Clinical trial • Phase III • Rare Disease
PITOLISANT HYDROCHLORIDE for Prader-Willi syndrome
Phase III trial of PITOLISANT HYDROCHLORIDE for Prader-Willi syndrome.
Overview
- Trial Therapeutic Area
- Rare Disease
- Trial Disease
- Prader-Willi syndrome
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 07-10-2024
- First CTIS Authorization Date
- 10-02-2025
Trial design
Randomised, open-label, placebo arm - matching placebo as a single dose once daily during 77-day double-blind treatment period; investigational arm - pitolisant as a single dose once daily during 77-day double-blind treatment period (matching tablets described: 4.45 mg and 17.8 mg matching tablets; specific starting dose/titration details not specified in provided data).-controlled Phase III trial in Italy, Denmark, Germany and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Placebo arm - matching placebo as a single dose once daily during 77-day Double-Blind Treatment Period; Investigational arm - pitolisant as a single dose once daily during 77-day Double-Blind Treatment Period (matching tablets described: 4.45 mg and 17.8 mg matching tablets; specific starting dose/titration details not specified in provided data).
- Target Sample Size
- 67
- Trial Duration For Participant
- 516
Eligibility
Recruits 67 paediatric patients.
- Pregnancy Exclusion
- 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.
- Vulnerable Population
- Participants include minors (children aged ≥6 years) and patients who may require caregiver assistance. Consent is required from parent(s)/caregiver(s)/legal guardian(s) (written informed consent) and, where appropriate, voluntary written assent from the patient. The protocol requires a consistent parent/caregiver able to complete study assessments. Multiple age-specific assent and caregiver/parent ICF documents are provided (examples: pediatric information/assent forms for 6-9 years, 10-13 years, 14 years and above, adolescent assent 12-17, and caregiver/parent ICFs) and translated materials are provided per country.
Inclusion criteria
- {"criterion_text":"- 1.\tMale or female ages ≥6 years at the time of Screening.\n- 11.\tPatients with a history of seizures must have a stable seizure history (e.g., frequency and severity) for at least 6 months prior to Screening.\n- 12.\tA 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.\n- 13.\tHas a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.\n- 14.\tIn 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.\n- 2.\tAbility to provide voluntary, written informed consent (parent[s]/caregiver[s]/legal guardian[s]) and, where applicable, voluntary, written assent (patient, as appropriate).\n- 3.\tA 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.\n- 4. & 5.\tPatient meets criteria for EDS per questionnaires\n- 6.\tPatient meets appropriate number of hours of sleep per night based on their age.\n- 7.\tIf 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.\n- 8.\tIf 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.\n- 9.\tIf 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.\n- 10.\t 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."}
Exclusion criteria
- {"criterion_text":"- 1.\tDiagnosis of sleep apnea (OSA, CSA) that is not adequately controlled at the discretion of the Investigator.\n- 10.\tHas a QTcF with a mean value of >450 msec (QTcF=QT/3√ RR) at Screening based on the mean of triplicate 12-lead ECGs.\n- 11.\tHas a family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.\n- 12.\tHas a current or recent (within 1 year) history of a substance use disorder or dependence disorder as defined in the DSM-V.\n- 13.\tHas surgery planned during the Double-Blind Treatment Period of the study.\n- 14.\tIs 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.\n- 15.\tIs 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.\n- 16.\tIs 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.\n- 17.\tHas 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.\n- 18.\tIs 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.\n- 19. Has been deprived of liberty by administrative or judicial decision.\n- 2.\tHas a diagnosis of hypersomnia due to another sleep/medical disorder.\n- 20. Has a known hypersensitivity to the inactive ingredients of pitolisant or placebo tablets.\n- 21.\tBased 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.\n- 3.\tHas previously taken pitolisant.\n- 4.\tParticipation 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.\n- 5.\tHas a primary psychiatric diagnosis of ps5.\tHas a primary psychiatric diagnosis of psychosis or schizophrenia.ychosis or schizophrenia.\n- 6.\tHas a history of moderate hepatic impairment (Child-Pugh Class B) or severe hepatic impairment (Child-Pugh C).\n- 7.\tHas a history of eGFR <60 mL/min/1.73 m2.\n- 8.\tHas abnormal laboratory values at Screening that are clinically significant as determined by the Investigator.\n- 9.\tHas 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)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Efficacy: Change in severity of EDS as measured by PROMIS SRI T-score from Baseline to the end of the Double Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by PROMIS SRI T-score change from Baseline to Day 77 (end of Double-Blind Treatment Period)."}
