Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
186
Countries
5
Sites
33
Turner Syndrome (TS)
To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents
Key facts
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- 15 Apr 2026 → ongoing
- Decision date (initial)
- 2026-02-27
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents
Secondary objectives 2
- To evaluate lonapegsomatropin as compared to somatropin on additional measures of efficacy in children and adolescents
- To evaluate the safety and tolerability of lonapegsomatropin in children and adolescents
Conditions and MedDRA coding
Turner Syndrome (TS)
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Chronological age between ≥2 and <18 years, at start of screening.
- Naïve to growth hormone and growth hormone promoting therapies.
- Prepubertal.
- 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 5
- Advanced bone age X-ray by central reading defined as >20% above chronological age in months (Greulich 1959).
- Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males.
- 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.
- 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.
- 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 and secondary outcome measures (English text)
Primary endpoints 1
- Annualized height velocity (AHV) at Week 52
Secondary endpoints 7
- AHV (Week 104)
- Change from baseline in height SDS (CDC-based) (Weeks 52 and 104)
- Change from baseline in height SDS (condition specific if applicable) (Weeks 52 and 104)
- Treatment-emergent adverse events (AEs)
- Safety labs, vital signs, physical exam, local tolerability assessments, and fundoscopy
- Bone age (Weeks 52 and 104)
- Bone age:chronological age ratio (Weeks 52 and 104)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 14
PRD12890314 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890317 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890306 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890309 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890315 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890311 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890312 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890307 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890310 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890318 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890308 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890305 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890316 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
PRD12890313 · Product
- Active substance
- Lonapegsomatropin
- Substance synonyms
- TRANSCONPEG-HGH, RHGH-PEG, ACP-011, TRANSCON PEG40 HGH
- Pharmaceutical form
- POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 104 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S
- Paediatric formulation
- No
- Orphan designation
- No
Comparator 1
Norditropin FlexPro, injektionsvæske, opløsning i fyldt pen
PRD341428 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 52 Week(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- 45069
- MA holder
- NOVO NORDISK A/S
- MA country
- Denmark
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ascendis Pharma Endocrinology Division A/S
- Sponsor organisation
- Ascendis Pharma Endocrinology Division A/S
- Address
- Tuborg Boulevard 12
- City
- Hellerup
- Postcode
- 2900
- Country
- Denmark
Scientific contact point
- Organisation
- Ascendis Pharma Endocrinology Division A/S
- Contact name
- Clinical Trial Information Desk
Public contact point
- Organisation
- Ascendis Pharma Endocrinology Division A/S
- Contact name
- Clinical Trial Information Desk
Third parties 13
| Organisation | City, country | Duties |
|---|---|---|
| GBA Central Lab Services GmbH ORG-100017343
|
Schwentinental, Germany | Other |
| Clario Medical Imaging Inc. ORG-100052770
|
Seattle, United States | Other |
| Icon (Lr) Limited ORG-100042612
|
Dublin 18, Ireland | On site monitoring, Code 12, Other, Code 5, Code 8 |
| Icon Laboratory Services Inc. ORG-100037135
|
Farmingdale, United States | Other |
| SVAR Life Science AB ORG-100046037
|
Malmo, Sweden | Other |
| Icon (Lr) Limited ORG-100042612
|
Dublin 18, Ireland | Laboratory analysis |
| Bioagilytix Labs LLC ORG-100013030
|
Durham, United States | Other |
| Centogene GmbH ORG-100043695
|
Rostock, Germany | Other |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| 4G Clinical B.V. ORG-100044721
|
Amsterdam, Netherlands | Other |
| Veeva Systems Inc. ORG-100006053
|
Pleasanton, United States | E-data capture |
| Cognizant Technology Solutions India Private Limited ORG-100012904
|
Navi Mumbai, India | Other |
| Celerion Inc. ORG-100029202
|
Lincoln, United States | Other |
Locations
5 EU/EEA countries · 33 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 14 | 7 |
| Germany | Authorised, recruiting | 14 | 9 |
| Italy | Authorised, recruitment pending | 18 | 8 |
| Romania | Authorised, recruitment pending | 6 | 3 |
| Spain | Ongoing, recruiting | 14 | 6 |
| Rest of world
United States, Korea, Republic of
|
— | 120 | — |
Investigational sites
Groupe D’Etude Des Histiocytoses
Service de Physiologie – Explorations fonctionnelles pédiatriques, 26 Avenue Du Docteur Arnold Netter, 75012, Paris
Centre Hospitalier Universitaire De Toulouse
Endocrinology, Genetics and Medical Gynecology, 330 Avenue De Grande Bretagne, 31059, Toulouse Cedex 9
Assistance Publique Hopitaux De Paris
Pediatric Endocrinology and Diabetes for Children, 78 Rue Du General Leclerc, 94270, Le Kremlin-Bicetre
Centre Hospitalier Universitaire D'Angers
Pediatric Endocrinology, 4 Rue Larrey, 49100, Angers
Centre Hospitalier Universitaire De Montpellier
