Fibrous Dysplasia of BoneEfficienceNCT05422833
Effectiveness of Medical Management of Fibrous Dysplasia of Bone.
An observational study of Fibrous Dysplasia of Bone and McCune Albright Syndrome, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
The objective of our study was to assess the effectiveness of our reference center since its constitution. In a retrospective cohort study, we compared the activity of our center, including the time elapsed between diagnosis and access to the center and the diagnostic delay of patients with fibrous dysplasia between… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrous Dysplasia of Bone, McCune Albright Syndrome
- What is being tested?
- Evaluation and analysis of clinical data
- How many people
- 528 participants
- Where
- 1 siteFrance
- Study dates
- Jan 1996to Dec 2019
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children and adults with FD/MAS who visited at least once in our center
Who may be able to join
- We have included children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). The diagnosis was established by an expert of the center, based on clinical, biological, imaging and/or histological arguments
Who cannot join
- The absence of detectable bone lesion
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effectiveness of Medical Management in the Field of Rare Diseases. The Example of Fibrous Dysplasia of Bone.
The objective of our study was to assess the effectiveness of our reference center since its constitution.
In a retrospective cohort study, we compared the activity of our center, including the time elapsed between diagnosis and access to the center and the diagnostic delay of patients with fibrous dysplasia between two periods, 1996-2006 (before certification of our center) and 2007-2019 (after certification of our center).
- Being tested
- Evaluation and analysis of clinical data
- Keywords
- Fibrous Dysplasia of Bone, McCune Albright Syndrome, rare, bone disease, healthcare organization
- Medical subject headings
- Fibrous Dysplasia of Bone, Fibrous Dysplasia, Polyostotic, Bone Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Evaluation and analysis of clinical dataOther
Evaluation and analysis of clinical data (baseline demographic features (sex, age at diagnosis, age at first symptoms, age at first visit), initial presenting symptoms, affected bone sites (monostotic FD or polyostotic FD), renal phosphate wasting, MAS, Mazabraud syndrome, endocrine disease, fractures, bone specific treatment especially bisphosphonate and surgery, pain and disease's severity.
| Study group | Role | What they receive |
|---|---|---|
| Before | Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients).
and with the first visit in the center included between 1996 and 2006 - before certification of our center. Receives: Other: Evaluation and analysis of clinical data | |
| After | Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients).
and with the first visit in the center included between 2007 and 2019 - after certification of our center. Receives: Other: Evaluation and analysis of clinical data |
What is being measured
The primary outcome is the main question the study is designed to answer.
to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD
The main objective was to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD, over 2 periods of time: 1996-2006 (before certification) and 2007-2019 (after certification).
Measured: 1996-2019
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2022
- Study startJan 1996
- Primary completionDec 2019
- Study completionDec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrous Dysplasia of BoneStudies 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 528 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
- NCT05422833
- Sponsor's protocol ID
- 370
- Acronym
- Efficience
- Status last verified
- Jun 2022
- First submitted
- 10 May 2022
- Last updated
- 21 Jun 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 21 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Medical Management in the Field of Rare Diseases. The Example of Fibrous Dysplasia of Bone.
- ClinicalTrials.gov ID
- NCT05422833
- Acronym / sponsor ID
- Efficience / 370
- Keywords
- Fibrous Dysplasia of Bone, McCune Albright Syndrome, rare, bone disease, healthcare organization
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 528 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Jan 1996 (actual)
- Primary completion
- Dec 2019 (actual)
- Study completion
- Dec 2019 (actual)
- First posted
- 21 Jun 2022 (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.
The objective of our study was to assess the effectiveness of our reference center since its constitution.
In a retrospective cohort study, we compared the activity of our center, including the time elapsed between diagnosis and access to the center and the diagnostic delay of patients with fibrous dysplasia between two periods, 1996-2006 (before certification of our center) and 2007-2019 (after certification of our center).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Before | – | Other: Evaluation and analysis of clinical dataChildren and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 1996 and 2006 - before certification of our center. |
| After | – | Other: Evaluation and analysis of clinical dataChildren and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 2007 and 2019 - after certification of our center. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children and adults with FD/MAS who visited at least once in our center
Key inclusion criteria (1 listed)
- We have included children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). The diagnosis was established by an expert of the center, based on clinical, biological, imaging and/or histological arguments
Key exclusion criteria (1 listed)
- The absence of detectable bone lesion
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 |
|---|---|---|
| Evaluation and analysis of clinical dataEvaluation and analysis of clinical data (baseline demographic features (sex, age at diagnosis, age at first symptoms, age at first visit), initial presenting symptoms, affected bone sites (monostotic FD or polyostotic FD), renal phosphate wasting, MAS, Mazabraud syndrome, endocrine disease, fractures, bone specific treatment especially bisphosphonate and surgery, pain and disease's severity. | Other | After, Before |
Primary outcome
to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD
Time frame: 1996-2019
The main objective was to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD, over 2 periods of time: 1996-2006 (before certification) and 2007-2019 (after certification).
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 21 Jun 2022.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fibrous Dysplasia of Bone and McCune Albright Syndrome, run by Hospices Civils de Lyon 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 1 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: finished, as of the sponsor's last update on 21 Jun 2022. 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?
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?
to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD (time frame: 1996-2019). 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 21 Jun 2022.