SAPHO SyndromePhase 1NCT02544659
Efficacy of Bisphosphonates in Patients With Synovitis, Acne, Pustulosis, Hyperostosis, and Osteitis (SAPHO) Syndrome
A Phase 1 study testing pamidronate disodium in people with SAPHO Syndrome, run by Peking Union Medical College Hospital.
Trial summary
This study is designed to evaluate long term efficacy of intravenous bisphosphonates for bone marrow edema in patients with SAPHO syndrome. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- SAPHO Syndrome
- What is being tested?
- pamidronate disodium
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Oct 2015to Oct 2016
- Phase
- Phase 1
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female patients between the ages of 18 and 70 years
- Only 1 of the 4 inclusion criteria is sufficient to arrive at a diagnosis of SAPHO syndrome：
- Osteo-articular manifestations of acne conglobata, acne fulminans, or hidradenitis suppurativa
- Osteo-articular manifestations of PPP
- Hyperostosis (of the anterior chest wall, limbs or spine) with or without dermatosis
- CRMO involving the axial or peripheral skeleton with or without dermatosis Palmoplantar pustulosis(PPP); chronic recurrent multifocal osteomyelitis(CRMO)
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- MRI shows bone marrow edema in affected site in patients
- Blood serum of patients show the normal white blood cell count, liver and renal function
- Patients who like to be followed up for 1 years
- Other protocol defined inclusion criteria may apply
Who cannot join
- Women in pregnancy or lactation.
- Septic osteomyelitis
- Infectious chest wall arthritis
- Infections PPP
- Palmo-plantar keratodermia
- DISH except for fortuitous association
- Osteoarticular manifestations of retinoid therapy
- Other protocol defined exclusion criteria may apply
About this study
Official title: Unrandomized, Open Label Single-center Study to Assess the Long Term Efficacy of Intravenous Bisphosphonates for Bone Marrow Edema in Patients With SAPHO Syndrome
This study is designed to evaluate long term efficacy of intravenous bisphosphonates for bone marrow edema in patients with SAPHO syndrome.
Read the detailed description
The study will evaluate the long term efficacy at 3 days, 3 and 6 months after pamidronate disodium administered. Patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 months (up to month 6).
- Conditions
- SAPHO Syndrome
- Being tested
- pamidronate disodium
- Keywords
- Treatment, Bisphosphonates, Response rate
- Medical subject headings
- Acquired Hyperostosis Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pamidronate disodiumDrug
the patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 month (up to month 6)
| Study group | Role | What they receive |
|---|---|---|
| pamidronate disodium | Experimental | the patients will be administered intravenous pamidronate disodium Receives: Drug: pamidronate disodium |
What is being measured
The primary outcome is the main question the study is designed to answer.
response to the treatment by the change of MRI result at screening, baseline, month 3 and month 6
Measured: 3 and 6 month (up to 6 month)
Secondary outcomes (10)
physician's global assessment of disease activity at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of Visual Analogue ale/Score (VAS) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis disease activity index (BASDAI) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis functional index (BASFI) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis metroloty index (BASMI) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of ankylosing spondylitis disease activity score (ASDAS) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of erythrocyte sedimentation rate (ESR) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of high sensitivity-C reaction protein (hs-CRP) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of Osteocalcin at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of beta-Crosslaps (beta-CTx) at screening, baseline, month 3 and month 6
Measured: 3 days, 3 and 6 month (up to 6 month)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2015 (estimated)
- Study startOct 2015
- Primary completionOct 2016
- Study completionOct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SAPHO SyndromeStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chen Li, Master, Principal investigator, Department of Traditional Chinese Medicine, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02544659
- Sponsor's protocol ID
- PUMCH-SAPHO-01
- Status last verified
- Sep 2015
- First submitted
- 28 Aug 2015
- Last updated
- 13 Oct 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Unrandomized, Open Label Single-center Study to Assess the Long Term Efficacy of Intravenous Bisphosphonates for Bone Marrow Edema in Patients With SAPHO Syndrome
- ClinicalTrials.gov ID
- NCT02544659
- Acronym / sponsor ID
- PUMCH-SAPHO-01
- Conditions
- SAPHO Syndrome
- Keywords
- Treatment, Bisphosphonates, Response rate
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Oct 2015
- Primary completion
- Oct 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 9 Sep 2015 (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 study is designed to evaluate long term efficacy of intravenous bisphosphonates for bone marrow edema in patients with SAPHO syndrome.
Detailed description
The study will evaluate the long term efficacy at 3 days, 3 and 6 months after pamidronate disodium administered. Patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 months (up to month 6).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| pamidronate disodium | Experimental | Drug: pamidronate disodiumthe patients will be administered intravenous pamidronate disodium |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male and female patients between the ages of 18 and 70 years
- Only 1 of the 4 inclusion criteria is sufficient to arrive at a diagnosis of SAPHO syndrome：
- MRI shows bone marrow edema in affected site in patients
- Blood serum of patients show the normal white blood cell count, liver and renal function
- Patients who like to be followed up for 1 years
Key exclusion criteria (8 listed)
- Women in pregnancy or lactation.
- Septic osteomyelitis
- Infectious chest wall arthritis
- Infections PPP
- Palmo-plantar keratodermia
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 |
|---|---|---|
| pamidronate disodiumthe patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 month (up to month 6) | Drug | pamidronate disodium |
Primary outcome
response to the treatment by the change of MRI result at screening, baseline, month 3 and month 6
Time frame: 3 and 6 month (up to 6 month)
10 secondary outcomes
physician's global assessment of disease activity at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of Visual Analogue ale/Score (VAS) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis disease activity index (BASDAI) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis functional index (BASFI) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of bath ankylosing spondylitis metroloty index (BASMI) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of ankylosing spondylitis disease activity score (ASDAS) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of erythrocyte sedimentation rate (ESR) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of high sensitivity-C reaction protein (hs-CRP) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of Osteocalcin at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
response to the treatment by the change of beta-Crosslaps (beta-CTx) at screening, baseline, month 3 and month 6
Time frame: 3 days, 3 and 6 month (up to 6 month)
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 13 Oct 2016.
Related trials for SAPHO Syndrome
RecruitingPhase 2/3
Study of the Efficacy and Safety of Etanercept Treatment in Patients With SAPHO Syndrome
SAPHO Syndrome
RecruitingObservational study
Study of the Pathophysiological Mechanisms Involved in the SAPHO Syndrome: Genetic Component and Immune Response
SAPHO Syndrome
RecruitingObservational study
Typological Study of Sleep Pathologies During Psoriatic Rheumatism and SAPHO Syndrome: Prospective Study Within the Paris Saint-Joseph Hospital Group"
Psoriasis Arthritis, SAPHO Syndrome
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.
Chen Li, Master principal investigator
Department of Traditional Chinese Medicine, Peking Union Medical College Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing pamidronate disodium in people with SAPHO Syndrome, run by Peking Union Medical College Hospital.
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 10 inclusion and 8 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 13 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
response to the treatment by the change of MRI result at screening, baseline, month 3 and month 6 (time frame: 3 and 6 month (up to 6 month)). 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 13 Oct 2016.