Fibrous Dysplasia of BonePhase 2/3PROFIDYSNCT00445575
Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone
A Phase 2/3 study testing risedronate in people with Fibrous Dysplasia of Bone, run by Institut National de la Santé Et de la Recherche Médicale, France at 8 sites in 4 countries.
Trial summary
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Fibrous Dysplasia of Bone
- What is being tested?
- risedronate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 8 sitesin 4 countries
- Study dates
- 22 Jul 2007to 7 Dec 2017
- Phase
- Phase 2/3
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Study I: patients with FD, with bone pain intensity above 3 on visual analogical scale from 0 to 10
- Study II: patients with FD with at least one osteolytic lesion and no current bone pain
Who cannot join
- patients < 8 years old
- other diseases affecting bone metabolism
- patients with malignant diseases or other conditions likely to reduce their life expectancy to less than 3 years
- patients with history of significant upper gastrointestinal disorders
- renal failure (creatinine clearance < 25 ml/mn)
- severe liver disease
Show all 12Show fewer
- history of iritis or uveitis
- rickets or osteomalacia
- allergy to bisphosphonates
- pregnancy or lactation
- prior treatment with a bisphosphonate
- laboratory abnormalities that may be considered as clinically significant by trial physicians
Trial locations and contacts
8 sites in 4 countries.
8 sites in 4 countries
France3 sites
Hopital E Herriot
Lyon, 69437
Hopital Lariboisiere
Paris, 75475
Hopital Cochin
Paris, 75679
Germany3 sites
Hospital Benjamin Franklin
Berlin, 12200
Cologne Clinical Centre
Cologne, 50924
Heildeberg Clinical Centre
Heidelberg, 69120
About this study
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone.
Read the detailed description
In open pilot studies, it has been suggested that bisphosphonates may alleviate bone pain and help decrease the surface of osteolytic lesion in patients with fibrous dysplasia of bone (FD). So, in this randomized placebo controlled trial, we test the hypothesis that the bisphosphonate risedronate reduces bone pain in patients with FD (study I, one year duration) and decrease osteolytic lesions (study II, three years duration). Patients will take risedronate during 2 months courses, every 6 months or a matching placebo. Dosage will be : 30mg tablet/day for adults and 5mg tablet x 2,4 according to the age and weight of the child. All participants will receive calcium and vitamin D. All patients with renal phosphate wasting will receive an oral phosphate supplement.
- Conditions
- Fibrous Dysplasia of Bone
- Being tested
- risedronate, placebo
- Keywords
- fibrous dysplasia of bone, Mac Cune Albright syndrome, bisphosphonates, risedronate
- Medical subject headings
- Fibrous Dysplasia of Bone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
risedronateDrug
During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.
placeboDrug
placebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.
risedronateDrug
During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.
placeboDrug
placebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | treatment duration: 1 year Receives: Drug: risedronate |
| 2 | Placebo comparator | treatment duration: 1 year Receives: Drug: placebo |
| 3 | Experimental | duration treatment: 3 years Receives: Drug: risedronate |
| 4 | Placebo comparator | treatment duration: 3 years Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intensity of bone pain, assessed by visual analogical scale ranging from 0 to 10, on the most painful site.
Measured: one year
Surface of osteolytic lesions at three years. Radiological improvement.