Nephrology and endocrinology unit – multidisciplinary pediatrics department, 371 Avenue Du Doyen Gaston Giraud, 34090, Montpellier
Hospices Civils De Lyon
ediatric endocrinology and diabetology, 59 Boulevard Pinel, 69500, Bron
Assistance Publique Hopitaux De Paris
Service d’endocrinologie diabétologie pédiatrique, 48 Boulevard Serurier, 75019, Paris
Medical Center - University Of Freiburg
Children and Adolescent Clinic – Pediatric Endorcinology and Diabetology, Breisacher Strasse 62, Stuehlinger, Freiburg Im Breisgau
Universitaetsklinikum Augsburg
Clinic for Children and Adolescent Medicine, Stenglinstrasse 2, Kriegshaber, Augsburg
Universitaetsklinikum Erlangen AöR
Children and Adolescent Clinic, Loschgestrasse 15, Innenstadt, Erlangen
Universitaetsklinikum Tuebingen AöR
Clinic for Children and Adolescent Medicine, Hoppe-Seyler-Strasse 1, Nordstadt, Tuebingen
Universitaetsklinikum Koeln AöR
Clinic and Polyclinic for Pediatrics and Adolescent Medicine, Kerpener Strasse 62, Lindenthal, Cologne
Universitaetsklinikum Duesseldorf AöR
Department of General Pediatrics, Neonatology and Pediatric Cardiology, Moorenstrasse 5, Bilk, Duesseldorf
Otto Von Guericke Universitaet Magdeburg
Pediatric Endorcinology and Diabetology, Leipziger Strasse 44, Leipziger Str., Magdeburg
Hannoversche Kinderheilanstalt
Diabetology, endocrinology, gastroenteology and clinical research, Janusz Korczak Avenue 12, Bult, Hanover
Universitaet Des Saarlandes
Division of Pediatric Endorcinology and Diabetes, Kirrberger Strasse 100, 66421, Homburg
Azienda Ospedaliera Universitaria Federico II Di Napoli
Pediatrics Endocrinology, Via Sergio Pansini 5, 80131, Naples
Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
Pediatrics Unit, Via Dell' Istria 65/1, 34137, Trieste
Ospedale Pediatrico Bambino Gesu
Endocrinology and Diabetes Unit, Piazza Di Sant'onofrio 4, 00165, Rome
Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
Department of Woman, child, general and specialized surgery, Via Santa Maria Di Costantinopoli 104, 80138, Naples
Azienda Ospedaliera Universitaria Gaetano Martino Messina
DAI Materno- Intfantile - UOC Pediatria, Via Consolare Valeria N 1, 98124, Messina
IRCCS Istituto Giannina Gaslini
UOC Clinica Pediatrica Endocrinologia, Via Gerolamo Gaslini 5, 16147, Genoa
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Struttura Complessa Pediatria Specialistica U, Piazza Polonia 94, 10126, Turin
Azienda Ospedaliera Universitaria Meyer IRCCS
Unità Auxoendocrinologia, Viale Gaetano Pieraccini 24, 50139, Florence
Spitalul Clinic Judetean De Urgenta Sf. Spiridon Iasi
Endocrinology, Bulevardul Independentei 1, 700111, Jassi
Spitalul Clinic Judetean Mures
Endocrinology, Str Gh Marinescu Nr 46, 540136, Targu Mures
National Institute Of Endocrinology C.I. Parhon
Endocrinology, Bulevardul Aviatorilor 34-38, 011863, Bucharest
Hospital Universitario Regional De Malaga
Pediatric endocrinology, Avenida De Carlos De Haya S/N, 29010, Malaga
Hospital Universitario Miguel Servet
Pediatric endocrinology, Paseo De Isabel La Catolica 1-3, 50009, Zaragoza
Hospital Universitari Vall D Hebron
Pediatric endocrinology, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
Complexo Hospitalario Universitario De Santiago
Pediatric endocrinology, Calle Choupana Da S/n, 15706, Santiago De Compostela
Hospital Sant Joan De Deu Barcelona
Pediatric endocrinology, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Hospital Universitario La Paz
Pediatric endocrinology, Paseo De La Castellana 261, 28046, Madrid
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2026-05-11 | 2026-05-12 | |||
| Germany | 2026-05-19 | ||||
| Spain | 2026-04-15 | 2026-05-25 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 81 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2025-523079-44-00 Redacted | 3.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Lonapegsomatropin_DE | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Lonapegsomatropin_ES | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Lonapegsomatropin_FR | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Lonapegsomatropin_IT | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Lonapegsomatropin_RO | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Somatropin_DE | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Somatropin_ES | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Somatropin_FR | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Somatropin_IT | 1.0 |
| Protocol (for publication) | D4_Patient facing documents_Participant Diary_Somatropin_RO | 1.0 |
| Recruitment arrangements (for publication) | K1_DE_Recruitment Procedure | 2.0 |
| Recruitment arrangements (for publication) | K1_ES_Recruitment Procedure | 2.0 |
| Recruitment arrangements (for publication) | K1_FR_Additional Document_French_redacted | N/A |
| Recruitment arrangements (for publication) | K1_FR_Recruitment Procedure_Bilingual | 2.0 |
| Recruitment arrangements (for publication) | K1_IT_Recruitment Procedure | N/A |
| Recruitment arrangements (for publication) | K1_RO_Recruitment Procedure | 2.0 |
| Recruitment arrangements (for publication) | K2_DE_Recruitment Material_Clinician Card_German_redacted | N/A |
| Recruitment arrangements (for publication) | K2_DE_Recruitment Material_Patient Facing Card_German | N/A |
| Recruitment arrangements (for publication) | K2_DE_Recruitment Material_Pocket Referral Card_German | N/A |
| Recruitment arrangements (for publication) | K2_ES_Recruitment material_Clinician Card_Spanish_redacted | N/A |
| Recruitment arrangements (for publication) | K2_ES_Recruitment material_Patient card_Spanish | N/A |
| Recruitment arrangements (for publication) | K2_ES_Recruitment material_Pocket Referral Card_Spanish | N/A |
