Clinical trial • Phase III • Musculoskeletal | Respiratory
Colecalciferol for Cystic fibrosis | Bone metabolism disorder | Low bone mineral density | Skeletal muscle weakness
Phase III trial of Colecalciferol for Cystic fibrosis | Bone metabolism disorder | Low bone mineral density | Skeletal muscle weakness.
Overview
- Trial Therapeutic Area
- Musculoskeletal | Respiratory
- Trial Disease
- Cystic fibrosis | Bone metabolism disorder | Low bone mineral density | Skeletal muscle weakness
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 06-09-2024
- First CTIS Authorization Date
- 02-10-2024
Trial design
Randomised, comparator arm: cholecalciferol 1000 iu per day; intervention arm: cholecalciferol 6000 iu per day (daily dosing).-controlled Phase III trial across 1 site in Czechia.
- Randomised
- Yes
- Comparator
- Comparator arm: cholecalciferol 1000 IU per day; Intervention arm: cholecalciferol 6000 IU per day (daily dosing).
- Target Sample Size
- 64
- Trial Duration For Participant
- 730
Eligibility
Recruits 64 paediatric patients.
- Pregnancy Exclusion
- Pregnancy
- Vulnerable Population
- Participants are children (age range 6 to 16 years). Consent is required from the parent or legal representative ('Willingness of the parent or legal representative to sign the informed consent and allow the child participate in the study'). Age-specific information and consent/assent forms are provided (documents include ICFs for ages 12-14, 15-17, legal representative versions and ICFs for 18-year-olds), indicating assent/age-appropriate information for minors and parental/legal representative consent.
Inclusion criteria
- {"criterion_text":"- Diagnosis of cystic fibrosis\n- Age from 6 to 16 (included) years\n- Willingness of the parent or legal representative to sign the informed consent and allow the child participate in the study\n- Woman of childbearing potential need to comply with adequate contraception methods per CTFG guideline"}
Exclusion criteria
- {"criterion_text":"- Contraindication done by SmPC of Vigantol\n- Lung transplantation (done or planned)\n- Bisphosphonate treatment in the last 18 months\n- Disease other than CF with known impact on bone metabolism (based on consultancy with main investigator)\n- Hypersensitivity to the IMP or additive substance (as per SmPC)\n- Hypercalcaemia\n- Renal osteodystrophy with hyperphosphatemia\n- Pregnancy"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Serum concentrations of 25-OH vitamin D and parathormone at visits V3-V7","definition_or_measurement_approach":"Measured at specified visits V3-V7; no further measurement method details provided in the source."}
- {"endpoint_text":"- Lumbar Spine aBMD at visit V7","definition_or_measurement_approach":"Measured at visit V7; no further measurement method details provided in the source."}
- {"endpoint_text":"- Dynamic muscle force and power at visit V7","definition_or_measurement_approach":"Assessed at visit V7; no further measurement method details provided in the source."}
Recruitment
- Planned Sample Size
- 64
- Recruitment Window Months
- 49
- Consent Approach
- Informed consent must be signed by the parent or legal representative. Age-specific subject information and assent/consent forms are provided (documents include ICFs for ages 12-14, 15-17, legal representative versions, and documents for 18-year-olds). Available forms in the dossier indicate materials in Czech (file names include 'CZ').
Geography
- Total Number Of Sites
- 1
- Total Number Of Participants
- 64
Czechia
- Earliest CTIS Part Ii Submission Date
- 06-09-2024
- Latest Decision Or Authorization Date
- 02-10-2024
- Processing Time Days
- 26
- Number Of Sites
- 1
- Number Of Participants
- 64
Sites
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Pediatric Clinic
- Principal Investigator Name
- Ondřej Souček
- Principal Investigator Email
- Ondrej.Soucek@fnmotol.cz
- Contact Person Name
- Ondřej Souček
- Contact Person Email
- Ondrej.Soucek@fnmotol.cz
- Number Of Participants
- 64
Sponsor
Primary sponsor
- Full Name
- Fakultni Nemocnice V Motole
- Organisation Type
- Hospital/Clinic/Other health care facility
- Country Of Registered Address
- Czechia
Investigational products
- Investigational Product Name
- Vigantol 0,5 mg/ml perorální kapky, roztok
- Active Substance
- Colecalciferol
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- Marketing authorised in CZ (marketingAuthNumber: 86/1140/93-C)
- Starting Dose
- 6000 IU/day (intervention) and 1000 IU/day (comparator)
- Dose Levels
- 1000 IU/day | 6000 IU/day
- Frequency
- Once daily
- Maximum Dose
- 6000 IU/day
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