Tolerogenic Dendritic Cells as a Therapeutic Strategy for the Treatment of Multiple Sclerosis Patients (TOLERVIT-MS)

NCT ID: NCT02903537

Last Updated: 2023-07-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-06

Study Completion Date

2023-12-31

Brief Summary

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The purpose of this study is to determine the safety and tolerability of the intranodal administration of autologous monocyte-derived dendritic cells tolerised with Vitamin-D3 and pulsed with myelin peptides (tolDC-VitD3) in multiple sclerosis patients . To select the most appropriate regime for the development of future therapeutic trials.

To evaluate the preliminary proof of concept by clinical and/or radiological activity and immunological markers.

Detailed Description

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Phase I dose ascending ("best of five") clinical trial.

First group will start by intranodal injection in cervical lymph nodes of 5\*10\^6 tolDC-VitD3.

Up titration depending on security outcomes to 10\*10\^6 tolDC-VitD3, same route in second cohort dose and next uptitration to 15\*10\^6 tolDC-VitD3.

Six cycles per patient with the following schema: for the first four cycles the administration will be each 2 weeks, for the remaining 2 cycles administration each 4 weeks.

A last cohort with the dose identified in the previous groups, administered in patients treated with beta interferon, same route, same dose schema.

Conditions

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Multiple Sclerosis, Relapsing-Remitting Multiple Sclerosis, Chronic Progressive

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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5 * 10 ^6 tolDC-VitD3

5 million autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Group Type EXPERIMENTAL

Autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Intervention Type DRUG

Intranodal administration

10 * 10 ^6 tolDC-VitD3

10 million autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3).

Group Type EXPERIMENTAL

Autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Intervention Type DRUG

Intranodal administration

15 * 10 ^6 tolDC-VitD3

15 million autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3).

Group Type EXPERIMENTAL

Autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Intervention Type DRUG

Intranodal administration

Interferon-beta

Additional group (patients treated with beta-interferon) will receive the selected dose of those 3 previously cohorts studied

Group Type EXPERIMENTAL

Autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Intervention Type DRUG

Intranodal administration

Interferon-beta

Intervention Type DRUG

Subcutaneous administration interferon-beta

Interventions

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Autologous VitD3 tolerogenic monocyte-derived dendritic cells loaded with a pool of myelin peptides (tolDC-VitD3)

Intranodal administration

Intervention Type DRUG

Interferon-beta

Subcutaneous administration interferon-beta

Intervention Type DRUG

Other Intervention Names

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tolDC-VitD3 beta-interferon

Eligibility Criteria

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Inclusion Criteria

1. EDSS of 0.0 - 6.5.
2. Multiple Sclerosis according to 2010 revised Mc Donald criteria, and less than 15 years of evolution of disease.
3. Patients with:

* Active relapsing remitting multiple sclerosis (RRMS) (more than 1 relapse in last year and/or occurrence of ≥3 new T2 lesions or Gd positive) who not wish to be treated with current therapies.
* Low activity RRMS (1 relapse in last year or occurrence of 1 or 2 T2 lesions or Gd positive) without treatment.
* Progressive forms of MS with activity (at least 1 relapse in last year or occurrence 1 or 2 T2 lesions or Gd positive).
* RRMS treated with interferon beta (Additional group)
4. T cell proliferation to the pool of myelin peptides against which is to induce immune tolerance: Myelin basic protein (MBP)13-32, MBP83-99, MBP111-129, MBP146-170, proteolipid protein (PLP) 139-154, Myelin oligodendrocyte glycoprotein (MOG)1-20, MOG35 -55).
5. Adequate peripheral venous access.
6. Signed informed consent.

