HB-adMSCs vs Placebo for the Treatment of Juvenile Idiopathic Arthritis

Phase 2

Not yet recruiting

• Conditions: Juvenile Idiopathic Arthritis (JIA)
• Interventions: Biological: allogeneic Hope Biosciences adipose-derived mesenchymal stem cells; Other: Normal Saline Solution 0.9%

• Registration Number: NCT06623240
• Lead Sponsor: Hope Biosciences

Brief Summary

Methodology: Randomized, double-blind, AB/BA cross-over study with a washout period of 12 weeks.

Treatment Duration: 8 weeks per group

General Objectives: To assess the efficacy and safety of multiple intravenous infusions of allogeneic HB-adMSCs by improving signs and symptoms of juvenile idiopathic arthritis in this subject population.

Number of Subjects: 66 (6 subjects in Cohort 1 and 60 subjects in Cohort 2)

Indication: Juvenile Idiopathic Arthritis

Primary Objectives:

* To assess the safety of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by the incidence of adverse events or serious adverse events. (Time Frame: Week 0 to Week 72).

* To investigate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in ACR Pedi 30 scores. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

Secondary Objectives

* To evaluate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in ACR Pedi 50 scores. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

* To evaluate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in ACR Pedi 70 scores. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

* To evaluate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in CRP values. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

* To evaluate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in ESR values. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

* To evaluate the efficacy of intravenous infusions of allogeneic HB-adMSCs vs placebo in patients with oligoarticular or polyarticular juvenile idiopathic arthritis as determined by improvements in Peds QL scores. (Time Frame: Week 0 to Week 52 for Group AB and Week 0 to Week 72 for Group BA).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-30
• Study First Submitted QC: 2024-10-01
• Study First Posted: 2024-10-02
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-22
• Last Update Posted: 2024-11-26
• Study Start: 2025-01
• Primary Completion: 2027-12
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 66

Inclusion Criteria

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.

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 &gt; 10 kg at the screening visit.

6. Subjects without a current established treatment for JIA, 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 &gt; 1 mg/dL. Abnormal Erythrocyte Sedimentation Rate (ESR) value defined as &gt;15 mm/hr for males and &gt;20 mm/hr for females.

8. Female study subjects should not be pregnant or plan to become pregnant during study participation and for 6 months after last investigational product administration.

9. Male subjects if their sexual partners can become pregnant should use a method of contraception during study participation and for 6 months after the last administration of the investigated product.

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.

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Exclusion Criteria

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

a. WBC: &lt;3000 cells/μL OR &gt;15000 cells/μL (&lt;3 K cells/μL or &gt;15 K cells/μL) b. Hemoglobin: &lt;8 g/dL c. Absolute Neutrophil Count: &lt;1500 cells/μL d. Platelet: &lt;150000 cells/μL (&lt;150 K cells/μL) e. Sodium: &lt;120 mEq/L OR &gt;150 mEq/L f. Glucose: &gt;150 mg/dL g. Potassium: &lt;3.5 mEq/L OR &gt;6 mEq/L h. BUN: &gt;25 mg/dL i. Creatinine: &gt;2 mg/dL j. BUN/Creatinine ratio: &gt;50 k. AST: &gt;100 U/L l. ALT: &gt;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:

4. Diabetes Mellitus

5. Crohn's Disease

6. Lupus

7. 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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Cohort 2: Group AB	allogeneic Hope Biosciences adipose-derived mesenchymal stem cells	Cohort 2 - Group AB will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at study weeks 0, 4, and 8. Then, Group AB will receive placebo (Sterile Saline Solution 0.9%) at weeks 20, 24, and 28 after a washout period of 12 weeks between active treatment and placebo.
Cohort 2: Group AB	Normal Saline Solution 0.9%	Cohort 2 - Group AB will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at study weeks 0, 4, and 8. Then, Group AB will receive placebo (Sterile Saline Solution 0.9%) at weeks 20, 24, and 28 after a washout period of 12 weeks between active treatment and placebo.
Cohort 2: Group BA	allogeneic Hope Biosciences adipose-derived mesenchymal stem cells	Cohort 2 - Group BA will receive placebo (Sterile Saline Solution 0.9%) at study weeks 0, 4, and 8. Then, Group BA will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at weeks 20, 24, and 28 after a washout period of 12 weeks between placebo and active treatment.
Cohort 2: Group BA	Normal Saline Solution 0.9%	Cohort 2 - Group BA will receive placebo (Sterile Saline Solution 0.9%) at study weeks 0, 4, and 8. Then, Group BA will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at weeks 20, 24, and 28 after a washout period of 12 weeks between placebo and active treatment.
Cohort 1: Group A	allogeneic Hope Biosciences adipose-derived mesenchymal stem cells	Cohort 1 - Group A will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at study weeks 0, 4, and 8.

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).	Baseline (Week 0) up to Week 72	Treatment-emergent adverse events are defined as any adverse events which occur after the first infusion with HB-adMSCs up to the week 12 visit for Group AB or the week 32 visit for group BA.
Incidence and risk of AEs of particular interest (serious or nonserious), including thromboembolic events, infections, and hypersensitivities.	Baseline (Week 0) up to Week 72	Treatment-emergent adverse events are defined as any adverse events which occur after the first infusion with HB-adMSCs up to the week 12 visit for Group AB or the week 32 visit for group BA.
Clinically significant changes in laboratory values, vital signs, weight, and physical examination results.	Baseline (Week 0) up to Week 72	Changes from Baseline in laboratory values, vital signs, weight, and physical examination results in patients
Clinically significant changes in ACR Pedi 30	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in ACR Pedi 30 in patients

Secondary Outcome Measures

Name	Time	Method
Clinically significant changes in ACR Pedi 50	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in ACR Pedi 50 in patients
Clinically significant changes in ACR Pedi 70	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in ACR Pedi 70 in patients
Clinically significant changes in C-reactive protein values	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in C-reactive protein values in patients
Clinically significant changes in erythrocyte sedimentation rate values	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in erythrocyte sedimentation rate values in patients
Clinically significant changes in PedsQL	Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA	Changes from Baseline in PedsQL in patients

Trial Locations

Locations (1)

Hope Biosciences Research Foundation; 🇺🇸 Sugar Land, Texas, United States