Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients with ALS

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 13-009171
    NCT ID: NCT02017912
    Sponsor Protocol Number: BCT-001-US

About this study

This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn™) in patients with ALS .

MSC-NTF cells are a novel cell-therapeutic approach which is expected to effectively deliver Neurotrophic factors, which are potent survival factors for neurons, directly to the site of damage.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  1. Males and females ages 18 to 75 years old, inclusive.
  2. ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
  3. Disease symptom onset ≤ 24 months, as determined by the first reported occurrence of symptomatic weakness of a limb or of spasticity, and ALSFRS-R ≥30 at the Screening Visit.
  4. Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and age at the Screening Visit.
  5. Subjects must be taking a stable dose of Riluzole for at least 30 days prior to enrollment or not be on Riluzole, and not have been on it for at least 30 days prior to enrollment (Riluzole-naïve subjects are permitted in the study).
  6. Capable of providing informed consent and willing and able to follow study procedures, including willingness to undergo lumbar puncture.
  7. Geographic accessibility to the study site and willingness and ability to comply with follow-up.
  8. Women of child-bearing potential must agree not to become pregnant for the duration of the study. Women must be willing to consistently use two forms of contraceptive therapy throughout the course of the trial, and undergo a pregnancy test one week before bone marrow aspiration. Men must be willing to consistently use two forms of contraceptive if their partners are of child-bearing age.
  9. Citizen or permanent resident of the United States.
  10. History of a chronic onset of a progressive motor weakness of greater than one year, but less than two years duration

Exclusion Criteria:

  1. Prior stem cell therapy of any kind.
  2. 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.
  3. History of autoimmune disease, myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
  4. Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, 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 patients.
  5. Any history of malignancy including any malignancy affecting the central nervous system and melanoma, within the previous 5 years, with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
  6. Serum AST or ALT value >3.0 times the upper normal limit.
  7. Serum creatinine value >2.0 times the upper normal limit.
  8. Positive test for Hepatitis B, Hepatitis C, HIV.
  9. Current use of immunosuppressant medication or use of such medication within 4 weeks of Screening visit (Visit 1).
  10. Any history of acquired or inherited immune deficiency syndrome.
  11. Exposure to any other experimental agent (off-label use or investigational) or participation in a clinical trial within 30 days prior to Screening Visit (Visit 1).
  12. Use of non-invasive ventilation (NIV) or invasive ventilation (tracheostomy).
  13. Any history of either substance abuse within the past year, or unstable psychiatric disease according to PI judgment.
  14. Placement or usage of feeding tube
  15. Pregnant women or women currently breastfeeding

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Anthony Windebank, M.D.

Closed for enrollment

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CLS-20119562

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