Multiple SclerosisPhase 1NCT03113162
Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) for Multiple Sclerosis
A Phase 1 study testing Autologous Hematopoietic Stem Cell and BEAM Regimen in people with Multiple Sclerosis, run by Makati Medical Center at 1 site in Philippines.
Trial summary
This is a patient-sponsored study that evaluates the safety and efficacy of reduced-intensity immunoablation followed by a single dose autologous hematopoetic stem cell transplantation in patients diagnosed with multiple sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Autologous Hematopoietic Stem Cell and BEAM Regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 sitePhilippines
- Study dates
- 29 May 2015to 29 May 2022
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosed with progressive multiple sclerosis with or without relapses
- EDSS score between 1.5 and 7.0, including documented rapid progression over the previous year unresponsive to conventional therapies or no available treatment options
- Aged between 18 and 60 with a history of at least one enhancing lesion on brain MRI
- With absolute neutrophil count ≥ 1,000/mm^3, platelet count ≥ 100,000/mm^3 and hemoglobin ≥ 9.0 g/dL
Who cannot join
- Patients with cardiac, renal, pulmonary, hepatic, or other organ impairment that would limit their ability to receive dose-intensive immunosuppressive therapy, high-dose chemotherapy, and/or Autologous HSCT
- Patients with any active or chronic infection e.g. uncontrolled viral, fungal, or bacterial infection
- Uncontrolled diabetes
- Patients who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C
- Patients whose life expectancy is severely limited by another illness
- Patients with evidence of myelodysplasia or other non-autoimmune cytopenia
Show all 10Show fewer
- Patients having received a cytotoxic agent within one month prior to this study
- Patients who are pregnant or at risk of pregnancy, including those unwilling to practice
- Patients with psychiatric illness, mental deficiency, or cognitive dysfunction
- Patients unable to give written informed consent in accordance with research ethics board guidelines
Trial locations and contacts
1 site in 1 country.
Philippines1 site, 1 recruiting
- Recruiting
Makati Medical Center
Makati, 1229
Marviel T Berboso, RN, 8888999 ext. 3613, Inquiry.CTC@makatimed.net.ph
About this study
Official title: Evaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis
This is a patient-sponsored study that evaluates the safety and efficacy of reduced-intensity immunoablation followed by a single dose autologous hematopoetic stem cell transplantation in patients diagnosed with multiple sclerosis. Patients are followed-up after 1 month, 3 months, 6 months and 12 months post-transplantation.
- Conditions
- Multiple Sclerosis
- Being tested
- Autologous Hematopoietic Stem Cell, BEAM Regimen
- Keywords
- multiple sclerosis, HSCT
- Medical subject headings
- Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Stem CellBiological
BEAM RegimenDrug
Also called BCNU, Etoposide, Cytarabine, Melphalan
Reduced-intensity BEAM for Immunoablation
| Study group | Role | What they receive |
|---|---|---|
| Autologous Hematopoietic Stem Cell with BEAM Regimen | Experimental | Autologous HSCT following Reduced-Intensity BEAM Regimen Receives: Biological: Autologous Hematopoietic Stem Cell; Drug: BEAM Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Adverse Events
Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities
Measured: 12 months
Secondary outcomes (2)
Efficacy: EDSS Score
Measurement of disease progression by change in baseline of EDSS score
Measured: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
Efficacy: RAND-36 Score
Measurement of Quality of Life by change in baseline of RAND-36 score
Measured: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2017
- Study start29 May 2015
- Primary completion29 May 2020 (estimated)
- Study completion29 May 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (21)Papers the sponsor has linked to this study.
- Other trials for Multiple SclerosisStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Makati Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Darwin A. Dasig, Makati Medical Center
- Investigators
- Darwin Albert A Dasig, MD, Principal investigator, Makati Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03113162
- Sponsor's protocol ID
- MMCIRB 2015-024
- Status last verified
- May 2017
- First submitted
- 10 Apr 2017
- Last updated
- 5 May 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Akkok CA, Liseth K, Hervig T, Ryningen A, Bruserud O, Ersvaer E. Use of different DMSO concentrations for cryopreservation of autologous peripheral blood stem cell grafts does not have any major impact on levels of leukocyte- and platelet-derived soluble mediators. Cytotherapy. 2009;11(6):749-60. doi: 10.3109/14653240902980443.
