An open-label study to assess safety and tolerability of NurOwn® in participants with early stage of Alzheimer's Disease

Phase 1

• Conditions: Alzheimer's Disease; MedDRA version: 20.0Level: HLTClassification code 10001897Term: Alzheimer's disease (incl subtypes)System Organ Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2020-002872-11-NL
• Lead Sponsor: Brainstorm Cell Therapeutics, Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-11-13
• Last Update Posted: 23 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1.Males and females, ages 50 to 75 years old, inclusive, at the Screening Visit (Visit 1).
2.Clinical diagnosis of prodromal to mild AD at least 6 months prior to enrollment, based on the International Working Group (IWG-2) diagnostic criteria or the National Institute on Aging-Alzheimer's Association (NIA-AA) diagnostic criteria.
3.Mini Mental State Examination (MMSE) of 20-30, inclusive, and Clinical dementia rating-global score (CDR-GS) of 0.5 or 1.0
4.Demonstrated abnormal memory function
5.CSF profile consistent with AD: Amyloid Beta 42 (Aß42) concentration of <1000 pg/ml AND P-tau >19 pg/ml or ratio of p-tau/Abeta > 0.024 (Elecsys assay)
6.If currently treated with Cholinesterase Inhibitors (AChEI) (donepezil, galantamine, or rivastigmine) or Memantine the dose should be stable for at least 12 weeks prior to Screening (Visit 1).
7.Must have a caregiver that is willing to participate in the study and is able to provide accurate information on the participant's cognitive and functional ability.
8.Must consent to apolipoprotein E (ApoE) genotyping or willing to provide previous test results.
9.If sexually active and of childbearing potential, both females and males must agree to use an effective birth control method during the study and for at least 3 months following the last transplantation, such as: abstinence, intrauterine device (IUD), oral contraception, barrier and spermicide or hormonal implant.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1.Prior stem cell therapy of any kind.
2.Active participation in any other interventional study or use of unapproved AD investigational therapy within 60 days prior to the Screening Visit (Visit 1), unless proven to have been on placebo.
3.Inability to lie flat for the duration of intrathecal cell administration and/or bone marrow aspiration, or inability to tolerate study procedures for any other reason.
4.History of clinically significant autoimmune disease (excluding thyroid disease) that may confound study results, myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
5.Any poorly controlled clinically significant medical condition other than Alzheimer’s disease (e.g., within six months of Screening Visit (Visit 1), such as myocardial infarction, angina pectoris, congestive heart failure, or hypertension (repeated blood pressure >180 mmHg systolic or 100 mmHg diastolic) as well as clinically significant coagulopathy, treatment with anticoagulants that, in the opinion of the investigator, would compromise the safety of participants.
6.Any history of malignancy (e.g., myeloproliferative disorder, leukemia or lymphoma) within the previous 5 years, except for non-melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of Screening Visit (Visit 1)).
7.Any history of acquired or inherited immune deficiency syndrome.
8.Platelet count, INR, PT or PTT not within the normal range (local protocol) or other risk for increased or uncontrolled bleeding (safety for lumbar puncture and bone marrow).
9.Presence of contraindication to lumbar puncture as judged by local PI, e.g. need for anticoagulant or antiplatelet medications other than aspirin at a dose of = 100 mg/day or clopidogrel.
10.MRI evidence of a) more than three lacunar infarcts, b) territorial infarct or macroscopic hemorrhage, or c) deep white matter lesions (corresponding to a Fazekas score of 3).
11.Pregnant women or women currently breastfeeding.
12.Positive test result for Hepatitis B virus (HBV; surface antigen (HBsAg) and antibodies to core antigen (IgG and IgM anti-HBc)), Hepatitis C virus (HCV), Human Immune deficiency Virus (HIV) 1 and 2.
13.Any medical or neurological/neurodegenerative condition other than AD (e.g.,vascular dementia, dementia with Lewy bodies, and Parkinson’s disease) that, in the opinion of the Investigator, might be a contributing cause to the participant's cognitive impairment or could lead to discontinuation, lack of compliance, interference with study assessments, or safety concerns (includes, low vitamin B12 level, low Hb level (<100); hypothyroid disease).
14.History within the past 6 months or evidence of clinically significant psychiatric illness (e.g., major depression, suicidality, schizophrenia, or bipolar affective disorder).
15.Any condition, which in the opinion of the investigator or the Sponsor makes the patient unsuitable for inclusion.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified