Avascular Necrosis of BonePhase 1/2NCT02448121
Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis in Sickle Cell Disease
A Phase 1/2 study testing Autologous bone marrow stem cell graft in people with Avascular Necrosis of Bone and Sickle Cell Disease, run by Federal University of Bahia.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Avascular Necrosis of Bone, Sickle Cell Disease
- What is being tested?
- Autologous bone marrow stem cell graft
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Aug 2006to Aug 2018
- Phase
- Phase 1/2
- 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Stable sickle cell disease patients
- FICAT classification of osteonecrosis, inclusive of Stage II.
- Diagnosis will be based on X-Ray and magnetic resonance imaging (MRI).
- No infection in affected bones at the time of surgery.
- Patient competent to give informed consent.
- Scoring at least 20 points on the pain and daily life activities questionnaire.
Who cannot join
- FICAT stage III or more
- Patients with a history of corticosteroids or on active therapy
- Bone infection at the limb affected by necrosis
- Recurrent painful crises,
- Immunosuppressive drug therapy,
- Pregnancy,
- Presence of neoplastic disease or any other clinical concurrent condition other than sickle cell disease that predisposed them to the development of osteonecrosis of the femoral head
About this study
Official title: Treatment for Hip, Knee, Ankle and Shoulder Osteonecrosis With Autologous Mononuclear Cells Transplantation in People With Sickle Cell Disease
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis
Read the detailed description
The aim of this study is to evaluate autologous bone marrow stem cells transplantation as a safe and potentially beneficial treatment for sickle cell disease patients with osteonecrosis.
- Conditions
- Avascular Necrosis of Bone, Sickle Cell Disease
- Being tested
- Autologous bone marrow stem cell graft
- Keywords
- Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell, Humeral Osteonecrosis, Knee Osteonecrosis, Ankle Osteonecrosis
- Medical subject headings
- Osteonecrosis, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous bone marrow stem cell graftProcedure or surgery
All patients will undergo autologous bone marrow stem cell implantation into the necrotic lesion area using a minimally invasive technique.
| Study group | Role | What they receive |
|---|---|---|
| Autologous bone marrow stem cell graft | Experimental | Autologous bone marrow stem cell implantation Receives: Procedure: Autologous bone marrow stem cell graft |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Functional / Clinical Score of 10 points or more
Standardized and objective scoring system to rate patient's functional abilities
Measured: 60 months
Secondary outcomes (4)
Numeric pain intensity scale (0-10)
VAS scoring system
Measured: 60 months
Disease progression defined as progression to a fractural stage of osteonecrosis
Radiographic signals
Measured: 60 months
Reoperation rate
Need for further surgery or hip replacement
Measured: 60 months
Radiological progression
Radiological progression is defined according to the development of the Ficat- and Steinberg-staging system of osteonecrosis. It is based on radiological findings in X-Ray and MRI scans, respectively, and it is useful in predicting outcomes.
Measured: 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2015 (estimated)
- Study startAug 2006
- Primary completionJul 2018 (estimated)
- Study completionAug 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Avascular Necrosis of BoneStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federal University of Bahia
- Sponsor type
- University, hospital or other
- Collaborators
- Hospital Universitário Professor Edgard Santos, Oswaldo Cruz Foundation
- Responsible party
- Principal investigator, Vitor Fortuna, Federal University of Bahia
- Investigators
- GIldasio Daltro, MD, Study director, Federal University of BahiaVitor Fortuna, PhD, Principal investigator, Federal University of BahiaRoberto Meyer, MD, Study chair, Federal University of BahiaRadovan Borojevic, PhD, Study chair, Federal University of Bahia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02448121
- Sponsor's protocol ID
- CAAE 25000039812/2005-99
- Other IDs
- 11738
- Status last verified
- Nov 2017
- First submitted
- 15 May 2015
- Last updated
- 20 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Hernigou P, Daltro G, Filippini P, Mukasa MM, Manicom O. Percutaneous implantation of autologous bone marrow osteoprogenitor cells as treatment of bone avascular necrosis related to sickle cell disease. Open Orthop J. 2008 Apr 25;2:62-5. doi: 10.2174/1874325000802010062.
PubMed 19478932Hernigou P, Daltro G, Flouzat-Lachaniette CH, Roussignol X, Poignard A. Septic arthritis in adults with sickle cell disease often is associated with osteomyelitis or osteonecrosis. Clin Orthop Relat Res. 2010 Jun;468(6):1676-81. doi: 10.1007/s11999-009-1149-3. Epub 2009 Nov 3.
PubMed 19885711Hernigou P, Flouzat-Lachaniette CH, Daltro G, Galacteros F. Talar Osteonecrosis Related to Adult Sickle Cell Disease: Natural Evolution from Early to Late Stages. J Bone Joint Surg Am. 2016 Jul 6;98(13):1113-21. doi: 10.2106/JBJS.15.01074.
PubMed 27385685Daltro GC, Fortuna V, de Souza ES, Salles MM, Carreira AC, Meyer R, Freire SM, Borojevic R. Efficacy of autologous stem cell-based therapy for osteonecrosis of the femoral head in sickle cell disease: a five-year follow-up study. Stem Cell Res Ther. 2015 May 29;6(1):110. doi: 10.1186/s13287-015-0105-2.
