Abili-T: Study of Tcelna (Imilecleucel-T) in Secondary Progressive Multiple Sclerosis

Sponsor

Opexa Therapeutics, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT01684761

Collaborator

(none)

183

Enrollment

Locations

Arms

Duration (Months)

5.1

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to determine whether Tcelna (imilecleucel-T, autologous T-Cell Immunotherapy) is effective in the treatment of secondary progressive multiple sclerosis (SPMS).

Condition or Disease	Intervention/Treatment	Phase
Autoimmune Diseases of the Nervous System Multiple Sclerosis Secondary Progressive Multiple Sclerosis Disease Progression Brain Atrophy	Biological: Tcelna Biological: Placebo	Phase 2

Detailed Description

Subjects whose myelin reactive T-cell can be identified by EPA will are randomized and provide blood to manufacture Tcelna. Approximately 5 weeks after receipt of the subject's whole blood procurement, the subjects will receive either Tcelna or placebo and will complete baseline assessments and will receive study treatments at Weeks 0, 4, 8, 12, and 24 (Visits 3-7), totaling 5 doses in year one.

Approximately one month prior to the Week 52 visit a second blood procurement will be performed and the subject will receive the second series of treatments as received in the first year study schedule. Subjects will be evaluated for changes in disability and cognitive function every 3 months, and radiographic changes annually.

Study Design

Study Type:

Interventional

Actual Enrollment :

183 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

A Phase 2 Double-Blind, Placebo Controlled Multi-Center Study to Evaluate the Efficacy and Safety of Tcelna in Subjects With Secondary Progressive Multiple Sclerosis

Study Start Date :

Aug 1, 2012

Actual Primary Completion Date :

Oct 1, 2016

Actual Study Completion Date :

Oct 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Tcelna 30-45 x 10E6 total cells in 2 ml. Subjects receive two annual courses of 5 subcutaneous doses each year (at 0, 4, 8, 12 and 24 weeks).	Biological: Tcelna Autologous pool of myelin reactive T-cells (MRTC) expanded ex vivo with immunodominant epitopes selected from the three myelin antigens, MBP, PLP and MOG on a per subject basis. Attenuated by irradiation to prevent further proliferation before releasing product for administration.
Placebo Comparator: Placebo Tcelna inactive ingredients (without cells) totaling 2 ml per dose. Administered subcutaneously with same two year treatment regimen as experimental treatment arm.	Biological: Placebo 2 ml of Tcelna excipients, prepared daily as individual doses and irradiated before releasing product for administration.

Arm

Intervention/Treatment

Experimental: Tcelna

30-45 x 10E6 total cells in 2 ml. Subjects receive two annual courses of 5 subcutaneous doses each year (at 0, 4, 8, 12 and 24 weeks).

Biological: Tcelna

Autologous pool of myelin reactive T-cells (MRTC) expanded ex vivo with immunodominant epitopes selected from the three myelin antigens, MBP, PLP and MOG on a per subject basis. Attenuated by irradiation to prevent further proliferation before releasing product for administration.

Placebo Comparator: Placebo

Tcelna inactive ingredients (without cells) totaling 2 ml per dose. Administered subcutaneously with same two year treatment regimen as experimental treatment arm.

Biological: Placebo

2 ml of Tcelna excipients, prepared daily as individual doses and irradiated before releasing product for administration.

Outcome Measures

Primary Outcome Measures

Brain Atrophy [2 Years]
The percentage of brain volume change (atrophy) as measured on 24 month MRIs calculated by the central MRI facility.

