Clinical trial • Phase III • Neurology|Rare Disease
ROZANOLIXIZUMAB for Generalized myasthenia gravis
Phase III trial of ROZANOLIXIZUMAB for Generalized myasthenia gravis. open-label. 7 participants.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Generalized myasthenia gravis
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody|Peptide/protein/enzyme
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 03-10-2023
- First CTIS Authorization Date
- 09-02-2024
Trial design
open-label Phase III trial across 5 sites in Poland, Italy.
- Open Label
- Yes
- Target Sample Size
- 7
- Trial Duration For Participant
- 365
Eligibility
Recruits 7 paediatric patients.
- Vulnerable Population
- Includes pediatric participants (aged ≥2 at the time of MG0006 screening). Vulnerable population considerations: parental consent forms and age-specific assent forms are provided (documents include assent for ages 6-11, assent for ages 12-17, parental/guardian ICFs, and adult/participant ICFs). Country-specific ICF procedures and bilingual/translated ICFs are available (Polish and Italian sets present; English ICF procedure for Italy is available). The trial record flags vulnerable population selection = true.
Inclusion criteria
- {"criterion_text":"-\tStudy participant must meet one of the following: -\t−Study participant completed MG0006 according to the protocol -\t−Study participant completed the MG0006 Treatment Period and has a worsening of gMG symptoms in the Observation Period of MG0006"}
Exclusion criteria
- {"criterion_text":"-\tStudy participant met any mandatory withdrawal or mandatory permanent IMP discontinuation criteria in MG0006 or permanently discontinued IMP -\tStudy participant has a known hypersensitivity to any components of the IMP or other FcRn drugs -\tStudy participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant’s ability to participate in this study"}
Endpoints
Primary endpoints
- {"endpoint_text":"-\tOccurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit -\tOccurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit -\tOccurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit","definition_or_measurement_approach":"Occurrence of the listed safety events recorded and assessed up to the End of Study (EOS) Visit; measured as incidence/occurrence counts of serious TEAEs, TEAEs leading to permanent IMP withdrawal, and AESMs up to EOS."}
Secondary endpoints
- {"endpoint_text":"-\tPercent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP) -\tAbsolute change in total IgG from Baseline to the end of Week 6 of each TP -\tChange from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP -\tChange from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP","definition_or_measurement_approach":"Endpoints measured as change from Baseline to end of Week 6 of each Treatment Period (TP): percent and absolute change in total IgG; change in MG-ADL total score; change in QMG total score. Measurements taken at Baseline and at Week 6 of each TP per protocol schedules."}
Recruitment
- Registry Or Advocacy Recruitment
- True - Center For Information And Study On Clinical Research Participation Inc.
- Digital Remote Recruitment
- True - study materials and ICFs reference a 'Little Journey' app (documents include 'parent little journey app' and 'LJ app' ICFs), indicating use of a digital app for participant/parent interaction or e-consent/assent in some countries.
- Planned Sample Size
- 7
- Recruitment Window Months
- 46
- Consent Approach
- Informed consent and assent: parental/guardian consent required for minors; assent forms provided for younger participants (documents indicate assent 6-11 years and assent 12-17 years). Separate participant/adult ICFs and parental ICFs are available. Country-specific ICF procedures and bilingual/translated ICFs are provided (Polish and Italian ICFs present; an English Country ICF Procedure for Italy is present). Public/sponsor contact (UCB Cares) is listed for queries.
Methods
- Site-based recruitment through participating hospitals/clinics in Italy and Poland (documents: 'ITA Recruitment Other Italian MG0008 Public' and 'POL-Recruitment Flyer-Polish-MG0008-Public' are present).
- Country-specific recruitment materials/flyers and recruitment arrangement documents available for Poland and Italy (document titles listed in CTIS documents).
Geography
- Total Number Of Sites
- 5
- Total Number Of Participants
- 7
Poland
- Earliest CTIS Part Ii Submission Date
- 09-01-2024
- Latest Decision Or Authorization Date
- 09-01-2026
- Processing Time Days
- 731
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
- Department Name
- #40155: Klinika Neurologii
- Principal Investigator Name
- Anna Kostera-Pruszczyk
- Principal Investigator Email
- anna.kostera-pruszczyk@wum.edu.pl
- Contact Person Name
- Anna Kostera-Pruszczyk
- Contact Person Email
- anna.kostera-pruszczyk@wum.edu.pl
- Site Name
- Amicare Sp. z o.o. S.K.