Exclusion Criteria

1. Use of corticosteroids during the prior 4 weeks.
2. Use of interferon beta -in patients who is retired by inefficiency or other causes- and glatiramer acetate in the 4 weeks prior.
3. Use of fingolimod, dimethylfumarate, natalizumab, immunoglobulins or plasmapheresis at 12 weeks; and teriflunomide in the 15 weeks prior.
4. Use of azathioprine, mitoxantrone, rituximab, methotrexate, cyclophosphamide, cyclosporine, alemtuzumab or other immunosuppressive drug, except corticosteroids, at any time.
5. Bone marrow or stem cell transplant at any time.
6. Relapse during the month prior of starting treatment. If it appears and the patient meets the eligibility criteria, must wait long enough until the end of the 30 days free of relapse. If corticosteroids are administered, the MRI performed during this period should not be considered, and a new MRI will be performed at 4 weeks after administration of corticosteroids.
7. Pregnancy or planning pregnancy within the next 12 months and breastfeeding.
8. Fertile patients who are not using an appropriate method of contraception. If the patient is menopausal or sterile it must be documented in the medical record.
9. Abusing drugs or alcohol.
10. Inability to undergo MRI evaluations.
11. Seropositivity for HIV, hepatitis B or C and/or syphilis.
12. History of oncological disease.
13. Clinically relevant concomitant disease: cardiac, pulmonary, neurological, renal or other major illness.
14. Splenectomy.
15. Dementia, psychiatric problems or other comorbidities that might interfere protocol compliance.
16. To be participating in another clinical study or to have participated in one in the last 3 months.
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Clinica Universidad de Navarra, Universidad de Navarra

OTHER

Sponsor Role collaborator

Fundació Institut Germans Trias i Pujol

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cristina Ramo, MD.PhD.Neurologist

Role: PRINCIPAL_INVESTIGATOR

Badalona Hospital Germans Trias i Pujol. Neurology service. Multiple Sclerosis department

Eva Martínez-Cáceres, MD.PhD.Immunology

Role: STUDY_DIRECTOR

Badalona Hospital Germans Trias i Pujol. Immunology

Locations

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Hospital Universitari Germans Trias i Pujol

Badalona, Barcelona, Spain

Site Status RECRUITING

Clínica Universidad de Navarra

Pamplona, Navarre, Spain

Site Status NOT_YET_RECRUITING

Countries

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Spain

Central Contacts

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Cristina Ramo, MD.PhD. Neurologist

Role: CONTACT

+34934978433

Ana M Barriocanal, MD.PhD.Clinical Pharmacologist

Role: CONTACT

+34934978488

Facility Contacts

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Cristina Ramo, MD.PhD

Role: primary

+34934978433

Ana M Barriocanal, MD.PhD

Role: backup

+34934978488

Jaime Gállego, MD.PhD

Role: primary

+34948255400 ext. 3594

Amaya Izal

Role: backup

+34948255400 ext. 2724

References

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Lutterotti A, Yousef S, Sputtek A, Sturner KH, Stellmann JP, Breiden P, Reinhardt S, Schulze C, Bester M, Heesen C, Schippling S, Miller SD, Sospedra M, Martin R. Antigen-specific tolerance by autologous myelin peptide-coupled cells: a phase 1 trial in multiple sclerosis. Sci Transl Med. 2013 Jun 5;5(188):188ra75. doi: 10.1126/scitranslmed.3006168.

Reference Type BACKGROUND
PMID: 23740901 (View on PubMed)

Giannoukakis N, Phillips B, Finegold D, Harnaha J, Trucco M. Phase I (safety) study of autologous tolerogenic dendritic cells in type 1 diabetic patients. Diabetes Care. 2011 Sep;34(9):2026-32. doi: 10.2337/dc11-0472. Epub 2011 Jun 16.

Reference Type BACKGROUND
PMID: 21680720 (View on PubMed)

Benham H, Nel HJ, Law SC, Mehdi AM, Street S, Ramnoruth N, Pahau H, Lee BT, Ng J, Brunck ME, Hyde C, Trouw LA, Dudek NL, Purcell AW, O'Sullivan BJ, Connolly JE, Paul SK, Le Cao KA, Thomas R. Citrullinated peptide dendritic cell immunotherapy in HLA risk genotype-positive rheumatoid arthritis patients. Sci Transl Med. 2015 Jun 3;7(290):290ra87. doi: 10.1126/scitranslmed.aaa9301.

Reference Type BACKGROUND
PMID: 26041704 (View on PubMed)

Naranjo-Gomez M, Raich-Regue D, Onate C, Grau-Lopez L, Ramo-Tello C, Pujol-Borrell R, Martinez-Caceres E, Borras FE. Comparative study of clinical grade human tolerogenic dendritic cells. J Transl Med. 2011 Jun 9;9:89. doi: 10.1186/1479-5876-9-89.