PubMed 19878061Appelbaum FR, Bacigalupo A, Soiffer R. Anti-T cell antibodies as part of the preparative regimen in hematopoietic cell transplantation--a debate. Biol Blood Marrow Transplant. 2012 Jan;18(1 Suppl):S111-5. doi: 10.1016/j.bbmt.2011.11.002. No abstract available.
PubMed 22226093Burt RK, Balabanov R, Han X, Sharrack B, Morgan A, Quigley K, Yaung K, Helenowski IB, Jovanovic B, Spahovic D, Arnautovic I, Lee DC, Benefield BC, Futterer S, Oliveira MC, Burman J. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA. 2015 Jan 20;313(3):275-84. doi: 10.1001/jama.2014.17986.
PubMed 25602998Burt RK, Loh Y, Cohen B, Stefoski D, Balabanov R, Katsamakis G, Oyama Y, Russell EJ, Stern J, Muraro P, Rose J, Testori A, Bucha J, Jovanovic B, Milanetti F, Storek J, Voltarelli JC, Burns WH. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. Lancet Neurol. 2009 Mar;8(3):244-53. doi: 10.1016/S1474-4422(09)70017-1. Epub 2009 Jan 29.
PubMed 19186105Bruck W. The pathology of multiple sclerosis is the result of focal inflammatory demyelination with axonal damage. J Neurol. 2005 Nov;252 Suppl 5:v3-9. doi: 10.1007/s00415-005-5002-7.
PubMed 16254699von Budingen HC, Kuo TC, Sirota M, van Belle CJ, Apeltsin L, Glanville J, Cree BA, Gourraud PA, Schwartzburg A, Huerta G, Telman D, Sundar PD, Casey T, Cox DR, Hauser SL. B cell exchange across the blood-brain barrier in multiple sclerosis. J Clin Invest. 2012 Dec;122(12):4533-43. doi: 10.1172/JCI63842. Epub 2012 Nov 19.
PubMed 23160197Cabezudo E, Dalmases C, Ruz M, Sanchez JA, Torrico C, Sola C, Querol S, Garcia J. Leukapheresis components may be cryopreserved at high cell concentrations without additional loss of HPC function. Transfusion. 2000 Oct;40(10):1223-7. doi: 10.1046/j.1537-2995.2000.40101223.x.
PubMed 11061859Costantino CM, Baecher-Allan C, Hafler DA. Multiple sclerosis and regulatory T cells. J Clin Immunol. 2008 Nov;28(6):697-706. doi: 10.1007/s10875-008-9236-x. Epub 2008 Sep 2.
PubMed 18763026De Boer F, Drager AM, Van der Wall E, Pinedo HM, Schuurhuis GJ. Changes in L-selectin expression on CD34-positive cells upon cryopreservation of peripheral blood stem cell transplants. Bone Marrow Transplant. 1998 Dec;22(11):1103-10. doi: 10.1038/sj.bmt.1701495.
PubMed 9877274Farge D, Labopin M, Tyndall A, Fassas A, Mancardi GL, Van Laar J, Ouyang J, Kozak T, Moore J, Kotter I, Chesnel V, Marmont A, Gratwohl A, Saccardi R. Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica. 2010 Feb;95(2):284-92. doi: 10.3324/haematol.2009.013458. Epub 2009 Sep 22.
PubMed 19773265Fassas A, Anagnostopoulos A, Kazis A, Kapinas K, Sakellari I, Kimiskidis V, Tsompanakou A. Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study. Bone Marrow Transplant. 1997 Oct;20(8):631-8. doi: 10.1038/sj.bmt.1700944.
PubMed 9383225Fassas A, Kimiskidis VK. Autologous hemopoietic stem cell transplantation in the treatment of multiple sclerosis: rationale and clinical experience. J Neurol Sci. 2004 Aug 15;223(1):53-8. doi: 10.1016/j.jns.2004.04.020.
PubMed 15261561Frischer JM, Bramow S, Dal-Bianco A, Lucchinetti CF, Rauschka H, Schmidbauer M, Laursen H, Sorensen PS, Lassmann H. The relation between inflammation and neurodegeneration in multiple sclerosis brains. Brain. 2009 May;132(Pt 5):1175-89. doi: 10.1093/brain/awp070. Epub 2009 Mar 31.
