Safety Study of Human Neural Stem Cells Injections for Secondary Progressive Multiple Sclerosis Patients (NSC-SPMS)

Casa Sollievo della Sofferenza IRCCS

Status and phase: Completed; Phase 1

Conditions: Secondary-progressive Multiple Sclerosis

Treatments: Biological: Human Neural Stem Cells

Study type: Interventional

Funder types: Other

Identifiers

NCT03282760

NSC SPMS

2015-004855-37 (EudraCT Number)

Details and patient eligibility

About

This will be a phase I, open, multicenter, international study performed by 3 participating centres across two countries (Italy and Switzerland). Fifteen to 24 patients affected by SPMS will be enrolled, according to a "standard" phase I design over 18 months. All patients will enter a 3 months run in phase. Thereafter they will receive one of four different doses of allogenic hNSCs (dose A=5 millions hNSCs; dose B=10 millions hNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs). Following hNSCs injection, all SPMS patients will receive immunosuppression with tacrolimus for 6 months. Patients will be clinically followed monthly for 1 year and then every 6 months for the 5 years following the study completion (possibly all life long).

MRI assessments will be performed monthly for the first 6 months and then every 3 months for 5 years following the study completion.

Enrollment: 24 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. SPMS with progressive accumulation of disability after initial relapsing course, with or without disease activity (Lublin et al. 2014).
2. EDSS ≥ 6.5 and ≤ 8
3. EDSS progression over the 2 years prior to study start of ≥ 1.0 point for patients with EDSS =6.5 at the time of inclusion , and of ≥ 0.5 points for patients with EDSS > 6.5 at the time of inclusion
4. Age ≥ 18 and ≤ 60 years
5. Failure of best medical treatment as judged by the treating neurologist and declared absence of therapeutic alternatives

Exclusion criteria

1. Neurological conditions other than MS.
2. Psychiatric disorders, severe cognitive decline and personality and relational disorders.
3. History or known presence of significant systemic, infectious, oncologic or metabolic disorders.
4. Presence of any other autoimmune disease.
5. Chronic infections (HBV, HCV, HIV, tuberculosis).
6. Inability to perform MRI scans.
7. Immunomodulant/immunosuppressive treatments in the last 6 months before inclusion.
8. Current participation to other experimental studies.
9. Inability to provide informed consent.
10. Any contra-indication to lumbar puncture and the surgical procedure (e.g. use of anticoagulants)
11. Pregnancy and breast feeding.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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24 participants in 1 patient group

Human Neural Stem Cells Suspension

Experimental group

Description:

Intraventricular injections of Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10,16 or 24 millions)

Treatment:

Biological: Human Neural Stem Cells