Osteogenesis ImperfectaNCT00187018
Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
A study testing Bone marrow transplant in people with Osteogenesis Imperfecta, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure. OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and causes their bones to bend and break easily. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Bone marrow transplant
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2004to Aug 2007
- Phase
- Interventional
- 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
Who may be able to join
- Must have been previously enrolled on TOIT protocol at St. Jude Children's Research Hospital
- Must have original bone marrow donor available and willing to participate as a donor
- Normal liver function
- Hemoglobin >10gm/dl
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure. OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and causes their bones to bend and break easily. Some forms of osteogenesis imperfecta may cause severe disability and even death. In previous research studies performed at St. Jude, it was found that children treated with bone marrow transplant (infusion of healthy immature blood-forming cells) began to grow faster, had more minerals (material that helps make the bones strong) in their bones, and broke their bones less often than before the bone marrow transplant. Several months after the bone marrow transplant however, body growth once again began to slow down. In this research study, children with osteogenesis imperfecta will receive another infusion of bone marrow cells but without any chemotherapy. The marrow cells will come from the same bone marrow donor as their previous bone marrow transplant. It is hoped that by removing the CD3+ cells (a type of white blood cells that attack other cells that are not like themselves) from the donated bone marrow, the subject's body will be infused quite safely and that body growth and bone strength will increase. The CD3+ cells will be removed from the donor bone marrow by use of a machine called the CliniMACS System. This machine has not been approved for use in the United States by the Food and Drug Administration (FDA). The use of this device is considered experimental.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- Bone marrow transplant
- Keywords
- Collagen Disease
- Medical subject headings
- Osteogenesis Imperfecta, Collagen Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone marrow transplantBiological
What is being measured
The primary outcome is the main question the study is designed to answer.
To find out the effects (good and bad) of bone marrow cell infusions using donor bone marrow that has had CD3+ cells removed
To find out if there is any effect on the growth rate of children with osteogenesis imperfecta who receive donor bone marrow which has had CD3+ cells removed
To find out if there is any effect on the total bone mineral content of children with OI
who receive donor bone marrow which has had CD3+ cells removed
Secondary outcomes (1)
To find out the effect of the CD3 washed-out marrow cell therapy on the growth rate of the children
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startMar 2004
- Primary completionAug 2007
- Study completionAug 2007
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.
- More trials run by St. Jude Children's Research 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gregory Hale, M.D., Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00187018
- Sponsor's protocol ID
- STOD2
- Status last verified
- May 2007
- First submitted
- 12 Sep 2005
- Last updated
- 4 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Otsuru S, Gordon PL, Shimono K, Jethva R, Marino R, Phillips CL, Hofmann TJ, Veronesi E, Dominici M, Iwamoto M, Horwitz EM. Transplanted bone marrow mononuclear cells and MSCs impart clinical benefit to children with osteogenesis imperfecta through different mechanisms. Blood. 2012 Aug 30;120(9):1933-41. doi: 10.1182/blood-2011-12-400085. Epub 2012 Jul 24.
PubMed 22829629
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
- ClinicalTrials.gov ID
- NCT00187018
- Acronym / sponsor ID
- STOD2
- Conditions
- Osteogenesis Imperfecta
- Keywords
- Collagen Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Mar 2004
- Primary completion
- Aug 2007 (actual)
- Study completion
- Aug 2007 (actual)
- First posted
- 16 Sep 2005 (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.
Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure. OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and causes their bones to bend and break easily. Some forms of osteogenesis imperfecta may cause severe disability and even death. In previous research studies performed at St. Jude, it was found that children treated with bone marrow transplant (infusion of healthy immature blood-forming cells) began to grow faster, had more minerals (material that helps make the bones strong) in their bones, and broke their bones less often than before the bone marrow transplant. Several months after the bone marrow transplant however, body growth once again began to slow down. In this research study, children with osteogenesis imperfecta will receive another infusion of bone marrow cells but without any chemotherapy. The marrow cells will come from the same bone marrow donor as their previous bone marrow transplant. It is hoped that by removing the CD3+ cells (a type of white blood cells that attack other cells that are not like themselves) from the donated bone marrow, the subject's body will be infused quite safely and that body growth and bone strength will increase. The CD3+ cells will be removed from the donor bone marrow by use of a machine called the CliniMACS System. This machine has not been approved for use in the United States by the Food and Drug Administration (FDA). The use of this device is considered experimental.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Must have been previously enrolled on TOIT protocol at St. Jude Children's Research Hospital
- Must have original bone marrow donor available and willing to participate as a donor
- Normal liver function
- Hemoglobin >10gm/dl
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Bone marrow transplant | Biological | – |
Primary outcome
To find out the effects (good and bad) of bone marrow cell infusions using donor bone marrow that has had CD3+ cells removed
To find out if there is any effect on the growth rate of children with osteogenesis imperfecta who receive donor bone marrow which has had CD3+ cells removed
To find out if there is any effect on the total bone mineral content of children with OI
who receive donor bone marrow which has had CD3+ cells removed
1 secondary outcome
To find out the effect of the CD3 washed-out marrow cell therapy on the growth rate of the children
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 4 Mar 2015.
- Derived from this study: PubMed 22829629Otsuru S, Gordon PL, Shimono K, Jethva R, Marino R, Phillips CL, Hofmann TJ, Veronesi E, Dominici M, Iwamoto M, Horwitz EM. Transplanted bone marrow mononuclear cells and MSCs impart clinical benefit to children with osteogenesis imperfecta through different mechanisms. Blood. 2012 Aug 30;120(9):1933-41. doi: 10.1182/blood-2011-12-400085. Epub 2012 Jul 24.
- St. Jude Children's Research HospitalLink provided by the sponsor.
Related trials for Osteogenesis Imperfecta
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
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Setrusumab vs Placebo for Osteogenesis Imperfecta
Osteogenesis Imperfecta
Active, not recruitingPhase 3
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Osteogenesis Imperfecta
RecruitingPhase 2
Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
Osteogenesis Imperfecta (OI)
Active, not recruitingPhase 3
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
Osteogenesis Imperfecta
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.
Gregory Hale, M.D. principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Bone marrow transplant in people with Osteogenesis Imperfecta, run by St. Jude Children's Research Hospital at 1 site in United States.
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 4 inclusion and 0 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 4 Mar 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 United States. 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 find out the effects (good and bad) of bone marrow cell infusions using donor bone marrow that has had CD3+ cells removed. The study lists 4 primary outcomes in all. 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 NCT00187018, 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 4 Mar 2015.