Clinical trial • Phase III • Musculoskeletal
LONAPEGSOMATROPIN for Turner syndrome | Idiopathic short stature | SHOX deficiency | Small for gestational age
Phase III trial of LONAPEGSOMATROPIN for Turner syndrome | Idiopathic short stature | SHOX deficiency | Small for gestational age.
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Turner syndrome | Idiopathic short stature | SHOX deficiency | Small for gestational age
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 29-10-2025
- First CTIS Authorization Date
- 27-02-2026
Trial design
Randomised, open-label, norditropin flexpro (somatropin) — daily somatropin (active comparator; referred to as daily somatropin in title/documentation).-controlled Phase III trial in France, Germany, Italy and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Norditropin FlexPro (somatropin) — daily somatropin (active comparator; referred to as daily somatropin in title/documentation).
- Target Sample Size
- 120
- Trial Duration For Participant
- 728
Eligibility
Recruits 120 paediatric patients.
- Vulnerable Population
- Children and adolescents are included (isVulnerablePopulationSelected = true). The trial uses age‑specific assent and consent documents: parent/legal guardian informed consent forms and multiple pediatric assent forms stratified by age (examples in the dataset: assent forms for 'up to 6 yrs', '7-10 yrs', '11-14 yrs', '12-17 yrs', '13 years and older', and 'children up to 8 years'). Scout Clinical standalone consent is available. Documents are provided in multiple languages (French, German, Italian, Romanian, Spanish) as per country-specific SIS/ICF files.
Inclusion criteria
- {"criterion_text":"- Chronological age between ≥2 and <18 years, at start of screening."}
- {"criterion_text":"- Naïve to growth hormone and growth hormone promoting therapies."}
- {"criterion_text":"- Prepubertal."}
- {"criterion_text":"- Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria: TS or SHOX-D (Léri-Weill dyschondrosteosis) a. Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis. For karyotypes, a minimum of 20 cells must be counted. b. Impaired growth or short stature defined as: (i.) AHV <25th percentile over a time span of 6-16 months prior to screening utilizing a historical height properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height <5th percentile for sex and age according to the Centers for Disease Control Growth Charts for the United States SGA without catch-up growth c. Birth weight and/or birth length < -2.0 SDS for gestational age according to the 2006 World Health Organization Child Growth Standards. For infants born premature, the Fenton Preterm Infant Growth Chart (Fenton 2013) should be used. d. Impaired growth or short stature defined as: (i.) AHV <25th percentile over a time span of 6-16 months prior to screening properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height < -2.0 SDS for age and sex according to the 2000 Centers for Disease Control Growth Charts for the United States for children ≥ 3 years or height < -2.5 SDS for age and sex according to the for children ≥ 2 years and < 3 years ISS e. Height < -2.25 SDS for sex and age according to the Centers for Disease Control Growth Charts for the United States with no identifiable cause for short stature. f. Documented normal GH-IGF-1 axis, defined as either: (i.) IGF-1 SDS >0 at screening based on central laboratory OR (ii.) Historical documentation of normal peak GH upon stimulation test (as defined by local institution) g. 46,XX chromosome as determined by karyotype or microarray if female. For karyotypes, a minimum of 30 cells must be counted."}
Exclusion criteria
- {"criterion_text":"- Advanced bone age X-ray by central reading defined as >20% above chronological age in months (Greulich 1959)."}
- {"criterion_text":"- Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males."}
- {"criterion_text":"- Exclusion Criteria only applicable to TS: a. Presence of Y chromosome material on genetic testing without history of gonadectomy. b. Less than 10% of 45,X mosaicism. c. Any known, clinically significant, congenital or acquired cardiovascular dysfunction that might interfere with growth."}
- {"criterion_text":"- Exclusion Criteria only applicable to SGA: a. Any known clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: (i.) Chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors OR (ii) Congenital abnormalities (causing skeletal abnormalities), including but not limited to skeletal dysplasias."}
- {"criterion_text":"- Exclusion Criteria only applicable to ISS: a. Known history of any condition that causes disproportionate short stature (i.e. skeletal dysplasias), chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Annualized height velocity (AHV) at Week 52","definition_or_measurement_approach":"Annualized height velocity measured at Week 52 (AHV at Week 52)."}
Secondary endpoints
- {"endpoint_text":"- AHV (Week 104)","definition_or_measurement_approach":"Annualized height velocity measured at Week 104."}
- {"endpoint_text":"- Change from baseline in height SDS (CDC-based) (Weeks 52 and 104)","definition_or_measurement_approach":"Change from baseline in height standard deviation score using CDC growth charts evaluated at Weeks 52 and 104."}
- {"endpoint_text":"- Change from baseline in height SDS (condition specific if applicable) (Weeks 52 and 104)","definition_or_measurement_approach":"Change from baseline in height SDS using condition‑specific references where applicable, assessed at Weeks 52 and 104."}
- {"endpoint_text":"- Treatment-emergent adverse events (AEs)","definition_or_measurement_approach":"Recording and summarising treatment-emergent adverse events during the study period."}
- {"endpoint_text":"- Safety labs, vital signs, physical exam, local tolerability assessments, and fundoscopy","definition_or_measurement_approach":"Standard safety assessments including laboratory tests, vital signs, physical examinations, local tolerability checks and fundoscopy evaluations."}
- {"endpoint_text":"- Bone age (Weeks 52 and 104)","definition_or_measurement_approach":"Bone age assessment at Weeks 52 and 104 (central reading referenced elsewhere)."}
- {"endpoint_text":"- Bone age:chronological age ratio (Weeks 52 and 104)","definition_or_measurement_approach":"Ratio of bone age to chronological age evaluated at Weeks 52 and 104."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Groupe D’Etude Des Histiocytoses
- Planned Sample Size
- 120
- Recruitment Window Months
- 36
- Consent Approach
- Informed consent from parent(s)/legal guardian(s) for minors with multiple age-specific pediatric assent forms used depending on participant age (examples: assent forms for 'up to 6 yrs', '7-10 yrs', '11-14 yrs', '12-17 yrs', '13 years and older', and parent/legal guardian forms). Scout Clinical standalone consent is provided for certain procedures. Subject information and informed consent documents are available in multiple languages per country (French, German, Italian, Romanian, Spanish as evidenced by country-specific SIS/ICF documents).
