Overview
Sponsor-declared trial summary
Cystic fibrosis and problems with bone metabolism, bone density, skeletal muscle force
The goal of this study is to assess whether 6000 IU of cholecalciferol per day given over two years to children with cystic fibrosis is superior to 1000 IU of cholecalciferol in normalizing their bone metabolism parameters and in improving their bone density and skeletal muscle force.
Key facts
- Sponsor
- Fakultni Nemocnice V Motole
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Respiratory Tract Diseases [C08]
- Trial duration
- 2 Oct 2024 → ongoing
- Decision date (initial)
- 2024-10-02
- Transition trial
- Yes
- Low-intervention
- Yes
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-517886-18-00
- EudraCT number
- 2022-002330-13
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Dose response, Prophylaxis
The goal of this study is to assess whether 6000 IU of cholecalciferol per day given over two years to children with cystic fibrosis is superior to 1000 IU of cholecalciferol in normalizing their bone metabolism parameters and in improving their bone density and skeletal muscle force.
Conditions and MedDRA coding
Cystic fibrosis and problems with bone metabolism, bone density, skeletal muscle force
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Diagnosis of cystic fibrosis
- Age from 6 to 16 (included) years
- Willingness of the parent or legal representative to sign the informed consent and allow the child participate in the study
- Woman of childbearing potential need to comply with adequate contraception methods per CTFG guideline
Exclusion criteria 8
- Contraindication done by SmPC of Vigantol
- Lung transplantation (done or planned)
- Bisphosphonate treatment in the last 18 months
- Disease other than CF with known impact on bone metabolism (based on consultancy with main investigator)
- Hypersensitivity to the IMP or additive substance (as per SmPC)
- Hypercalcaemia
- Renal osteodystrophy with hyperphosphatemia
- Pregnancy
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Serum concentrations of 25-OH vitamin D and parathormone at visits V3-V7
- Lumbar Spine aBMD at visit V7
- Dynamic muscle force and power at visit V7
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Vigantol 0,5 mg/ml perorální kapky, roztok
PRD7579007 · Product
- Active substance
- Colecalciferol
- Pharmaceutical form
- ORAL DROPS, SOLUTION
- Route of administration
- ORAL
- Max daily dose
- 6000 IU international unit(s)
- Max total dose
- 6000 IOU international opacity unit(s)
- Max treatment duration
- 24 Month(s)
- Authorisation status
- Authorised
- ATC code
- A11CC05 — COLECALCIFEROL
- Marketing authorisation
- 86/1140/93-C
- MA holder
- P&G HEALTH GERMANY GMBH
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fakultni Nemocnice V Motole
- Sponsor organisation
- Fakultni Nemocnice V Motole
- Address
- V Uvalu 84/1, Motol Motol
- City
- Prague
- Postcode
- 150 00
- Country
- Czechia
Scientific contact point
- Organisation
- Fakultni Nemocnice V Motole
- Contact name
- Principal Investigator
Public contact point
- Organisation
- Fakultni Nemocnice V Motole
- Contact name
- The Clinical Trials Department
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ongoing, recruiting | 64 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| Czechia | 2024-10-02 | 2024-10-02 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 11 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | Protocol_COAM-CFD-01_ver2_13JUN2023 | 2 |
| Recruitment arrangements (for publication) | COAM-CFD-01 - Sablona 1 cj_aj_final | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac12-14let_v1 | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac12-14let_v1_redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac15-17let_v1 | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac15-17let_v1_redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac18letavice_v1 | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_Pac18letavice_v1_redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_ZakonnyZastupce_v1 | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_CZ_COAM-CFD-01_ICF_ZakonnyZastupce_v1_redacted | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | SPC_ vigantol | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-06 | Czechia | Acceptable with conditions 2024-10-02
|
2024-10-02 |