NCT03799718已完成2 期
A Phase 2 Open-label Multicenter Study to Evaluate the Safety and Efficacy of Repeated Administration of NurOwn® [Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors (NTF), MSC-NTF] Cells in Participants With Progressive MS
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 8
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events
研究概览
简要总结
A multidose open-label study with autologous Mesenchymal Stromal Stem Cells Secreting Neurotrophic Factors (MSC-NTF cells) involving 20 participants with progressive MS at multiple investigational study sites.
详细描述
An open-label study with a single treatment arm involving 20 participants with progressive MS at multiple investigational study sites. After providing informed consent, participants meeting the inclusion and exclusion criteria will be randomized and approximately 4 weeks later will undergo a bone-marrow aspiration (BMA). Each participants will receive three Intrathecal cell transplantations within 16 weeks and will be followed for 12 weeks for safety and efficacy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females ages 18 to 65 years old, inclusive, at the Screening Visit.
- •Clinical diagnosis of Progressive MS (Primary and Secondary) based on the 2017 revised MacDonald Criteria and confirmation by the Investigator that the disease has entered the progressive stage for at least 6 months prior to enrollment.
- •No evidence of clinical MS relapse or high dose pulse corticosteroid treatment within 6 months prior to screening
- •Disability status at screening with an Expanded Disability Status Scale (EDSS) 3.0-6.5, inclusive.
- •Able to walk 25 feet in 60 seconds or less.
- •Stable dose of non-excluded MS Disease Modifying Therapy for at least 6 months prior to Screening Visit (Visit 1).
- •Women of childbearing potential shall either be surgically sterile, or must agree not to become pregnant for the duration of the study. Women must be willing to undergo a serum pregnancy test at screening, and at the conclusion of the study. Participants of childbearing potential must agree to use a medically approved form of birth control (abstinence, intrauterine device (IUD), oral contraception, barrier and spermicide or hormonal implant) throughout the duration of the study and for at least 3 months following the last transplantation. For those women who are sexually active and using oral contraceptives, a second form of barrier contraception is required. Men must be willing to consistently use two forms of contraceptive if their partners are of childbearing age.
- •Capable of providing informed consent and willing and able to follow study procedures, including willingness to undergo multiple/repeated lumbar puncture.
排除标准
- •Prior stem cell therapy of any kind.
- •Active participation in any other MS interventional study or use of unapproved MS investigational therapy within 90 days prior to the Screening Visit (Visit 1).
- •Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate study procedures for any other reason.
- •History of clinically significant autoimmune disease (excluding thyroid disease) that may confound study results, in the opinion of the Investigator and the medical monitor, myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
- •Any unstable clinically significant medical condition other than multiple sclerosis (e.g., within six months of Screening Visit (Visit 1), had myocardial infarction, angina pectoris, and/or congestive heart failure), treatment with anticoagulants that, in the opinion of the investigator, would compromise the safety of participants.
- •Any history of malignancy within the previous 5 years, except for non-melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or reoccurrence within three years of Screening Visit (Visit 1)).
- •Serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper normal limit.
- •Serum creatinine value >2.0 times the upper normal limit.
- •Positive test for Hepatitis B (HBV; surface antigen (HBsAg) and antibodies to core antigen (IgG and IgM anti-HBc)), Hepatitis C (HCV), or human immunodeficiency virus (HIV) 1 and
- •Current use of immunosuppressant medication or use of such medication within 6 months of study enrollment (aside from Rituximab or other approved B-cell immunotherapy). Alemtuzumab (Lemtrada), Cladribine (NDA submitted), Natalizumab (Tysabri), S1P modulators (Gilenya) are excluded for safety reasons due to the known risk of systemic autoimmune disease, malignancy, opportunistic infections, and cardiovascular toxicity associated with these therapies, as well as theoretical effects on MSC-NTF cell homing and migration, that may be associated with Natalizumab and/or S1P modulators (Gilenya).
- •Any history of acquired or inherited immune deficiency syndrome.
- •Any history of either substance abuse within the past year, or unstable psychiatric disease according to the Investigator's judgment.
- •Pregnant women or women currently breastfeeding.
