Hypophosphatemic RicketsHYPO-CLASTENCT04184661
Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts
An observational study of Hypophosphatemic Rickets, run by Hospices Civils de Lyon at 3 sites in France.
Trial summary
Fibroblast growth factor 23 (FGF23) is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of Parathyroid hormone (PTH) in most tissues. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Hypophosphatemic Rickets
- What is being tested?
- blood sample
- How many people
- 52 participants
- Where
- 3 sitesFrance
- Study dates
- 20 Jan 2021to 27 Oct 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients with hypophosphatemic rickets and controls patients without hypophosphatemic rickets
Who may be able to join
- children from 2 yars-old to 18 years old and adults
- patients with HR followed in the center of calcium and phosphorus metabolism rare diseases in Lyon-
- Patients and parent / holder of parental authority who have been informed of the study and do not object to participate
- children from 2 years-old to 18 years old and adults
- patients with normal renal function (Schwartz glomerular filtration rate (GFR) >90 ml/min/1.73m²)
- Patients and parent / holder of parental authority who have been informed of the study and do not object to participate
Who cannot join
- Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.
- Patients under tutorship or curatorship
- Pregnant and / or breastfeeding woman
- Patient deprived of liberty
- Controls patients:
- Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.
Show all 11Show fewer
- Patients under tutorship or curatorship
- Pregnant and / or breastfeeding woman
- Patient deprived of liberty
- Patient treated with immunosuppressive drugs
- Patient with inflammatory disease
Trial locations and contacts
3 sites in 1 country.
France3 sites
Hôpital Femme mère enfant
Bron, 69677
Hôpital Edouard Herriot
Lyon
Hôpital Bicêtre Paris Saclay
Paris
About this study
Official title: Effect of Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts From Patients With Hypophosphatemic Rickets (HR)
Fibroblast growth factor 23 (FGF23) is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of Parathyroid hormone (PTH) in most tissues.
The specific role of FGF23 on bone has yet to be demonstrated. In some diseases such as hypophosphatemic rickets (HR), the direct role of FGF23 on bone has not yet been studied to our knowledge, whereas these genetic hypophosphatemias are secondary to overexpression of FGF23, whether an activating mutation of FGF23 or inhibitory mutations of its inhibitors (Dentin matrix acidic phosphoprotein 1 (DMP1) and Phosphate-regulating neutral endopeptidase, X-linked (PHEX)). However, patients with X-linked hypophosphatemic rickets (XLH) have higher circulating FGF23 levels than healthy controls and these levels are higher in treated patients.
Read more
Management of XLH consists primarily of correcting the native vitamin D defect by prescribing active vitamin D analogs as well as phosphate supplementation to improve bone mineralization and decrease dental complications, growth, and bone deformities. Recently, a new therapeutic option has been developed for XLH, burosumab, a human monoclonal antibody that binds and inhibits FGF23 activity. The use of burosumab is currently authorized in France in some pediatric patients with severe forms of XLH.
Independently of the indirect bone effects of phosphate correction and vitamin D levels, the direct role of burosumab on bone cells has never been studied. The objective of this project is to study the osteoclastic biology of patients with HR compared to control patients, and to evaluate the direct impact of the treatments used in this pathology on human osteoclasts.
- Conditions
- Hypophosphatemic Rickets
- Being tested
- blood sample
- Medical subject headings
- Rickets, Hypophosphatemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood sampleOther
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.
| Study group | Role | What they receive |
|---|---|---|
| hypophosphatemic rickets patients | 30 hypophosphatemic rickets patients older than 2 years will be included in this study Receives: Other: blood sample | |
| controls patients | 10 controls patients from pediatric nephrology unit without hypophosphatemic rickets, older than 2 years will be included in this study Receives: Other: blood sample |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of osteoclastic cells obtained after at the end of differentiation
The analysis of osteoclastic differentiation will be obtained from the bone cells from patients with burosumab and/or 1-25 (OH) vitamin D
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2019
- Study start20 Jan 2021
- Primary completion27 Oct 2021
- Study completion27 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypophosphatemic RicketsStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04184661
- Sponsor's protocol ID
- 69HCL19_0725
- Other IDs
- 2019-A02914-53
- Acronym
- HYPO-CLASTE
- Status last verified
- Oct 2022
- First submitted
- 28 Nov 2019
- Last updated
- 6 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts From Patients With Hypophosphatemic Rickets (HR)
- ClinicalTrials.gov ID
- NCT04184661
- Acronym / sponsor ID
- HYPO-CLASTE / 69HCL19_0725
- Conditions
- Hypophosphatemic Rickets
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 20 Jan 2021 (actual)
- Primary completion
- 27 Oct 2021 (actual)
- Study completion
- 27 Oct 2021 (actual)
- First posted
- 3 Dec 2019 (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.