Secondary endpoints
- {"endpoint_text":"- Efficacy: Change in severity of irritable and disruptive behaviors as measured by ABC-C Irritability Domain score from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by ABC-C Irritability Domain score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in overall severity of EDS as measured by the CaGI-S for EDS score from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by CaGI-S for EDS score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in overall severity of EDS as measured by the CGI-S for EDS score from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by CGI-S for EDS score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in overall severity of irritable and disruptive behaviors as measured by the CaGI-S for Irritable and/or Disruptive Behaviors score from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by CaGI-S for Irritable and/or Disruptive Behaviors score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in severity of hyperphagia as measured by HQ CT score in conjunction with the FSZQ score, from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by HQ-CT score in conjunction with FSZQ score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in severity of EDS as measured by ESS CHAD (parent/caregiver version) total score from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by ESS-CHAD parent/caregiver total score change from Baseline to Day 77."}
- {"endpoint_text":"- Efficacy: Change in severity of other behavioral problems as measured by the ABC-C Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, and Stereotypic Behavior Domain scores from Baseline to the end of the Double-Blind Treatment Period (Day 77)","definition_or_measurement_approach":"Measured by changes in ABC-C domain scores (Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, Stereotypic Behavior) from Baseline to Day 77."}
- {"endpoint_text":"- Safety and Pharmacokinetic: Percentage of patients reporting TEAEs during each study period and during the entire study","definition_or_measurement_approach":"Reported percentage of patients with treatment-emergent adverse events (TEAEs) during each study period and overall."}
- {"endpoint_text":"- Safety and Pharmacokinetic: Measured concentration of pitolisant","definition_or_measurement_approach":"Measured plasma concentration of pitolisant (PK sampling as specified in protocol)."}
Recruitment
- Registry Or Advocacy Recruitment
- True, documents reference engagement with PWS advocacy (document: Site-PWS-Advocacy-Introductory-Letter_DNK_Danish_Public and PWS brochures/flyers across countries).
- Digital Remote Recruitment
- True, the study uses web-site screening reports, app subject-facing screen reports, and telephone pre-ICF data consent procedures to support remote/digital screening.
- Planned Sample Size
- 67
- Recruitment Window Months
- 28
- Consent Approach
- Informed consent is provided by parent(s)/caregiver(s)/legal guardian(s) (written informed consent). Where applicable, voluntary written assent is required from the patient. The protocol includes multiple age-specific assent and pediatric information documents (e.g., Assent forms for 6-9 years, 10-13 years, 14 years and above, Adolescent assent 12-17 or 15-17 as provided) and caregiver/parent ICFs. Documents are provided in country/language-specific versions as listed (Italian, Danish, German, Spanish, French, Romanian, Swedish, Polish, Czech, Dutch/English/French for Belgium, etc.).
Methods
- Country-specific recruitment arrangements documents (K1) and materials including brochures, flyers, flipcharts, site introductory letters, GP letters and PWS brochures (documents exist for Italy, Denmark, Germany, Spain, France, Romania, Sweden, Belgium, Poland, Czechia).
- Use of web-site screening reports and app subject-facing screening reports (digital screening tools) to identify potential participants.
- Pre-ICF telephone data consent procedures and telephone-based pre-screening (documents: Pre-ICF Telephone Data Consent).
- Engagement with clinics and hospital sites (referral from paediatric/endocrinology/neurology departments) using site-level introductory letters.