- •Subjects for whom MRI is contraindicated (i.e., have a pacemaker or other metallic implanted device, or are unable to remain in the machine for period of time needed to acquire a scan.
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events
时间窗: Up to 28 weeks post-first treatment
Combined safety of all 3 intrathecal doses of NurOwn® (MSC-NTF cells) Number of participants who experienced Treatment-emergent Adverse Events during the study. Treatment-emergent Adverse Event is an adverse event that occurs for the first time after initiation of first treatment or if it had occurred prior to initiation first treatment, it worsens in severity after initiation of first treatment.
次要结局
- Number of Participants With 25% or Greater Improvement From Baseline in Time 25 Foot Walk (T25FW) Speed or Nine-Hole Peg Test (9-HPT)(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Number of Participants With 25% or Greater Improvement From Baseline to Week 28 in Timed 25 Foot Walk (T25FW) Speed(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Number of Participants With ≥10 Points Improvement From Baseline to Week 28 in Multiple Sclerosis Walking Scale (MSWS-12)(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Change in Concentration of Hepatocyte Growth Factor (HGF) Neuroprotective Biomarker From Baseline in the Cerebrospinal Fluid (CSF) 16 Weeks Following NurOwn® Treatment(From Baseline (pre-first treatment) to 16 weeks post first treatment)
- Change in Concentration of Vascular Endothelial Growth Factor (VEGF) Neuroprotective Biomarker From Baseline in the Cerebrospinal Fluid (CSF) 16 Weeks Following First NurOwn® Treatment(From Baseline (pre-first treatment) to 16 weeks post first treatment)
- Number of Participants With 25% or Greater Improvement From Baseline to Week 28 in 9-HPT(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Number of Participants With ≥8 Letter Improvement From Baseline to Week 28 in LCLA Binocular 2.5% Contrast Level(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Number of Participants Who Had >5.5 in Expanded Disability Status Scale (EDSS) at Baseline, With ≥0.5 Points Improvement From Baseline to Week 28(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
- Number of Participants With ≥ 3 Points Improvement From Baseline to Week 28 in Symbol Digit Modalities Test (SDMT) Score(From Baseline (pre-first treatment) to 28 weeks post-first treatment)
研究者
研究点 (8)
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相关资讯
BrainStorm Advances NurOwn Toward Phase 3b ENDURANCE Trial in ALS Under First-Ever SPA for an ALS Therapeutic- BrainStorm Cell Therapeutics is completing preparations to launch the Phase 3b ENDURANCE trial of NurOwn (MSC-NTF) in ALS, conducted under an FDA Special Protocol Assessment.
- The ENDURANCE trial plans to enroll about 200 ALS patients across a randomized, double-blind, placebo-controlled Part A and an open-label extension Part B, with ALSFRS-R change as the primary endpoint.
- BrainStorm appointed former FDA Associate Commissioner Peter J. Pitts as Executive Chairman to lead regulatory, operational, partnership, and capital-markets initiatives for the NurOwn program.
- The company reported cash of approximately $0.2 million as of June 30, 2026, and a net loss of approximately $3.9 million for the second quarter of 2026.last monthBrainStorm Advances NurOwn ALS Therapy with FDA-Aligned Phase 3b Trial Submission- BrainStorm Cell Therapeutics has submitted an IND amendment to the FDA for NurOwn, its autologous mesenchymal stem cell therapy for ALS, setting the stage for a pivotal Phase 3b clinical trial.
- The trial design received FDA validation through a Special Protocol Assessment agreement, confirming its potential to support a future Biologics License Application if successful.
- The Phase 3b study will enroll approximately 200 ALS patients in a two-part design, with the primary endpoint measuring changes in ALSFRS-R scores from baseline to week 24.last yearBrainStorm Announces First Patient in Phase 2 Clinical Trial for Progressive MSBrainStorm Cell Therapeutics Inc. has enrolled the first patient in its Phase 2 clinical trial for progressive Multiple Sclerosis (MS), utilizing its NurOwn® technology platform. This trial represents a significant step towards addressing the unmet medical needs in progressive MS, with top-line data expected by mid-2020.7 years ago