Secondary Outcome Measures

Disease Progression [2 Years]
The percentage of subjects with sustained progression with definitions of sustained effect at 3 months and 6 months.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Diagnosed with MS as defined by the modified McDonald criteria
SPMS defined as relapsing-remitting disease with recent progression in MS-related neurological deficits
EDSS score 3.0 - 6.0, inclusively
Presence of myelin reactive T-cells

Exclusion Criteria:

Diagnosed with primary progressive MS
Treatment with beta-interferon, glatiramer acetate or dimethyl fumarate 30 days prior to screening
Treatment with ACTH, any over-the-counter or prescription corticosteroids 60 days prior to screening
Treatment with IVIG, plasmapheresis or cytopheresis 90 days prior to screening
Treatment with mitoxantrone, teriflunomide, fingolimod, natalizumab, azathioprine, cyclosporine, methotrexate or mycophenolate mofetil 1 year prior to baseline
Any prior treatment with cladribine, cyclophosphamide, total lymphoid irradiation, T cell or T cell receptor products, or any therapeutic monoclonal antibody, except natalizumab
Previous treatment with any other MS investigational drug 1 year prior to screening
All non-MS investigational drugs must have a minimum washout of 30 days prior to screening or 5 half-lives, whatever is the longest period of time.
HIV or hepatitis infection
History of cancer
Any other significant medical condition that, in the opinion of the investigator, could cause CNS tissue damage or limit its repair.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	HOPE Research Institute	Phoenix	Arizona	United States	85050
2	Northwest NeuroSpecialists, LLC	Tucson	Arizona	United States	85741
3	Alta Bates Summit Medical Center, The Research and Education Development Institute	Berkeley	California	United States	94705
4	Neurology Associates, P.A.	Maitland	Florida	United States	32751
5	University of Miami	Miami	Florida	United States	33136
6	Collier Neurologic Specialists, LLC	Naples	Florida	United States	34102
7	Neurological Services of Orlando	Orlando	Florida	United States	32806
8	Meridien Research	Tampa	Florida	United States	33606
9	Vero Beach Neurology	Vero Beach	Florida	United States	32960
10	Shepherd Center	Atlanta	Georgia	United States	30309
11	Consultants In Neurology, Ltd.	Northbrook	Illinois	United States	60062
12	Fort Wayne Neurological Center	Fort Wayne	Indiana	United States	46804
13	Josephson Wallack Munshower Neurology, PC	Indianapolis	Indiana	United States	46256
14	University of Kansas Medical Center	Kansas City	Kansas	United States	66160
15	Associates in Neurology	Lexington	Kentucky	United States	40503
16	Saint Elizabeth's Medical Center	Boston	Massachusetts	United States	02135
17	Island Neurological Assoicates, PC	Plainview	New York	United States	11803
18	University Hospital and Medical Center Stony Brook New York	Stony Brook	New York	United States	11794-8121
19	The Neurological Institute, PA	Charlotte	North Carolina	United States	28204
20	Carolinas Medical Center Neurology	Charlotte	North Carolina	United States	28207
21	PMG Research of Charlotte	Charlotte	North Carolina	United States	28209
22	Neurology Specialists, Inc	Dayton	Ohio	United States	45408
23	Providence Medical Group - Medford	Medford	Oregon	United States	97504
24	Providence St. Vincent Medical Center - Northwest MS Center	Portland	Oregon	United States	97225
25	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
26	The Maxine Mesinger MS Clinic/Baylor College of Medicine	Houston	Texas	United States	77030
27	Central Texas Neurology	Round Rock	Texas	United States	78681
28	Integra Clinical Research, LLC	San Antonio	Texas	United States	78229
29	Fletcher Allen Health Care - Neurology Service	Burlington	Vermont	United States	05401
30	Hampton Roads Neurology	Newport News	Virginia	United States	23601
31	Neurological Associates, Inc	Richmond	Virginia	United States	23226
32	Swedish Neuroscience Institute	Issaquah	Washington	United States	98029
33	Swedish Neuroscience Institute	Seattle	Washington	United States	98122
34	University of Ottawa	Ottawa	Ontario	Canada	K1H 8L6
35	Recherche Sepmus Inc.	Greenfield Park	Quebec	Canada	J4V 2J2
36	Montreal Neurological Institute and Hospital	Montreal	Quebec	Canada	H3A 2B4