Geography
- Total Number Of Sites
- 26
- Total Number Of Participants
- 67
Italy
- Earliest CTIS Part Ii Submission Date
- 22-10-2024
- Latest Decision Or Authorization Date
- 25-03-2026
- Processing Time Days
- 489
- Number Of Sites
- 8
- Number Of Participants
- 14
Sites
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- UO Pediatria
- Principal Investigator Name
- Roberta Pajno
- Principal Investigator Email
- pajno.roberta@hsr.it
- Contact Person Name
- Roberta Pajno
- Contact Person Email
- pajno.roberta@hsr.it
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- SOC Diabetologia ed Endocrinologia
- Principal Investigator Name
- Stefano Stagi
- Principal Investigator Email
- stefano.stagi@meyer.it
- Contact Person Name
- Stefano Stagi
- Contact Person Email
- stefano.stagi@meyer.it
- Site Name
- Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
- Department Name
- Pediatria - Endocrinologia, auxologia e diabetologia
- Principal Investigator Name
- Gianluca Tornese
- Principal Investigator Email
- gianluca.tornese@burlo.trieste.it
- Contact Person Name
- Gianluca Tornese
- Contact Person Email
- gianluca.tornese@burlo.trieste.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- UO Endocrinologia e Diabetologia
- Principal Investigator Name
- Danilo Fintini
- Principal Investigator Email
- danilo.fintini@opbg.net
- Contact Person Name
- Danilo Fintini
- Contact Person Email
- danilo.fintini@opbg.net
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- U.O.C. Clinica Pediatrica, Endocrinologia
- Principal Investigator Name
- Mohamad Maghnie
- Principal Investigator Email
- mohamadmaghnie@ospedale-gaslini.ge.it
- Contact Person Name
- Mohamad Maghnie
- Contact Person Email
- mohamadmaghnie@ospedale-gaslini.ge.it
- Site Name
- Azienda Ospedaliero Universitaria Di Modena
- Department Name
- Scienze Mediche e Chirurgiche Materno-Infantili e dell'Adulto
- Principal Investigator Name
- Lorenzo Iughetti
- Principal Investigator Email
- iughetti.lorenzo@unimore.it
- Contact Person Name
- Lorenzo Iughetti
- Contact Person Email
- iughetti.lorenzo@unimore.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- UOSD Endocrinologia e Adolescentologia Pediatrica
- Principal Investigator Name
- Laura Guazzarotti
- Principal Investigator Email
- laura.guazzarotti@aopd.veneto.it
- Contact Person Name
- Laura Guazzarotti
- Contact Person Email
- laura.guazzarotti@aopd.veneto.it
Denmark
- Earliest CTIS Part Ii Submission Date
- 15-01-2025
- Latest Decision Or Authorization Date
- 10-04-2026
- Processing Time Days
- 450
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Rigshospitalet
- Department Name
- Centre for Rare Diseases, Paediatric and Adolescent medicine
- Principal Investigator Name
- Stense Farholt
- Principal Investigator Email
- stense.farholt@regionh.dk
- Contact Person Name
- Stense Farholt
- Contact Person Email
- stense.farholt@regionh.dk
- Site Name
- Region Midtjylland
- Department Name
- Aarhus University Hospital, Centre for Rare Diseases, Paediatric and Adolescent medicine
- Principal Investigator Name
- Cecilie Ejerskov
- Principal Investigator Email
- ceej@clin.au.dk
- Contact Person Name
- Cecilie Ejerskov
- Contact Person Email
- ceej@clin.au.dk
Germany
- Earliest CTIS Part Ii Submission Date
- 29-01-2025
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 419
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Klinik für Kinderheilkunde II
- Principal Investigator Name
- Cordula Kiewert
- Principal Investigator Email
- cordula.kiewert@uk-essen.de
- Contact Person Name
- Cordula Kiewert
- Contact Person Email
- cordula.kiewert@uk-essen.de
Spain
- Earliest CTIS Part Ii Submission Date
- 13-01-2025
- Latest Decision Or Authorization Date
- 13-04-2026
- Processing Time Days
- 455
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- Pediatric Service
- Principal Investigator Name
- Irene Lázaro Rodríguez
- Principal Investigator Email
- irene.lazaro@salud.madrid.org
- Contact Person Name
- Irene Lázaro Rodríguez
- Contact Person Email
- irene.lazaro@salud.madrid.org
- Site Name
- Parc Tauli Hospital Universitari
- Department Name
- Pediatric Service