Methods
- Distribution of clinician referral cards and patient-facing cards (country-specific K2 recruitment materials; e.g. Germany and Spain documents named 'K2_* Recruitment Material').
- Site-based recruitment at pediatric endocrinology and university hospital clinics (multiple sites listed per country in trialSites).
- Country-specific recruitment procedures documented (K1 recruitment procedure documents available for France, Italy, Romania, Germany, Spain).
- Use of a patient organisation as contact/partner at least in France (site entry for 'Groupe D’Etude Des Histiocytoses').
- Third-party vendor support for travel, accommodation arrangement and patient reimbursement provided by Scout Clinical (explicit sponsor duty).
Geography
- Total Number Of Sites
- 25
- Total Number Of Participants
- 66
France
- Earliest CTIS Part Ii Submission Date
- 12-12-2025
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 77
- Number Of Sites
- 3
- Number Of Participants
- 14
Sites
- Site Name
- Groupe D’Etude Des Histiocytoses
- Department Name
- Service de Physiologie – Explorations fonctionnelles pédiatriques
- Contact Person Name
- Muriel HOUANG
- Contact Person Email
- muriel.houang@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- Endocrinology, Genetics and Medical Gynecology
- Contact Person Name
- Thomas EDOUARD
- Contact Person Email
- Edouard.t@chu-toulouse.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Pediatric Endocrinology and Diabetes for Children
- Contact Person Name
- Agnès LINGLART
- Contact Person Email
- Agnes.linglart@aphp.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 16-01-2026
- Latest Decision Or Authorization Date
- 04-05-2026
- Processing Time Days
- 108
- Number Of Sites
- 9
- Number Of Participants
- 14
Sites
- Site Name
- Universitaetsklinikum Erlangen AöR
- Department Name
- Children and Adolescent Clinic
- Contact Person Name
- Joachim Wölfle
- Contact Person Email
- Joachim.Woelfle@uk-erlangen.de
- Site Name
- Universitaet Des Saarlandes
- Department Name
- Division of Pediatric Endorcinology and Diabetes
- Contact Person Name
- Tilman Rohrer
- Contact Person Email
- tilman.rohrer@uks.eu
- Site Name
- Otto Von Guericke Universitaet Magdeburg
- Department Name
- Pediatric Endorcinology and Diabetology
- Contact Person Name
- Katja Palm
- Contact Person Email
- Katja.palm@med.ovgu.de
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Clinic for Children and Adolescent Medicine
- Contact Person Name
- Gerhard Binder
- Contact Person Email
- Gerhard.Binder@med.unituebingen.de
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- Children and Adolescent Clinic – Pediatric Endorcinology and Diabetology
- Contact Person Name
- Clemens Kamrath
- Contact Person Email
- clemens.kamrath@uniklinik-freiburg.de
- Site Name
- Hannoversche Kinderheilanstalt
- Department Name
- Diabetology, endocrinology, gastroenteology and clinical research
- Contact Person Name
- Felix Reschke
- Contact Person Email
- Felix.reschke@hka.de
- Site Name
- Universitaetsklinikum Koeln AöR
- Department Name
- Clinic and Polyclinic for Pediatrics and Adolescent Medicine
- Contact Person Name
- Heike Hoyer-Kuhn
- Contact Person Email
- heike-katharina.hoyerkuhn@uk-koeln.de
- Site Name
- Universitaetsklinikum Duesseldorf AöR
- Department Name
- Department of General Pediatrics, Neonatology and Pediatric Cardiology
- Contact Person Name
- Sebastian Kummer
- Contact Person Email
- Sebastian.kummer@med.uniduesseldorf.de
- Site Name
- Universitaetsklinikum Augsburg
- Department Name
- Clinic for Children and Adolescent Medicine
- Contact Person Name
- Desiree Dunstheimer
- Contact Person Email
- desiree.dunstheimer@uk-augsburg.de
Italy
- Earliest CTIS Part Ii Submission Date
- 05-02-2026
- Latest Decision Or Authorization Date
- 02-03-2026
- Processing Time Days
- 25
- Number Of Sites
- 4
- Number Of Participants
- 18
Sites
- Site Name
- Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
- Department Name
- Pediatrics Unit
- Contact Person Name
- Gianluca Tornese
- Contact Person Email
- gianluca.tornese@burlo.trieste.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- Endocrinology and Diabetes Unit
- Contact Person Name
- Stefano Cianfarani
- Contact Person Email
- stefano.cianfarani@uniroma2.it
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Pediatrics Endocrinology
- Contact Person Name
- Donatella Capalbo
- Contact Person Email
- donatella.capalbo@unina.it
- Site Name
- Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
- Department Name
- Department of Woman, child, general and specialized surgery
- Contact Person Name
- Anna Grandone
- Contact Person Email
- anna.grandone@unicampania.it
Romania
