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A Phase 3 Efficacy and Safety Study of Pitolisant in Patients with Prader-Willi Syndrome
EU CTIS ID: 2023-508307-21-00
What this study is testing
Efficacy: To evaluate the impact of pitolisant on severity of EDS in patients with PWS
- Therapeutic confirmatory (Phase III)
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
- 1. Male or female ages ≥6 years at the time of Screening.
- 11. Patients with a history of seizures must have a stable seizure history (e.g., frequency and severity) for at least 6 months prior to Screening.
- 12. A patient who is an FCBP must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline and agree to remain abstinent or use an effective method of nonhormonal contraception to prevent pregnancy for the duration of the study and for 21 days after final dose of study drug. Patients using hormonal contraception must also use an alternative nonhormonal contraceptive method during treatment with pitolisant and for at least 21 days after discontinuing treatment. An FCBP is defined as a female who is post-menarcheal, has an intact uterus and at least 1 ovary, and is <1 year postmenopausal. Male patients who are not azoospermic (vasectomized or due to a medical cause) must agree to use a barrier method of contraception for the duration of the study and for 21 days after the final dose of study drug.
- 13. Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
- 14. In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
- 2. Ability to provide voluntary, written informed consent (parent[s]/caregiver[s]/legal guardian[s]) and, where applicable, voluntary, written assent (patient, as appropriate).
You likely can't join if
- 1. Diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled at the discretion of the Investigator.
- 10. Has a QTcF with a mean value of >450 msec (QTcF=QT/3√ RR) at Screening based on the mean of triplicate 12-lead ECGs.
- 11. Has a family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
- 12. Has a current or recent (within 1 year) history of a substance use disorder or dependence disorder as defined in the DSM-V.
- 13. Has surgery planned during the Double-Blind Treatment Period of the study.
- 14. Is receiving a concomitant medication that is known to be a centrally acting H1R antagonist; patients who complete a washout for at least 5 half-lives prior to Screening are eligible.
See the full eligibility criteria
Who can join
- 1. Male or female ages ≥6 years at the time of Screening.
- 11. Patients with a history of seizures must have a stable seizure history (e.g., frequency and severity) for at least 6 months prior to Screening.
- 12. A patient who is an FCBP must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline and agree to remain abstinent or use an effective method of nonhormonal contraception to prevent pregnancy for the duration of the study and for 21 days after final dose of study drug. Patients using hormonal contraception must also use an alternative nonhormonal contraceptive method during treatment with pitolisant and for at least 21 days after discontinuing treatment. An FCBP is defined as a female who is post-menarcheal, has an intact uterus and at least 1 ovary, and is <1 year postmenopausal. Male patients who are not azoospermic (vasectomized or due to a medical cause) must agree to use a barrier method of contraception for the duration of the study and for 21 days after the final dose of study drug.
- 13. Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
- 14. In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
- 2. Ability to provide voluntary, written informed consent (parent[s]/caregiver[s]/legal guardian[s]) and, where applicable, voluntary, written assent (patient, as appropriate).
- 3. A diagnosis of PWS confirmed by genetic testing and patient medical records. Genetic testing for PWS will be provided by the Sponsor if not confirmed based on the review of the patient’s medical records.
- 4. & 5. Patient meets criteria for EDS per questionnaires
- 6. Patient meets appropriate number of hours of sleep per night based on their age.
- 7. If taking nonprohibited chronically administered concomitant medication or supplements, patient must be on a stable dose for at least 30 days prior to Screening and agree to remain on that stable dose during the Double-Blind Treatment Period or agree to washout of these medications or supplements for at least 5 half-lives prior to Screening.
- 8. If taking hormone treatments (including growth hormone, testosterone, and estrogen supplements), patient must be on a stable dose of these medications for 30 days prior to Screening and during the Double-Blind Treatment Period; 20% variability in hormone dose is allowed.
- 9. If using cannabidiol and/or tetrahydrocannabinol, patient must be on a stable dose for 30 days prior to Screening and agree to continue on that stable dose for the duration of the Double-Blind Treatment Period of the study or agree to washout of this treatment for at least 5 half-lives prior to Screening.
- 10. If taking a strong CYP2D6 inhibitor, patient must be on a stable dose for at least 30 days prior to Screening and remain on that stable dose during the Double-Blind Treatment Period of the study or agree to washout of the medication for at least for 5 half-lives prior to Screening.
What rules you out
- 1. Diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled at the discretion of the Investigator.
- 10. Has a QTcF with a mean value of >450 msec (QTcF=QT/3√ RR) at Screening based on the mean of triplicate 12-lead ECGs.
- 11. Has a family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
- 12. Has a current or recent (within 1 year) history of a substance use disorder or dependence disorder as defined in the DSM-V.
- 13. Has surgery planned during the Double-Blind Treatment Period of the study.
- 14. Is receiving a concomitant medication that is known to be a centrally acting H1R antagonist; patients who complete a washout for at least 5 half-lives prior to Screening are eligible.
- 15. Is receiving a concomitant medication that is known to be a strong CYP3A4 inducer; patients who complete a washout for at least 5 half-lives prior to Screening are eligible.
- 16. Is receiving a concomitant medication that is known to prolong the QT interval; patients who complete a washout for at least 5 half-lives prior to Screening are eligible.
- 17. Has a significant risk of committing suicide based on history, routine psychiatric examination, Investigator’s judgment, or answering "yes" to question 4 or 5 on the C-SSRS at Screening or Baseline, or with any suicidal behavior within the last 12 months before Screening.
- 18. Is currently breastfeeding or planning to breastfeed over the course of the study. Lactating women must agree not to breastfeed for the duration of the study and for 7 days after final dose of study drug.
- 19. Has been deprived of liberty by administrative or judicial decision.
- 2. Has a diagnosis of hypersomnia due to another sleep/medical disorder.
- 20. Has a known hypersensitivity to the inactive ingredients of pitolisant or placebo tablets.
- 21. Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to unstable or uncontrolled medical conditions (including psychiatric and neurological conditions) or a medical condition that might interfere with the conduct of the study, confound interpretation of study results, pose a health risk to the patient, or compromise the integrity of the study.
- 3. Has previously taken pitolisant.
- 4. Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or within 5 half-lives (whichever is longer) of the investigational medication prior to Screening.
- 5. Has a primary psychiatric diagnosis of ps5. Has a primary psychiatric diagnosis of psychosis or schizophrenia.ychosis or schizophrenia.
- 6. Has a history of moderate hepatic impairment (Child-Pugh Class B) or severe hepatic impairment (Child-Pugh C).
- 7. Has a history of eGFR <60 mL/min/1.73 m2.
- 8. Has abnormal laboratory values at Screening that are clinically significant as determined by the Investigator.
- 9. Has a known history of long QT syndrome or any significant history of a serious abnormality of the ECG (e.g., recent myocardial infarction, clinically significant arrhythmia).
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Australia
- United States
- Canada
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, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Australia; United States; Canada. Enter your location above to see the nearest site and check your eligibility.
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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.