- Principal Investigator Name
- Raquel Corripio Collado
- Principal Investigator Email
- rcorripio@tauli.cat
- Contact Person Name
- Raquel Corripio Collado
- Contact Person Email
- rcorripio@tauli.cat
- Site Name
- Hospital Universitario Virgen De La Victoria
- Department Name
- Endocrinology and Nutrition service
- Principal Investigator Name
- Francisco José Tinahones Madueño
- Principal Investigator Email
- fjtinahones@hotmail.com
- Contact Person Name
- Francisco José Tinahones Madueño
- Contact Person Email
- fjtinahones@hotmail.com
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Neurology
- Principal Investigator Name
- Oscar Sans capdevila
- Principal Investigator Email
- Oscarramon.sans@sjd.es
- Contact Person Name
- Oscar Sans capdevila
- Contact Person Email
- Oscarramon.sans@sjd.es
Belgium
- Earliest CTIS Part Ii Submission Date
- 10-01-2025
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 444
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- UZ Brussel
- Department Name
- Neurology
- Principal Investigator Name
- Tessa Wassenberg
- Principal Investigator Email
- tessa.wassenberg@uzbrussel.be
- Contact Person Name
- Tessa Wassenberg
- Contact Person Email
- tessa.wassenberg@uzbrussel.be
France
- Earliest CTIS Part Ii Submission Date
- 14-12-2024
- Latest Decision Or Authorization Date
- 15-04-2026
- Processing Time Days
- 487
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Hopital Des Enfants (CHU Toulouse)
- Department Name
- CHU Toulouse, Endocrinology Unit, Obesity, Bone Diseases, genetics and Medical Gynecology
- Principal Investigator Name
- Gwenaëlle DIENE
- Principal Investigator Email
- diene.g@chu-toulouse.fr
- Contact Person Name
- Gwenaëlle DIENE
- Contact Person Email
- diene.g@chu-toulouse.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Centre Hospitalier Universitaire d’Angers Endocrinologie, Diabétologie, Nutrition
- Principal Investigator Name
- Frederic ILLOUZ
- Principal Investigator Email
- frillouz@chu-angers.fr
- Contact Person Name
- Frederic ILLOUZ
- Contact Person Email
- frillouz@chu-angers.fr
Romania
- Earliest CTIS Part Ii Submission Date
- 22-10-2024
- Latest Decision Or Authorization Date
- 14-04-2026
- Processing Time Days
- 539
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- National Institute Of Endocrinology C.I. Parhon
- Department Name
- Sectia Clinica de Endocrinologie IV
- Principal Investigator Name
- Corin-Virgil Badiu
- Principal Investigator Email
- badicrin@yahoo.co.uk
- Contact Person Name
- Corin-Virgil Badiu
- Contact Person Email
- badicrin@yahoo.co.uk
- Site Name
- Centrul National Clinic De Recuperare Neuropsihomotorie Copii Dr. Nicolae Robanescu
- Department Name
- Sectia Neurologie Pediatrica
- Principal Investigator Name
- Madalina Cristina Leanca
- Principal Investigator Email
- mada_mada332@yahoo.com
- Contact Person Name
- Madalina Cristina Leanca
- Contact Person Email
- mada_mada332@yahoo.com
- Site Name
- National Institute Of Endocrinology C.I. Parhon (second entry)
- Department Name
- Sectia Endocrinologie Pediatrica
- Principal Investigator Name
- Iuliana Gherlan
- Principal Investigator Email
- iuliana.gherlan@parhon.ro
- Contact Person Name
- Iuliana Gherlan
- Contact Person Email
- iuliana.gherlan@parhon.ro
- Site Name
- Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara
- Department Name
- Sectia Clinica III Pediatrie
- Principal Investigator Name
- Otilia Marginean
- Principal Investigator Email
- omarginean@ymail.com
- Contact Person Name
- Otilia Marginean
- Contact Person Email
- omarginean@ymail.com
Sweden
- Earliest CTIS Part Ii Submission Date
- 10-01-2025
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 438
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Karolinska University Hospital
- Department Name
- Mottagning Endokrinologi Solna
- Principal Investigator Name
- Charlotte Höybye
- Principal Investigator Email
- charlotte.hoybye@regionstockholm.se
- Contact Person Name
- Charlotte Höybye
- Contact Person Email
- charlotte.hoybye@regionstockholm.se
Czechia
- Earliest CTIS Part Ii Submission Date
- 31-01-2025