PubMed 19339255Iannalfi A, Bambi F, Tintori V, Lacitignola L, Bernini G, Mariani MP, Sanvito MC, Pagliai F, Brandigi F, Muscarella E, Tapinassi F, Faulkner L. Peripheral blood progenitor uncontrolled-rate freezing: a single pediatric center experience. Transfusion. 2007 Dec;47(12):2202-6. doi: 10.1111/j.1537-2995.2007.01447.x. Epub 2007 Aug 21.
PubMed 17714421Mancardi GL, Sormani MP, Gualandi F, Saiz A, Carreras E, Merelli E, Donelli A, Lugaresi A, Di Bartolomeo P, Rottoli MR, Rambaldi A, Amato MP, Massacesi L, Di Gioia M, Vuolo L, Curro D, Roccatagliata L, Filippi M, Aguglia U, Iacopino P, Farge D, Saccardi R; ASTIMS Haemato-Neurological Collaborative Group, On behalf of the Autoimmune Disease Working Party (ADWP) of the European Group for Blood and Marrow Transplantation (EBMT); ASTIMS Haemato-Neurological Collaborative Group On behalf of the Autoimmune Disease Working Party ADWP of the European Group for Blood and Marrow Transplantation EBMT. Autologous hematopoietic stem cell transplantation in multiple sclerosis: a phase II trial. Neurology. 2015 Mar 10;84(10):981-8. doi: 10.1212/WNL.0000000000001329. Epub 2015 Feb 11.
PubMed 25672923
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis
- ClinicalTrials.gov ID
- NCT03113162
- Acronym / sponsor ID
- MMCIRB 2015-024
- Conditions
- Multiple Sclerosis
- Keywords
- multiple sclerosis, HSCT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Makati Medical CenterUniversity, hospital or other
- Study start
- 29 May 2015 (actual)
- Primary completion
- 29 May 2020 (estimated)
- Study completion
- 29 May 2022 (estimated)
- First posted
- 13 Apr 2017 (actual)
- 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 is a patient-sponsored study that evaluates the safety and efficacy of reduced-intensity immunoablation followed by a single dose autologous hematopoetic stem cell transplantation in patients diagnosed with multiple sclerosis. Patients are followed-up after 1 month, 3 months, 6 months and 12 months post-transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Hematopoietic Stem Cell with BEAM Regimen | Experimental | Biological: Autologous Hematopoietic Stem Cell, Drug: BEAM RegimenAutologous HSCT following Reduced-Intensity BEAM Regimen |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Diagnosed with progressive multiple sclerosis with or without relapses
- EDSS score between 1.5 and 7.0, including documented rapid progression over the previous year unresponsive to conventional therapies or no available treatment options
- Aged between 18 and 60 with a history of at least one enhancing lesion on brain MRI
- With absolute neutrophil count ≥ 1,000/mm^3, platelet count ≥ 100,000/mm^3 and hemoglobin ≥ 9.0 g/dL
Key exclusion criteria (10 listed)
- Patients with cardiac, renal, pulmonary, hepatic, or other organ impairment that would limit their ability to receive dose-intensive immunosuppressive therapy, high-dose chemotherapy, and/or Autologous HSCT
- Patients with any active or chronic infection e.g. uncontrolled viral, fungal, or bacterial infection
- Uncontrolled diabetes
- Patients who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C
- Patients whose life expectancy is severely limited by another illness
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 Hematopoietic Stem Cell | Biological | Autologous Hematopoietic Stem Cell with BEAM Regimen |
| BEAM RegimenAlso: BCNU, Etoposide, Cytarabine, MelphalanReduced-intensity BEAM for Immunoablation | Drug | Autologous Hematopoietic Stem Cell with BEAM Regimen |
Primary outcome
Safety: Adverse Events
Time frame: 12 months
Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities
2 secondary outcomes
Efficacy: EDSS Score
Time frame: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
Efficacy: RAND-36 Score
Time frame: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2017.
- Background: PubMed 19878061Akkok CA, Liseth K, Hervig T, Ryningen A, Bruserud O, Ersvaer E. Use of different DMSO concentrations for cryopreservation of autologous peripheral blood stem cell grafts does not have any major impact on levels of leukocyte- and platelet-derived soluble mediators. Cytotherapy. 2009;11(6):749-60. doi: 10.3109/14653240902980443.