Fibroblast growth factor 23 (FGF23) is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of Parathyroid hormone (PTH) in most tissues.
The specific role of FGF23 on bone has yet to be demonstrated. In some diseases such as hypophosphatemic rickets (HR), the direct role of FGF23 on bone has not yet been studied to our knowledge, whereas these genetic hypophosphatemias are secondary to overexpression of FGF23, whether an activating mutation of FGF23 or inhibitory mutations of its inhibitors (Dentin matrix acidic phosphoprotein 1 (DMP1) and Phosphate-regulating neutral endopeptidase, X-linked (PHEX)). However, patients with X-linked hypophosphatemic rickets (XLH) have higher circulating FGF23 levels than healthy controls and these levels are higher in treated patients.
Management of XLH consists primarily of correcting the native vitamin D defect by prescribing active vitamin D analogs as well as phosphate supplementation to improve bone mineralization and decrease dental complications, growth, and bone deformities. Recently, a new therapeutic option has been developed for XLH, burosumab, a human monoclonal antibody that binds and inhibits FGF23 activity. The use of burosumab is currently authorized in France in some pediatric patients with severe forms of XLH.
Independently of the indirect bone effects of phosphate correction and vitamin D levels, the direct role of burosumab on bone cells has never been studied. The objective of this project is to study the osteoclastic biology of patients with HR compared to control patients, and to evaluate the direct impact of the treatments used in this pathology on human osteoclasts.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| hypophosphatemic rickets patients | – | Other: blood sample30 hypophosphatemic rickets patients older than 2 years will be included in this study |
| controls patients | – | Other: blood sample10 controls patients from pediatric nephrology unit without hypophosphatemic rickets, older than 2 years will be included in this study |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with hypophosphatemic rickets and controls patients without hypophosphatemic rickets
Key inclusion criteria (6 listed)
- children from 2 yars-old to 18 years old and adults
- patients with HR followed in the center of calcium and phosphorus metabolism rare diseases in Lyon-
- Patients and parent / holder of parental authority who have been informed of the study and do not object to participate
- children from 2 years-old to 18 years old and adults
- patients with normal renal function (Schwartz glomerular filtration rate (GFR) >90 ml/min/1.73m²)
Key exclusion criteria (11 listed)
- Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.
- Patients under tutorship or curatorship
- Pregnant and / or breastfeeding woman
- Patient deprived of liberty
- Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.
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 |
|---|---|---|
| blood sample25 mL blood sample will be collected on citrate tubes for osteoclastic analysis. | Other | controls patients, hypophosphatemic rickets patients |
Primary outcome
number of osteoclastic cells obtained after at the end of differentiation
Time frame: 1 day
The analysis of osteoclastic differentiation will be obtained from the bone cells from patients with burosumab and/or 1-25 (OH) vitamin D
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2022.
Related trials for Hypophosphatemic Rickets
RecruitingPhase 1/2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
RecruitingObservational study
Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients
FGF23-related Hypophosphataemic Rickets and Osteomalacia
Active, not recruitingObservational study
X-linked Hypophosphatemia Disease Monitoring Program
X-linked Hypophosphatemia, Hypophosphatemic Rickets
Recruiting
Phosphorus-31 Spectroscopy in Phosphate Diabetes
Phosphate Diabetes, X-linked Hypophosphatemia
RecruitingObservational study
Registry for Patients With X-Linked Hypophosphatemia
X-Linked Hypophosphatemia
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypophosphatemic Rickets, run by Hospices Civils de Lyon at 3 sites 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 6 inclusion and 11 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: finished, as of the sponsor's last update on 6 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
number of osteoclastic cells obtained after at the end of differentiation (time frame: 1 day). 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 6 Oct 2022.