analgesics, other antihistamines with sedating properties (e.g. promethazine) and anticholinergic medication.
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, 0-17 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.