- Earliest CTIS Part Ii Submission Date
- 27-02-2026
- Latest Decision Or Authorization Date
- 09-03-2026
- Processing Time Days
- 10
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Spitalul Clinic Judetean De Urgenta Sf. Spiridon Iasi
- Department Name
- Endocrinology
- Contact Person Name
- Cristina Preda
- Contact Person Email
- contact@spitalspiridon.ro
- Site Name
- Spitalul Clinic Judetean Mures
- Department Name
- Endocrinology
- Contact Person Name
- Maria Ionela Pascanu
- Contact Person Email
- secretariat@spitaljudeteanmures.ro
Spain
- Earliest CTIS Part Ii Submission Date
- 05-12-2025
- Latest Decision Or Authorization Date
- 07-05-2026
- Processing Time Days
- 153
- Number Of Sites
- 7
- Number Of Participants
- 14
Sites
- Site Name
- Hospital Universitario Miguel Servet
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Antonio de Arriba Muñoz
- Contact Person Email
- adearriba@salud.aragon.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Maria Clemente Leon
- Contact Person Email
- maria.clemente@vallhebron.cat
- Site Name
- Hospital Universitario Regional De Malaga
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Isabel Leiva Gea
- Contact Person Email
- isabeleivag@gmail.com
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Paloma Cabanas
- Contact Person Email
- paloma.cabanas.rodriguez@sergas.es
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Silvia Marin del Barrio
- Contact Person Email
- silvia.marin@sjd.es
- Site Name
- Hospital Universitario La Paz
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Isabel Gonzalez Casado
- Contact Person Email
- igcasado@salud.madrid.org
- Site Name
- Hospital Universitario Miguel Servet (additional listing)
- Department Name
- Pediatric endocrinology
- Contact Person Name
- Antonio de Arriba Muñoz
- Contact Person Email
- adearriba@salud.aragon.es
Sponsor
Primary sponsor
- Full Name
- Ascendis Pharma Endocrinology Division A/S
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Denmark
Contract research organisations
- Name
- Icon (Lr) Limited
- Responsibilities
- Multiple duties including site platform solutions and other operational activities (codes 1,12,15,5,8 listed in sponsor duties).
- Name
- Icon Laboratory Services Inc.
- Responsibilities
- Laboratory services / endocrine laboratory support.
- Name
- 4G Clinical B.V.
- Responsibilities
- IRT (interactive response technology) management.
- Name
- Veeva Systems Inc.
- Responsibilities
- Clinical data management / systems (code 7).
- Name
- Cognizant Technology Solutions India Private Limited
- Responsibilities
- Pharmacovigilance vendor activities.
Third parties
- {"country":"Germany","full_name":"GBA Central Lab Services GmbH","duties_or_roles":"Central PD Lab","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Clario Medical Imaging Inc.","duties_or_roles":"Medical image analysis / Review X-ray","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon (Lr) Limited","duties_or_roles":"Codes: 1,12,15 (Site platform solutions),5,8","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"Endocrinology","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Sweden","full_name":"SVAR Life Science AB","duties_or_roles":"Immunogenicity assessments","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon (Lr) Limited (alternate address entry)","duties_or_roles":"Code: 4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Immunogenicity assessments","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"Genetic testing","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel / accommodation arrangement and patient reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"IRT","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"Code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"India","full_name":"Cognizant Technology Solutions India Private Limited","duties_or_roles":"PV vendor","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Celerion Inc.","duties_or_roles":"Pharmacokinetic Analysis","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Lonapegsomatropin
- Active Substance
- LONAPEGSOMATROPIN
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS
- Route
- SUBCUTANEOUS
- Authorisation Status
- prodAuthStatus: 1
- Frequency
- Once-weekly (per trial title for investigational arm)
- Investigational Product Name
- Norditropin FlexPro (somatropin)
- Active Substance
- SOMATROPIN
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS
- Route
- SUBCUTANEOUS
- Authorisation Status
- prodAuthStatus: 2 (marketing authorisation present, marketingAuthNumber: 45069 in Denmark)
- Frequency
- Daily (comparator described as daily somatropin)
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