Reference Type BACKGROUND
PMID: 21658226 (View on PubMed)

Raiotach-Regue D, Grau-Lopez L, Naranjo-Gomez M, Ramo-Tello C, Pujol-Borrell R, Martinez-Caceres E, Borras FE. Stable antigen-specific T-cell hyporesponsiveness induced by tolerogenic dendritic cells from multiple sclerosis patients. Eur J Immunol. 2012 Mar;42(3):771-82. doi: 10.1002/eji.201141835.

Reference Type BACKGROUND
PMID: 22488365 (View on PubMed)

Mansilla MJ, Selles-Moreno C, Fabregas-Puig S, Amoedo J, Navarro-Barriuso J, Teniente-Serra A, Grau-Lopez L, Ramo-Tello C, Martinez-Caceres EM. Beneficial effect of tolerogenic dendritic cells pulsed with MOG autoantigen in experimental autoimmune encephalomyelitis. CNS Neurosci Ther. 2015 Mar;21(3):222-30. doi: 10.1111/cns.12342. Epub 2014 Nov 18.

Reference Type BACKGROUND
PMID: 25403984 (View on PubMed)

Raich-Regue D, Naranjo-Gomez M, Grau-Lopez L, Ramo C, Pujol-Borrell R, Martinez-Caceres E, Borras FE. Differential effects of monophosphoryl lipid A and cytokine cocktail as maturation stimuli of immunogenic and tolerogenic dendritic cells for immunotherapy. Vaccine. 2012 Jan 5;30(2):378-87. doi: 10.1016/j.vaccine.2011.10.081. Epub 2011 Nov 12.

Reference Type BACKGROUND
PMID: 22085546 (View on PubMed)

Ten Brinke A, Hilkens CM, Cools N, Geissler EK, Hutchinson JA, Lombardi G, Lord P, Sawitzki B, Trzonkowski P, Van Ham SM, Martinez-Caceres EM. Clinical Use of Tolerogenic Dendritic Cells-Harmonization Approach in European Collaborative Effort. Mediators Inflamm. 2015;2015:471719. doi: 10.1155/2015/471719. Epub 2015 Dec 24.

Reference Type BACKGROUND
PMID: 26819498 (View on PubMed)

Bell GM, Anderson AE, Diboll J, Reece R, Eltherington O, Harry RA, Fouweather T, MacDonald C, Chadwick T, McColl E, Dunn J, Dickinson AM, Hilkens CM, Isaacs JD. Autologous tolerogenic dendritic cells for rheumatoid and inflammatory arthritis. Ann Rheum Dis. 2017 Jan;76(1):227-234. doi: 10.1136/annrheumdis-2015-208456. Epub 2016 Apr 26.

Reference Type BACKGROUND
PMID: 27117700 (View on PubMed)

Mansilla MJ, Contreras-Cardone R, Navarro-Barriuso J, Cools N, Berneman Z, Ramo-Tello C, Martinez-Caceres EM. Cryopreserved vitamin D3-tolerogenic dendritic cells pulsed with autoantigens as a potential therapy for multiple sclerosis patients. J Neuroinflammation. 2016 May 20;13(1):113. doi: 10.1186/s12974-016-0584-9.

Reference Type BACKGROUND
PMID: 27207486 (View on PubMed)

Willekens B, Presas-Rodriguez S, Mansilla MJ, Derdelinckx J, Lee WP, Nijs G, De Laere M, Wens I, Cras P, Parizel P, Van Hecke W, Ribbens A, Billiet T, Adams G, Couttenye MM, Navarro-Barriuso J, Teniente-Serra A, Quirant-Sanchez B, Lopez-Diaz de Cerio A, Inoges S, Prosper F, Kip A, Verheij H, Gross CC, Wiendl H, Van Ham MS, Ten Brinke A, Barriocanal AM, Massuet-Vilamajo A, Hens N, Berneman Z, Martinez-Caceres E, Cools N, Ramo-Tello C; RESTORE consortium. Tolerogenic dendritic cell-based treatment for multiple sclerosis (MS): a harmonised study protocol for two phase I clinical trials comparing intradermal and intranodal cell administration. BMJ Open. 2019 Sep 9;9(9):e030309. doi: 10.1136/bmjopen-2019-030309.

Reference Type DERIVED
PMID: 31501122 (View on PubMed)

Related Links

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http://www.afactt.eu

European Cooperation in Science and Technology (COST), Action BM1305 "Action to focus and accelerate cell-based tolerogenic therapies"

Other Identifiers

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2015-003541-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TOLERVIT-MS

Identifier Type: -

Identifier Source: org_study_id

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