Using stem cells to treat juvenile idiopathic arthritis

A Randomized, Double-Blind, Phase 2, Efficacy and Safety Cross-Over Study of Allogeneic HB-adMSCs vs Placebo for the Treatment of Oligoarticular and Polyarticular Juvenile Idiopathic Arthritis

This study is testing if giving stem cells to children with juvenile idiopathic arthritis can help reduce their symptoms better than a placebo.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 66 (estimated)
• Ages: 2 Years to 16 Years
• Sex: All
• Sponsor: Hope Biosciences (Industry-sponsored)
• Locations: 1 site (Sugar Land, Texas)
• Trial ID: NCT06623240 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of intravenous infusions of allogeneic Hope Biosciences adipose-derived mesenchymal stem cells (HB-adMSCs) compared to a placebo in children with juvenile idiopathic arthritis (JIA). It is a randomized, double-blind, AB/BA cross-over study with a washout period of 12 weeks and a treatment duration of 8 weeks per group. The primary objectives include assessing the incidence of adverse events and measuring improvements in ACR Pedi scores among participants. A total of 66 subjects will be enrolled, focusing on those with oligoarticular or polyarticular forms of JIA.

Who should consider this trial

Good fit: Ideal candidates are children aged 2 to 16 years diagnosed with oligoarticular or polyarticular juvenile idiopathic arthritis.

Not a fit: Patients currently receiving effective treatment for JIA or those who have not failed at least two approved medications may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the symptoms and quality of life for children suffering from juvenile idiopathic arthritis.

How similar studies have performed: While the use of stem cells in treating autoimmune conditions is a growing field, this specific approach is novel and has not been extensively tested in similar studies.

Eligibility criteria

Inclusion Criteria:

The subject will be eligible for inclusion in this clinical trial only if all of the following criteria apply:

1. Male and female subjects who are ≥ 2 years old and \< 17 years old.

   a.The first 6 subjects enrolled must be ≥ 12 years old and \< 17 years old
2. Must be diagnosed with Oligoarticular or Polyarticular Juvenile Idiopathic Arthritis by a Pediatric Rheumatologist.
3. Must have rheumatoid factor (RF) factor test result documented in medical records.
4. Must have at least 3 affected joints at the screening visit.
5. Must have a body weight of \> 10 kg at the screening visit.
6. Subjects without a current established treatment for JIA who are not on treatment because they have failed at least 2 approved medications for their condition, or if being treated, subjects who are on a stable dose of arthritis therapy regimen for ≥3 months prior to screening.
7. Must have an abnormal CRP result and/or abnormal ESR result at screening. Abnormal C-reactive protein (CRP) value defined as \> 1 mg/dL. Abnormal Erythrocyte Sedimentation Rate (ESR) value defined as \>15 mm/hr for males and \>20 mm/hr for females.
8. Female study subjects of childbearing potential should not be pregnant or plan to become pregnant during study participation and for 6 months after the last investigational product administration. Female study subjects of childbearing potential must confirm usage of one of the following contraceptive measures:

   * Hormonal contraceptives associated with ovulation inhibition (oral, injectable, implantable, patch, or intravaginal).
   * Intrauterine device (IUD), or intrauterine hormone-releasing system (IUS).
   * Barrier contraceptive methods (condoms, diaphragm, etc.).
9. Male subjects if their sexual partners can become pregnant should ensure the use one of the following methods of contraception during study participation and for 6 months after the last administration of the investigated product.

   * Hormonal contraceptives associated with ovulation inhibition (oral, injectable, implantable, patch, or intravaginal).
   * Intrauterine device (IUD), or intrauterine hormone-releasing system (IUS).
   * Barrier contraceptive methods (condoms, diaphragm, etc.).
10. Study subject's parent(s)/LAR is/are able and willing to comply with the requirements of this clinical trial.
11. Voluntarily signed informed consent from study subjects' parent(s) or legally authorized representative obtained before any clinical-trial related procedures are performed.

Exclusion Criteria:

The subject will not be eligible for inclusion in this clinical trial if any of the following criteria apply:

1. Study subject has any of the following laboratory results at the screening visit:

   1. WBC: \<3000 cells/μL OR \>15000 cells/μL (\<3 K cells/μL or \>15 K cells/μL)
   2. Hemoglobin: \<8 g/dL
   3. Absolute Neutrophil Count: \<1500 cells/μL
   4. Platelet: \<150000 cells/μL (\<150 K cells/μL)
   5. Sodium: \<120 mEq/L OR \>150 mEq/L
   6. Glucose: \>150 mg/dL
   7. Potassium: \<3.5 mEq/L OR \>6 mEq/L
   8. BUN: \>25 mg/dL
   9. Creatinine: \>2 mg/dL
   10. BUN/Creatinine ratio: \>50
   11. AST: \>100 U/L
   12. ALT: \>100 U/L
2. Study participant has any vital sign abnormalities at the screening visit as determined by the investigator.
3. Study subject has 1 or more significant uncontrolled concurrent medical conditions (verified by medical records), including the following:

   1. Diabetes Mellitus
   2. Crohn's Disease
   3. Lupus
   4. Multiple Sclerosis
4. Study subject has any active malignancy, including evidence of cutaneous basal, squamous cell carcinoma or melanoma.
5. Study subject has known alcoholic addiction or dependency or has current substance use or abuse.
6. Study subject has received any stem cell treatment within 1 year before first dose of investigational product other than stem cells produced by Hope Biosciences LLC.
7. Receiving any investigational therapy or any approved therapy for investigational use within 1 year prior first dose of the investigational product other than COVID-19 vaccines.
8. Study subject has any other laboratory abnormality or medical condition which, in the opinion of the investigator, poses a safety risk or will prevent the subject from completing the study.
9. Study subject's parent(s)/LAR unable to understand and provide signed informed consent.
10. Study subject and/or study subject's parent(s)/LAR unlikely to complete the study or adhere to the study procedures.
11. Study subject with known concurrent acute or chronic viral hepatis B or C or human immunodeficiency virus (HIV) infection.
12. Study subject with any systemic infection requiring treatment with antibiotics, antivirals, or antifungals within 30 days prior to first dose of the investigational product.

Where this trial is running

Sugar Land, Texas

• Hope Biosciences Research Foundation — Sugar Land, Texas, United States (Recruiting)

Study contacts

• Principal investigator: Thanh Cheng, MD — Hope Biosciences Research Foundation
• Study coordinator: David Gonzalez

Conditions Juvenile Idiopathic Arthritis; JIA; juvenile idiopathic arthritis; juvenile arthritis; oligoarticular; polyarticular; polyarticular juvenile idiopathic arthritis; oligoarticular juvenile idiopathic arthritis