Measured: Three years
Secondary outcomes (4)
Variation of biochemical markers of bone turnover at three years
Measured: three years
Number of painful sites
Measured: one year
Improvement in quality of life
Measured: one to three years
Variation in bone mineral density of the femoral neck at three years
Measured: three years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2007 (estimated)
- Study start22 Jul 2007
- Primary completionDec 2017
- Study completion7 Dec 2017
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 Institut National de la Santé Et de la Recherche Médicale, FranceThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Collaborators
- ZonMw: The Netherlands Organisation for Health Research and Development, Cliniques universitaires Saint-Luc- Université Catholique de Louvain, Charite University, Berlin, Germany
- Responsible party
- Sponsor
- Investigators
- ROLAND D CHAPURLAT, MD PhD, Principal investigator, Institut National de la Santé Et de la Recherche Médicale, FrancePHILIPPE ORCEL, MD PhD, Study director, HOPITAL LARIBOISIERESOCRATES D PAPAPOULOS, MD PhD, Study chair, Leiden University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00445575
- Sponsor's protocol ID
- RBM 03-54
- Other IDs
- AFSSAPS 060834
- Acronym
- PROFIDYS
- Status last verified
- Aug 2021
- First submitted
- 8 Mar 2007
- Last updated
- 30 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone
- ClinicalTrials.gov ID
- NCT00445575
- Acronym / sponsor ID
- PROFIDYS / RBM 03-54
- Conditions
- Fibrous Dysplasia of Bone
- Keywords
- fibrous dysplasia of bone, Mac Cune Albright syndrome, bisphosphonates, risedronate
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Collaborators
- ZonMw: The Netherlands Organisation for Health Research and Development, Cliniques universitaires Saint-Luc- Université Catholique de Louvain, Charite University, Berlin, Germany
- Study start
- 22 Jul 2007 (actual)
- Primary completion
- Dec 2017 (actual)
- Study completion
- 7 Dec 2017 (actual)
- First posted
- 9 Mar 2007 (estimated)
- 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.
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone.
Detailed description
In open pilot studies, it has been suggested that bisphosphonates may alleviate bone pain and help decrease the surface of osteolytic lesion in patients with fibrous dysplasia of bone (FD). So, in this randomized placebo controlled trial, we test the hypothesis that the bisphosphonate risedronate reduces bone pain in patients with FD (study I, one year duration) and decrease osteolytic lesions (study II, three years duration). Patients will take risedronate during 2 months courses, every 6 months or a matching placebo. Dosage will be : 30mg tablet/day for adults and 5mg tablet x 2,4 according to the age and weight of the child. All participants will receive calcium and vitamin D. All patients with renal phosphate wasting will receive an oral phosphate supplement.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: risedronatetreatment duration: 1 year |
| 2 | Placebo comparator | Drug: placebotreatment duration: 1 year |
| 3 | Experimental | Drug: risedronateduration treatment: 3 years |
| 4 | Placebo comparator | Drug: placebotreatment duration: 3 years |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Study I: patients with FD, with bone pain intensity above 3 on visual analogical scale from 0 to 10
- Study II: patients with FD with at least one osteolytic lesion and no current bone pain
Key exclusion criteria (12 listed)
- patients < 8 years old
- other diseases affecting bone metabolism
- patients with malignant diseases or other conditions likely to reduce their life expectancy to less than 3 years
- patients with history of significant upper gastrointestinal disorders
- renal failure (creatinine clearance < 25 ml/mn)
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 |
|---|---|---|
| risedronateDuring two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child. | Drug | 1 |
| placeboplacebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child. | Drug | 2 |
| risedronateDuring two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child. | Drug | 3 |
| placeboplacebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child. | Drug | 4 |
Primary outcome
Intensity of bone pain, assessed by visual analogical scale ranging from 0 to 10, on the most painful site.
Time frame: one year
Surface of osteolytic lesions at three years. Radiological improvement.
Time frame: Three years
4 secondary outcomes
Variation of biochemical markers of bone turnover at three years
Time frame: three years
Number of painful sites
Time frame: one year
Improvement in quality of life
Time frame: one to three years
Variation in bone mineral density of the femoral neck at three years
Time frame: three years
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 30 Aug 2021.
- Related InfoLink provided by the sponsor.
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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.
ROLAND D CHAPURLAT, MD PhD principal investigator
Institut National de la Santé Et de la Recherche Médicale, France
PHILIPPE ORCEL, MD PhD study director
HOPITAL LARIBOISIERE
SOCRATES D PAPAPOULOS, MD PhD study chair
Leiden University Medical Center
8 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing risedronate in people with Fibrous Dysplasia of Bone, run by Institut National de la Santé Et de la Recherche Médicale, France at 8 sites in 4 countries.
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 12 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 30 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in France, Germany, Belgium, Netherlands. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Intensity of bone pain, assessed by visual analogical scale ranging from 0 to 10, on the most painful site. (time frame: one year). The study lists 2 primary outcomes in all. 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 30 Aug 2021.