| Recruitment arrangements (for publication) | K2_FR_Recruitment Material_HighLiGHts Clinician Card_French_redacted | N/A |
| Recruitment arrangements (for publication) | K2_FR_Recruitment Material_HighLiGHts Patient Facing Card_French | N/A |
| Recruitment arrangements (for publication) | K2_FR_Recruitment Material_HighLiGHts Pocket Referral Card_French | N/A |
| Recruitment arrangements (for publication) | K2_IT_Recruitment Material_Clinician Card_Italian_redacted | N/A |
| Recruitment arrangements (for publication) | K2_IT_Recruitment Material_Patient Facing Card_Italian | N/A |
| Recruitment arrangements (for publication) | K2_IT_Recruitment Material_Pocket Referral Card_Italian | N/A |
| Recruitment arrangements (for publication) | K2_RO_Recruitment Material_Clinician Card_Romanian_redacted | N/A |
| Recruitment arrangements (for publication) | K2_RO_Recruitment Material_Patient Facing Card_Romanian | N/A |
| Recruitment arrangements (for publication) | K2_RO_Recruitment Material_Pocket Referral Card_Romanian | N/A |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Assent Children aged 12-17 Years Old_German_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Future Research_German_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Parent and Legal Guardian_German_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Participant of Legal Age_German_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Pregnant Participant_German_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Pregnant Partner_German_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Scout_German | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Assent (13-17)_Spanish_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Assent (up to 8)_Spanish | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Assent 9-12_Spanish_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Parent-Guardian_Spanish_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Scout_Spanish | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Assent Children 13 Years and older_French_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Assent Children aged 7-11 Years Old_German | 1.1 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Assent Children aged 9-12 Years Old_French_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Assent Children up to 8 Years Old_French | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Parent and Legal Guardian_French_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Participant of Legal Age_French_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Pregnant Participant_French_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Pregnant Partner_French_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Scout Clinical Standalone Consent_French | 1.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Assent 12-17_Italian_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Assent 6-11_Italian | 1.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Data Privacy ICF_Italian_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Parents_Italian_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Scout_Italian | 1 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Parent_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Parent_Romanian_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_11-14 yrs_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_11-14 yrs_Romanian_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_15 yrs-AoM_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_15 yrs-AoM_Romanian_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_7-10 yrs | 2.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_7-10 yrs_Romanian | 2.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_Up to 6 yrs | 3.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_Pediatric Assent ICF_Up to 6 yrs_Romanian | 3.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_SCOUT | 2.0 |
| Subject information and informed consent form (for publication) | L1_RO_SIS-ICF_SCOUT_Romanian | 2.0 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Norditropin | 1.0 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Skytrofa | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis 2025-523079-44-00_Layperson | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ES 2025-523079-44-00_Layperson | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ES 2025-523079-44-00_Scientific Redacted | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_FR 2025-523079-44-00_Layperson | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_FR 2025-523079-44-00_Scientific Redacted | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_IT 2025-523079-44-00_Layperson | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_IT 2025-523079-44-00_Scientific Redacted | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_RO 2025-523079-44-00_Layperson | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_RO 2025-523079-44-00_Scientific Redacted | 2.0 |
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-10-29 | Germany | Acceptable 2026-02-27
|
2026-02-27 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-03-24 | Germany | Acceptable 2026-02-27
|
2026-03-24 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-03-25 | Germany | Acceptable 2026-02-27
|
2026-03-25 |
| 4 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-03-30 | Acceptable | 2026-05-18 | |
| 5 | SUBSTANTIAL MODIFICATION | SM-2 | 2026-03-30 | Germany | Acceptable | 2026-05-04 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2026-03-30 | Acceptable | 2026-05-25 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-4 | 2026-03-31 | Acceptable | 2026-05-18 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-04-01 | Acceptable | 2026-05-07 |