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NCT Number: NCT02691247

Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)

This clinical trial will explore the safety and effect of autologous ex vivo expanded polyclonal regulatory T-cells on beta cell function in patients, aged 8 to 17, with recent onset T1DM. Other measures of diabetes severity and the autoimmune response underlying T1DM will also be explored. Eligible subjects will receive a single infusion of CLBS03 (high or low dose) or placebo.

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Key information

Conditions

Diabetes Mellitus Autoimmune Diseases Diabetes Mellitus, Type 1 Endocrine System Diseases Glucose Metabolism Disorders Immune System Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Age range

8 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Rady Children's Hospital, San Diego, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male and females aged 8 to 17 years of age
  • Diagnosis of T1DM within 100 days of receipt of study drug
  • Positive for at least one islet cell autoantibody
  • Peak MMTT-stimulated C-peptide level > 0.2 pmol/mL (at the screening visit)
  • Weight of ≥30 kg
  • Must agree to use a reliable and acceptable method of contraception for the duration of participation
  • Willing and medically acceptable to postpone live vaccine immunizations for one year after infusion
  • Written informed consent and written assent

Exclusion criteria

  • Hemoglobin less than the lower limit of normal
  • Leukocytes <3,000/μL; neutrophils <1,500/μL; lymphocytes <800/μL; platelets <100,000/μL
  • Regulatory T-cells present in peripheral blood at <20 cells per μL
  • Current or ongoing use of non-insulin pharmaceuticals (that may affect glycemic control)
  • Current or anticipated use of systemic corticosteroids or other immunomodulatory drugs
  • Recent serious bacterial, viral, fungal, or other opportunistic infections
  • History of malignancy or serious uncontrolled cardiovascular, nervous system, pulmonary, renal, or gastrointestinal disease
  • Serologic evidence of current or past viral infection: human immunodeficiency virus (HIV), Hepatitis B, Hepatitis C, and human T-lymphotropic virus (HTLV) 1/2
  • Positive QuantiFERON® tuberculosis (TB) test, purified protein derivative (PPD) skin test, history of tuberculosis, or active TB infection
  • Active infection with Epstein-Barr Virus or Cytomegalovirus
  • Liver disease
  • Pregnant or breast-feeding
  • Vaccination with a live virus within 8 weeks of receipt of study drug
  • Vaccination with a killed virus within 2 weeks of receipt of study drug
  • Participation in an investigational drug study within 90 days prior to screening
  • Previously treated with a T-Reg based cell therapy
  • History of allergy to gentamicin

Treatment and study plan

CLBS03 Low Dose

Biological

CLBS03 High Dose

Biological

Placebo

Biological

Primary outcomes

  1. Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52

    Time frame: Week 52

    The area-under-curve of sequential C-peptide concentrations (AUC-Cpep) during the mixed-meal tolerance test (MMTT) is the gold-standard method to assess residual beta-cell (ie, insulin) secretion in type 1 diabetes.

Secondary outcomes

  1. Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104

    Time frame: Week 104

    The area-under-curve of sequential C-peptide concentrations (AUC-Cpep) during the mixed-meal tolerance test (MMTT) is the gold-standard method to assess residual beta-cell (ie, insulin) secretion in type 1 diabetes.

  2. Change in Hemoglobin A1c (HbA1c)

    Time frame: Week 104

  3. Change From Baseline in Mean Daily Dose of Insulin

    Time frame: Week 104

Sponsors and collaborators

Lead sponsor

Lisata Therapeutics, Inc.

Industry

Collaborators

  • California Institute for Regenerative Medicine (CIRM)
  • Sanford Health

Registry information

Official study title

A Prospective Randomized Placebo-Controlled Double Blind Clinical Trial to Evaluate the Safety and Efficacy of CLBS03 (Autologous Ex Vivo Expanded Polyclonal Regulatory T-cells) in Adolescents With Recent Onset Type 1 Diabetes Mellitus (T1DM)

Important dates

Study start
2016
Primary completion
2019
Study completion
2020
First posted
Feb 25, 2016
Registry last updated
Jan 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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