- Latest Decision Or Authorization Date
- 25-03-2026
- Processing Time Days
- 418
- Number Of Sites
- 1
- Number Of Participants
- 8
Sites
- Site Name
- Vseobecna Fakultni Nemocnice V Praze
- Department Name
- III. interní klinika – Klinika endokrinologie a metabolismu
- Principal Investigator Name
- Martin Matoulek
- Principal Investigator Email
- martin.matoulek@vfn.cz
- Contact Person Name
- Martin Matoulek
- Contact Person Email
- martin.matoulek@vfn.cz
Poland
- Earliest CTIS Part Ii Submission Date
- 10-01-2025
- Latest Decision Or Authorization Date
- 27-03-2026
- Processing Time Days
- 441
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Uniwersytecki Szpital Kliniczny Nr 1 Im. Prof. Tadeusza Sokolowskiego Pum W Szczecinie
- Department Name
- Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
- Principal Investigator Name
- Elżbieta Petriczko
- Principal Investigator Email
- elzbietapetriczko@gmail.com
- Contact Person Name
- Elżbieta Petriczko
- Contact Person Email
- elzbietapetriczko@gmail.com
- Site Name
- Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
- Department Name
- Klinika Pediatrii, Endokrynologii, Diabetologii i chorób Metabolicznych
- Principal Investigator Name
- Beata Wikiera
- Principal Investigator Email
- wikierab@wp.pl
- Contact Person Name
- Beata Wikiera
- Contact Person Email
- wikierab@wp.pl
Sponsor
Primary sponsor
- Full Name
- Harmony Biosciences LLC
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- PPD Development LP
- Responsibilities
- Multiple trial support responsibilities (sponsor duties codes: 1,10,12,13,2,4,5,6,9) as listed in record
- Name
- Propharma Group LLC
- Responsibilities
- sponsor duty code 8 (clinical safety support contact listed)
- Name
- 4g Clinical LLC
- Responsibilities
- sponsor duty code 3
- Name
- Longboat Clinical Limited
- Responsibilities
- Site Training Portal and Prescreening
Third parties
- {"country":"United Kingdom","full_name":"The Doctors Laboratory","duties_or_roles":"genetic testing; sponsor duty code 4","organisation_type":"Industry"}
- {"country":"United States","full_name":"Propharma Group LLC","duties_or_roles":"sponsor duty code 8","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Signant Health Global Solutions Limited","duties_or_roles":"eCOA, RTQ, Telehealth","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient Travel and Reimbursement management","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"sponsor duty code 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Multiple sponsor duties (codes: 1,10,12,13,2,4,5,6,9) as listed","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Longboat Clinical Limited","duties_or_roles":"Site Training Portal and Prescreening","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"MVZ Medizinisches Labor Nord MLN GmbH","duties_or_roles":"genetic testing; sponsor duty code 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsor duty code 7","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- HBS-101
- Active Substance
- PITOLISANT HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- oral
- Authorisation Status
- authorised (prodAuthStatus=1 for some product entries)
- Frequency
- once daily
- Maximum Dose
- 44.5 mg (maxDailyDoseAmount)
- Investigational Product Name
- WAKIX
- Active Substance
- PITOLISANT HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- oral
- Authorisation Status
- authorised (prodAuthStatus=1 for some product entries)
- Frequency
- once daily
- Maximum Dose
- 44.5 mg (maxDailyDoseAmount)
- Investigational Product Name
- Matching placebo (tablets)
- Modality
- Other
- Frequency
- once daily (matching placebo during 77-day Double-Blind Treatment Period)
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- INFIGRATINIB for Hypochondroplasia
- BMN 333 for Achondroplasia
- INFIGRATINIB for Hypochondroplasia
- NAVENIBART for Hereditary angioedema|Hereditary angioedema type I|Hereditary angioedema type II
- Autologous haematopoietic stem and progenitor cell population containing CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA ex vivo expanded for Autosomal recessive osteopetrosis (TCIRG1 mutation)