- Department Name
- #40734: Neurology
- Principal Investigator Name
- Łukasz Przysło
- Principal Investigator Email
- l.przyslo@amicare.pl
- Contact Person Name
- Łukasz Przysło
- Contact Person Email
- l.przyslo@amicare.pl
Italy
- Earliest CTIS Part Ii Submission Date
- 10-01-2024
- Latest Decision Or Authorization Date
- 16-01-2026
- Processing Time Days
- 737
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Azienda Ospedaliera Santobono Pausilipon
- Department Name
- #40733: UOC Neuroliga Pediatrica – Dipartimento di Neuroscienze
- Principal Investigator Name
- Antonio Varone
- Principal Investigator Email
- varone@unina.it
- Contact Person Name
- Antonio Varone
- Contact Person Email
- varone@unina.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- #40144: Dipartimento Neuroscienze PediatricheSC Neuropsichiatria Infantile
- Principal Investigator Name
- Isabella Moroni
- Principal Investigator Email
- isabella.moroni@istituto-besta.it
- Contact Person Name
- Isabella Moroni
- Contact Person Email
- isabella.moroni@istituto-besta.it
- Site Name
- Azienda Unita Sanitaria Locale Di Bologna
- Department Name
- #40290: UOC Neuropsichiatria dell'età pediatrica
- Principal Investigator Name
- Anna Fetta
- Principal Investigator Email
- anna.fetta2@unibo.it
- Contact Person Name
- Anna Fetta
- Contact Person Email
- anna.fetta2@unibo.it
Sponsor
Primary sponsor
- Full Name
- UCB Biopharma
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- sponsorDuties codes: [1,11,12,13,2,5,6,8]; contact: april.taylor@parexel.com
- Name
- Medidata Solutions International Limited
- Responsibilities
- sponsorDuties codes: [7]; contact: dimpal.singh@medidata.com
- Name
- 4G Clinical B.V.
- Responsibilities
- sponsorDuties codes: [3]; contact: dave@4gclinical.com
- Name
- IQVIA Laboratories LLC
- Responsibilities
- sponsorDuties codes: [4]; contact: aniket.balki@iqvia.com
- Name
- Certara USA Inc.
- Responsibilities
- sponsorDuties codes: [7]; contact: ryan.miller@certara.com
- Name
- Drug Development Solutions Limited
- Responsibilities
- sponsorDuties codes: [4]; contact: groberts@resolian.com
Third parties
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: [1,11,12,13,2,5,6,8]; contact: april.taylor@parexel.com","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Medidata Solutions International Limited","duties_or_roles":"sponsorDuties codes: [7]; contact: dimpal.singh@medidata.com","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"sponsorDuties codes: [3]; contact: dave@4gclinical.com","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Belgium","full_name":"CluePoints","duties_or_roles":"sponsorDuties codes: [15] (Data quality assessment services); contact: contact@cluepoints.com","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"BioAgilytix Europe GmbH","duties_or_roles":"sponsorDuties codes: [4]; contact: christpher.tiedje@bioagilytix.com","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"sponsorDuties codes: [4]; contact: groberts@resolian.com","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"sponsorDuties codes: [15] (Scales & Linguistic validation services); contact: Mary.Smith@rws.com","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Advarra Inc.","duties_or_roles":"sponsorDuties codes: [15] (Site document repository); contact: charley.Colling@advarra.com","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Poland","full_name":"Curandus sp. z o.o.","duties_or_roles":"sponsorDuties codes: [15] (Site staff augmentation services, patients travel costs reimbursement services); contact: Malgorzata.Lezniak@accellacare.com","organisation_type":"Health care"}
- {"country":"United States","full_name":"Certara USA Inc.","duties_or_roles":"sponsorDuties codes: [7]; contact: ryan.miller@certara.com","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Center For Information And Study On Clinical Research Participation Inc.","duties_or_roles":"sponsorDuties codes: [11]; contact: mscanlon@ciscrp.org","organisation_type":"Patient organisation/association"}
- {"country":"United States","full_name":"IQVIA Laboratories LLC","duties_or_roles":"sponsorDuties codes: [4]; contact: aniket.balki@iqvia.com","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- Rozanolixizumab
- Active Substance
- ROZANOLIXIZUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- SUBCUTANEOUS USE
- Authorisation Status
- prodAuthStatus=1
- Orphan Designation
- Yes
- Investigational Product Name
- Immunoglobulins, normal human
- Active Substance
- Immunoglobulins, normal human
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- UNKNOWN USE
- Route
- UNKNOWN USE
- Authorisation Status
- prodAuthStatus=2
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