- Background: PubMed 22226093Appelbaum FR, Bacigalupo A, Soiffer R. Anti-T cell antibodies as part of the preparative regimen in hematopoietic cell transplantation--a debate. Biol Blood Marrow Transplant. 2012 Jan;18(1 Suppl):S111-5. doi: 10.1016/j.bbmt.2011.11.002. No abstract available.
- Background: PubMed 25602998Burt RK, Balabanov R, Han X, Sharrack B, Morgan A, Quigley K, Yaung K, Helenowski IB, Jovanovic B, Spahovic D, Arnautovic I, Lee DC, Benefield BC, Futterer S, Oliveira MC, Burman J. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA. 2015 Jan 20;313(3):275-84. doi: 10.1001/jama.2014.17986.
- Background: PubMed 19186105Burt RK, Loh Y, Cohen B, Stefoski D, Balabanov R, Katsamakis G, Oyama Y, Russell EJ, Stern J, Muraro P, Rose J, Testori A, Bucha J, Jovanovic B, Milanetti F, Storek J, Voltarelli JC, Burns WH. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. Lancet Neurol. 2009 Mar;8(3):244-53. doi: 10.1016/S1474-4422(09)70017-1. Epub 2009 Jan 29.
- Background: PubMed 16254699Bruck W. The pathology of multiple sclerosis is the result of focal inflammatory demyelination with axonal damage. J Neurol. 2005 Nov;252 Suppl 5:v3-9. doi: 10.1007/s00415-005-5002-7.
- Background: PubMed 23160197von Budingen HC, Kuo TC, Sirota M, van Belle CJ, Apeltsin L, Glanville J, Cree BA, Gourraud PA, Schwartzburg A, Huerta G, Telman D, Sundar PD, Casey T, Cox DR, Hauser SL. B cell exchange across the blood-brain barrier in multiple sclerosis. J Clin Invest. 2012 Dec;122(12):4533-43. doi: 10.1172/JCI63842. Epub 2012 Nov 19.
- Background: PubMed 11061859Cabezudo E, Dalmases C, Ruz M, Sanchez JA, Torrico C, Sola C, Querol S, Garcia J. Leukapheresis components may be cryopreserved at high cell concentrations without additional loss of HPC function. Transfusion. 2000 Oct;40(10):1223-7. doi: 10.1046/j.1537-2995.2000.40101223.x.
- Background: PubMed 18763026Costantino CM, Baecher-Allan C, Hafler DA. Multiple sclerosis and regulatory T cells. J Clin Immunol. 2008 Nov;28(6):697-706. doi: 10.1007/s10875-008-9236-x. Epub 2008 Sep 2.
- Background: PubMed 9877274De Boer F, Drager AM, Van der Wall E, Pinedo HM, Schuurhuis GJ. Changes in L-selectin expression on CD34-positive cells upon cryopreservation of peripheral blood stem cell transplants. Bone Marrow Transplant. 1998 Dec;22(11):1103-10. doi: 10.1038/sj.bmt.1701495.
- Background: PubMed 19773265Farge D, Labopin M, Tyndall A, Fassas A, Mancardi GL, Van Laar J, Ouyang J, Kozak T, Moore J, Kotter I, Chesnel V, Marmont A, Gratwohl A, Saccardi R. Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica. 2010 Feb;95(2):284-92. doi: 10.3324/haematol.2009.013458. Epub 2009 Sep 22.
- Background: PubMed 9383225Fassas A, Anagnostopoulos A, Kazis A, Kapinas K, Sakellari I, Kimiskidis V, Tsompanakou A. Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study. Bone Marrow Transplant. 1997 Oct;20(8):631-8. doi: 10.1038/sj.bmt.1700944.
- Background: PubMed 15261561Fassas A, Kimiskidis VK. Autologous hemopoietic stem cell transplantation in the treatment of multiple sclerosis: rationale and clinical experience. J Neurol Sci. 2004 Aug 15;223(1):53-8. doi: 10.1016/j.jns.2004.04.020.
- Individual participant dataSharing planned: No
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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.
Darwin Albert A Dasig, MD principal investigator
Makati Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Hematopoietic Stem Cell and BEAM Regimen in people with Multiple Sclerosis, run by Makati Medical Center at 1 site in Philippines.
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 10 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 5 May 2017. 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 Philippines. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Marviel T Berboso, RN (8888999 ext. 3613, Inquiry.CTC@makatimed.net.ph), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Adverse Events (time frame: 12 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 NCT03113162, which also lists 21 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 5 May 2017.