Osteogenesis ImperfectaPhase 2OI-AKNCT01799798
Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab
A Phase 2 study testing Denosumab in people with Osteogenesis Imperfecta, run by University of Cologne at 1 site in Germany.
Trial summary
Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Read more about this study
- Who is it for?
- Ages 5 to 11 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Denosumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteGermany
- Study dates
- Feb 2013to Jan 2015
- Phase
- Phase 2
- 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 5 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female subjects between 5 years and 10 years of age with molecular proven Osteogenesis imperfecta (COL1A1/A2 mutation)
- Subjects must have been treated for a minimum of 2 years with bisphosphonates prior to study entry
Who cannot join
- Hypocalcemia (<1.03 mmol/l ionized Calcium)
- Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
- Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting denosumab s.c.
Trial locations and contacts
1 site in 1 country.
Germany1 site
University Cologne, Childrens Hospital, Cologne, Germany
Cologne, North Rhine-Westphalia, 50924
About this study
Official title: TRANSLATIONAL THERAPY IN PATIENTS WITH OSTEOGENESIS IMPERFECTA - A PILOT TRIAL ON TREATMENT WITH THE RANKL-ANTIBODY DENOSUMAB
Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Efficacy will be assessed by DXA measurements at the lumbar spine of the areal bone mineral density (BMD) which is the most frequently used parameter in trials investigating osteoporosis.
The hypothesis of the study is:
Osteoclastic activity which is increased in OI could be reduced by inhibition of osteoclast maturation. Denosumab inhibits maturation of the osteoclasts by inhibiting RANKL. BMD could be increased during a 36 week treatment course with denosumab measured after 48 weeks.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- Denosumab
- Keywords
- Osteogenesis imperfecta, COL1A1/A2, Denosumab, Bisphosphonates, Children, Areal bone mineral density
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DenosumabDrug
Denosumab will be given subcutaneously in a dosage of 1mg/kg body weight every 12 weeks. 4 interventions are planned until trial week 36. There is no control group planned.
| Study group | Role | What they receive |
|---|---|---|
| Denosumab subcutaneously | Experimental | Not described Receives: Drug: Denosumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48.
Measured: 48 weeks
Secondary outcomes (4)
Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD).
Measured: 14 days (DPD)
Parathormone in study week 12, 24, 36 and 48 compared to baseline.
Descriptive statistical analysis
Measured: 12 weeks
N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.
descriptive statistical analysis
Measured: 12 weeks
Osteocalcin in study week 12, 24, 36 and 48.
descriptive statistical analysis
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2013 (estimated)
- Study startFeb 2013
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Cologne
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. med. Joerg Oliver Semler, University of Cologne
- Investigators
- Joerg Oliver Semler, MD, Principal investigator, University Cologne, Childrens Hospital, Cologne, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01799798
- Sponsor's protocol ID
- Uni-Koeln-1574
- Acronym
- OI-AK
- Status last verified
- Jan 2015
- First submitted
- 14 Feb 2013
- Last updated
- 27 Jan 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Semler O, Netzer C, Hoyer-Kuhn H, Becker J, Eysel P, Schoenau E. First use of the RANKL antibody denosumab in osteogenesis imperfecta type VI. J Musculoskelet Neuronal Interact. 2012 Sep;12(3):183-8.
PubMed 22947550
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- TRANSLATIONAL THERAPY IN PATIENTS WITH OSTEOGENESIS IMPERFECTA - A PILOT TRIAL ON TREATMENT WITH THE RANKL-ANTIBODY DENOSUMAB
- ClinicalTrials.gov ID
- NCT01799798
- Acronym / sponsor ID
- OI-AK / Uni-Koeln-1574
- Conditions
- Osteogenesis Imperfecta
- Keywords
- Osteogenesis imperfecta, COL1A1/A2, Denosumab, Bisphosphonates, Children, Areal bone mineral density
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of CologneUniversity, hospital or other
- Study start
- Feb 2013
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 27 Feb 2013 (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.
Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Efficacy will be assessed by DXA measurements at the lumbar spine of the areal bone mineral density (BMD) which is the most frequently used parameter in trials investigating osteoporosis.
The hypothesis of the study is:
Osteoclastic activity which is increased in OI could be reduced by inhibition of osteoclast maturation. Denosumab inhibits maturation of the osteoclasts by inhibiting RANKL. BMD could be increased during a 36 week treatment course with denosumab measured after 48 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Denosumab subcutaneously | Experimental | Drug: Denosumab |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Male or female subjects between 5 years and 10 years of age with molecular proven Osteogenesis imperfecta (COL1A1/A2 mutation)
- Subjects must have been treated for a minimum of 2 years with bisphosphonates prior to study entry
Key exclusion criteria (3 listed)
- Hypocalcemia (<1.03 mmol/l ionized Calcium)
- Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
- Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting denosumab s.c.
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 |
|---|---|---|
| DenosumabDenosumab will be given subcutaneously in a dosage of 1mg/kg body weight every 12 weeks. 4 interventions are planned until trial week 36. There is no control group planned. | Drug | Denosumab subcutaneously |
Primary outcome
Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48.
Time frame: 48 weeks
4 secondary outcomes
Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD).
Time frame: 14 days (DPD)
Parathormone in study week 12, 24, 36 and 48 compared to baseline.
Time frame: 12 weeks
N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.
Time frame: 12 weeks
Osteocalcin in study week 12, 24, 36 and 48.
Time frame: 12 weeks
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 27 Jan 2015.
- Background: PubMed 22947550Semler O, Netzer C, Hoyer-Kuhn H, Becker J, Eysel P, Schoenau E. First use of the RANKL antibody denosumab in osteogenesis imperfecta type VI. J Musculoskelet Neuronal Interact. 2012 Sep;12(3):183-8.
Related trials for Osteogenesis Imperfecta
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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.
Joerg Oliver Semler, MD principal investigator
University Cologne, Childrens Hospital, Cologne, Germany
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Denosumab in people with Osteogenesis Imperfecta, run by University of Cologne at 1 site in Germany.
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 3 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 27 Jan 2015. 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 Germany. 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.
What does this 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.
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?
Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48. (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01799798, which also lists 1 linked publication.
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 27 Jan 2015.