Idiopathic Infantile Hypercalcemia - Severe FormVaraD-CellNCT04987073
Vitamin D Analog in Vitamin D 24-hydroxylase Deficiency
A study testing skin biopsy in people with Idiopathic Infantile Hypercalcemia - Severe Form, run by University Hospital, Caen at 1 site in France.
Trial summary
Preclinical evaluation of a vitamin D analog for the treatment of vitamin D 24-hydroxylase using fibroblast from patients with CYP24A1 mutation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Infantile Hypercalcemia - Severe Form
- What is being tested?
- skin biopsy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- 17 Jul 2022to 1 Jul 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- biallelic mutation in CYP24A1
- consent
Who cannot join
- none
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Arnaud Molin
Caen, Normandy, 14033
Arnaud Molin, MD, PhD, 0231064502 ext. +33, molin-a@chu-caen.fr
Kara Ranguin, MSc, 0231272569 ext. +33, ranguin-k@chu-caen.fr
About this study
Official title: Preclinic Evaluation of a Vitamin D Analog in Vitamin D 24-hydroxylase Deficiency
Preclinical evaluation of a vitamin D analog for the treatment of vitamin D 24-hydroxylase using fibroblast from patients with CYP24A1 mutation.
- Being tested
- skin biopsy
- Keywords
- CYP24A1, vitamin D
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
skin biopsyProcedure or surgery
A skin biopsy to cultivate fibroblasts.
| Study group | Role | What they receive |
|---|---|---|
| patients with CYP24A1 mutation | Experimental | Not described Receives: Procedure: skin biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effects of the analog on vitamin D induced genes' expression
RNA quantification of genes induced by vitamin D in presence of analog in patients' fibroblast
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2021
- Study start17 Jul 2022
- Primary completion1 Jul 2026 (estimated)
- Study completion1 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Infantile Hypercalcemia - Severe FormStudies of the same condition, open and under way.
- More trials run by University Hospital, CaenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Caen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04987073
- Sponsor's protocol ID
- 2020-A03281-38
- Acronym
- VaraD-Cell
- Status last verified
- Jul 2025
- First submitted
- 22 Jul 2021
- Last updated
- 25 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Preclinic Evaluation of a Vitamin D Analog in Vitamin D 24-hydroxylase Deficiency
- ClinicalTrials.gov ID
- NCT04987073
- Acronym / sponsor ID
- VaraD-Cell / 2020-A03281-38
- Keywords
- CYP24A1, vitamin D
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University Hospital, CaenUniversity, hospital or other
- Study start
- 17 Jul 2022 (actual)
- Primary completion
- 1 Jul 2026 (estimated)
- Study completion
- 1 Jul 2027 (estimated)
- First posted
- 3 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Preclinical evaluation of a vitamin D analog for the treatment of vitamin D 24-hydroxylase using fibroblast from patients with CYP24A1 mutation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with CYP24A1 mutation | Experimental | Procedure: skin biopsy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- biallelic mutation in CYP24A1
- consent
Key exclusion criteria (1 listed)
- none
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| skin biopsyA skin biopsy to cultivate fibroblasts. | Procedure or surgery | patients with CYP24A1 mutation |
Primary outcome
Effects of the analog on vitamin D induced genes' expression
Time frame: 3 years
RNA quantification of genes induced by vitamin D in presence of analog in patients' fibroblast
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2025.
- Individual participant dataSharing planned: No
Related trials for Idiopathic Infantile Hypercalcemia - Severe Form
RecruitingPhase 2
Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
Idiopathic Infantile Hypercalcaemia - Severe Form, Genetic Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing skin biopsy in people with Idiopathic Infantile Hypercalcemia - Severe Form, run by University Hospital, Caen at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effects of the analog on vitamin D induced genes' expression (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2025.