Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ended
Participants planned
40
Countries
1
Sites
5
Growth Hormone Deficiency (GHD)
To compare near Final Height Standard Deviation Score (FH SDS) in the GH+ and GH- groups, Mid-parental Target Height (TH) (estimated from parental heights[1]), and TH-FH (in cm and SDS from FH reference data).
Key facts
- Sponsor
- University College London
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hormonal diseases [C19]
- Trial duration
- 17 May 2023 → 4 Dec 2024
- Decision date (initial)
- 2023-03-20
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2022-502008-62-00
- ISRCTN
- ISRCTN12552768
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Therapy
To compare near Final Height Standard Deviation Score (FH SDS) in the GH+ and GH- groups, Mid-parental Target Height (TH) (estimated from parental heights[1]), and TH-FH (in cm and SDS from FH reference data).
Secondary objectives 1
- To compare the lipid profiles (fasting lipids - serum triglyceride and serum total cholesterol) and bone health in the GH+ and GH- groups
Conditions and MedDRA coding
Growth Hormone Deficiency (GHD)
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- Children (8-15 years of age for girls, 9-17 years of age for boys) with reversed Isolated Growth Hormone Deficiency (I-GHD) in established puberty (Tanner stages B2/3 in girls & 6-12ml testes in boys) and normal brain Magnetic Resonance Imaging (MRI) scan result (incl. small anterior pituitary). Children will need to have completed a minimum 6-week period of discontinuation of GH medication prior to a GH re-test. Children demonstrating GHD reversal (defined as a peak stimulated GH equal to or greater than 6.7 μg/L using arginine or insulin tolerance test) will be eligible to participate in this trial. Written informed consent must be obtained.
Exclusion criteria 1
- Hypopituitarism with or without additional pituitary hormone supplementation, a known genetic cause for I-GHD, organic GHD, ectopic posterior pituitary, other indications for GH therapy, pregnancy or lactation, any malignancy, current participation in another CTIMP, receipt of prednisolone or dexamethasone during the (minimum) 6-week discontinuation period before GH re-test
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Final Height in Standard Deviation Score (FH SDS)
Secondary endpoints 1
- Growth Related: The proportion of children reaching normal adult height (- 2SD) The proportion reaching mid-parental Target Height (- 2SD) Difference in child's Target Height minus Final Height (in Standard Deviation Score and centimetres) Bone Related: Bone age delay at Final Height Bone age acceleration between enrolment and Final Height Bone health index at Final Height Biochemistry: Serum IGF-1 and lipid profiles (fasting lipids - serum triglyceride and total serum cholesterol) at Final Heig
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 6
Genotropin® MiniQuick 0,2 mg - Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
PRD498332 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- 1-22045
- MA holder
- PFIZER CORPORATION AUSTRIA GESELLSCHAFT M.B.H.
- MA country
- Austria
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NutropinAq 10 mg/2 ml (30 IU) solution for injection.
PRD3424385 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- EU/1/00/164/003
- MA holder
- IPSEN PHARMA
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Omnitrope 5 mg/1.5 ml solution for injection in cartridge
PRD6061048 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- EU/1/06/332/013
- MA holder
- SANDOZ GMBH
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Saizen 8 mg/ml Injektionslösung in einer Patrone
PRD360624 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION IN CARTRIDGE
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- 2-00389
- MA holder
- MERCK GESELLSCHAFT MBH
- MA country
- Austria
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
HUMATROPE 6 mg - Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
PRD328195 · Product
- Active substance
- Somatropin
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- 1-21767
- MA holder
- ELI LILLY GES. M.B.H
- MA country
- Austria
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Norditropin FlexPro 15 mg/1,5 ml Injektionslösung in einem Fertigpen
PRD322236 · Product
- Active substance
- Somatropin
- Substance synonyms
- RECOMBINANT HUMAN GROWTH HORMONE, SOMATOTROPHIN, CB-311, LY-137998
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- CUTANEOUS USE
- Max daily dose
- 1 mg/Kg milligram(s)/kilogram
- Max total dose
- 1 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01AC01 — SOMATROPIN
- Marketing authorisation
- 2-00369
- MA holder
- NOVO NORDISK PHARMA GMBH
- MA country
- Austria
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
University College London
- Sponsor organisation
- University College London
- Address
- 250 Euston Road
- City
- London
- Postcode
- NW1 2PG
- Country
- United Kingdom
Scientific contact point
- Organisation
- University College London
- Contact name
- Mehul Tulsidas Dattani
Public contact point
- Organisation
- University College London
- Contact name
- Mehul Tulsidas Dattani
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Johannes Kepler University ORG-100038960
|
Linz, Austria | On site monitoring, Code 13, Code 2, Code 5, Code 8 |
Locations
1 EU/EEA country · 5 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 40 | 5 |
| Rest of world | — | 0 | — |
Investigational sites
Johannes Kepler University
University Clinic for Pediatrics and Adolescent Medicine, Altenberger Strasse 69, 4040, Linz
Medical University Of Graz
Department of paediatrics, Neue Stiftingtalstrasse 6, 8010, Graz
Medizinische Universitaet Innsbruck
Department of paediatrics, Anichstraße 35, 6020, Innsbruck
Eb-Haus Austria Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH
Department of paediatrics, Müllner Hauptstraße 48, 5020, Salzburg
Klinikum Wels-Grieskirchen GmbH
Department of paediatrics, Grieskirchner Straße 42, 4600, Wels
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 |
|---|---|---|---|---|---|
| Austria | 2023-05-17 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Temporary halts 1 · Art. 38 CTR
Temporary halt TH-30644
- Halt date
- 2024-05-09
- Member states concerned
- Austria
- Publication date
- 2024-06-20
- Reason
- Feasibility (recruitment issues etc.)
- Explanation
- Clinical trial has enrolled but is currently on hold. Following the conclusion of the trial’s pilot phase the Funder have requested that the trial is closed as feasibility targets were not met (the trial was not put on hold for safety reasons). The GHD Reversal Trial team are preparing the costs of closing down the trial, as well as continuing the trial with an observational arm, for the Funder’s review.
- Benefit-risk balance changed
- No
- Treatment stopped
- No
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Final Analysis Summary Report SUM-112917
|
2025-12-23T15:21:20 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay Summary Report | 2025-12-23T15:22:58 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | Lay Summary Report 041125 | 1 |
| Summary of results (for publication) | Final Analysis Summary Report | 2.0 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2022-11-15 | Austria | Acceptable 2023-03-20
|
2023-03-20 |