PubMed 26021713Daltro G, Franco BA, Faleiro TB, Rosario DAV, Daltro PB, Meyer R, Fortuna V. Use of autologous bone marrow stem cell implantation for osteonecrosis of the knee in sickle cell disease: a preliminary report. BMC Musculoskelet Disord. 2018 May 22;19(1):158. doi: 10.1186/s12891-018-2067-x.
PubMed 29788942
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment for Hip, Knee, Ankle and Shoulder Osteonecrosis With Autologous Mononuclear Cells Transplantation in People With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02448121
- Acronym / sponsor ID
- CAAE 25000039812/2005-99
- Conditions
- Avascular Necrosis of Bone, Sickle Cell Disease
- Keywords
- Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell, Humeral Osteonecrosis, Knee Osteonecrosis, Ankle Osteonecrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Federal University of BahiaUniversity, hospital or other
- Collaborators
- Hospital Universitário Professor Edgard Santos, Oswaldo Cruz Foundation
- Study start
- Aug 2006
- Primary completion
- Jul 2018 (estimated)
- Study completion
- Aug 2018 (estimated)
- First posted
- 19 May 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.
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis
Detailed description
The aim of this study is to evaluate autologous bone marrow stem cells transplantation as a safe and potentially beneficial treatment for sickle cell disease patients with osteonecrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous bone marrow stem cell graft | Experimental | Procedure: Autologous bone marrow stem cell graftAutologous bone marrow stem cell implantation |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Stable sickle cell disease patients
- FICAT classification of osteonecrosis, inclusive of Stage II.
- Diagnosis will be based on X-Ray and magnetic resonance imaging (MRI).
- No infection in affected bones at the time of surgery.
- Patient competent to give informed consent.
Key exclusion criteria (7 listed)
- FICAT stage III or more
- Patients with a history of corticosteroids or on active therapy
- Bone infection at the limb affected by necrosis
- Recurrent painful crises,
- Immunosuppressive drug therapy,
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 |
|---|---|---|
| Autologous bone marrow stem cell graftAll patients will undergo autologous bone marrow stem cell implantation into the necrotic lesion area using a minimally invasive technique. | Procedure or surgery | Autologous bone marrow stem cell graft |
Primary outcome
Change in Functional / Clinical Score of 10 points or more
Time frame: 60 months
Standardized and objective scoring system to rate patient's functional abilities
4 secondary outcomes
Numeric pain intensity scale (0-10)
Time frame: 60 months
Disease progression defined as progression to a fractural stage of osteonecrosis
Time frame: 60 months
Reoperation rate
Time frame: 60 months
Radiological progression
Time frame: 60 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2017.
- Background: PubMed 19478932Hernigou P, Daltro G, Filippini P, Mukasa MM, Manicom O. Percutaneous implantation of autologous bone marrow osteoprogenitor cells as treatment of bone avascular necrosis related to sickle cell disease. Open Orthop J. 2008 Apr 25;2:62-5. doi: 10.2174/1874325000802010062.
- Background: PubMed 19885711Hernigou P, Daltro G, Flouzat-Lachaniette CH, Roussignol X, Poignard A. Septic arthritis in adults with sickle cell disease often is associated with osteomyelitis or osteonecrosis. Clin Orthop Relat Res. 2010 Jun;468(6):1676-81. doi: 10.1007/s11999-009-1149-3. Epub 2009 Nov 3.
- Background: PubMed 27385685Hernigou P, Flouzat-Lachaniette CH, Daltro G, Galacteros F. Talar Osteonecrosis Related to Adult Sickle Cell Disease: Natural Evolution from Early to Late Stages. J Bone Joint Surg Am. 2016 Jul 6;98(13):1113-21. doi: 10.2106/JBJS.15.01074.
- Results publication: PubMed 26021713Daltro GC, Fortuna V, de Souza ES, Salles MM, Carreira AC, Meyer R, Freire SM, Borojevic R. Efficacy of autologous stem cell-based therapy for osteonecrosis of the femoral head in sickle cell disease: a five-year follow-up study. Stem Cell Res Ther. 2015 May 29;6(1):110. doi: 10.1186/s13287-015-0105-2.
- Derived from this study: PubMed 29788942Daltro G, Franco BA, Faleiro TB, Rosario DAV, Daltro PB, Meyer R, Fortuna V. Use of autologous bone marrow stem cell implantation for osteonecrosis of the knee in sickle cell disease: a preliminary report. BMC Musculoskelet Disord. 2018 May 22;19(1):158. doi: 10.1186/s12891-018-2067-x.
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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.
GIldasio Daltro, MD study director
Federal University of Bahia
Vitor Fortuna, PhD principal investigator
Federal University of Bahia
Roberto Meyer, MD study chair
Federal University of Bahia
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Autologous bone marrow stem cell graft in people with Avascular Necrosis of Bone and Sickle Cell Disease, run by Federal University of Bahia.
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 6 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 20 Nov 2017. 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 combined 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.
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?
Results are not available in the source record.
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?
Change in Functional / Clinical Score of 10 points or more (time frame: 60 months). 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 NCT02448121, which also lists 5 linked publications.
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 20 Nov 2017.