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

NCT06623240 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: HBJIA01
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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

Conditions

Juvenile Idiopathic Arthritis (JIA)

Keywords

JIA; juvenile idiopathic arthritis; juvenile arthritis; oligoarticular; polyarticular; polyarticular juvenile idiopathic arthritis; oligoarticular juvenile idiopathic arthritis; arthritis

Outcome Measures

Primary Outcomes (36)

• Incidence of treatment-emergent adverse events (TEAEs).

  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.

  Baseline (Week 0) up to Week 72

• Incidence of serious adverse events (SAEs).

  Incidence of serious Adverse Events (SAEs)

  Baseline (Week 0) up to Week 72

• Incidence and risk of AEs of particular interest (serious or nonserious)

  Incidence and risk of AEs of particular interest (serious or nonserious), including thromboembolic events, infections, and hypersensitivities.

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (x10^3 Cells/uL)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (x10\^3 Cells/uL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (% of WBC)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (% of WBC)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (pg)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (pg)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (g/dL)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (g/dL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (fL)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (fL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (x10^6 Cells/uL)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (x10\^6 Cells/uL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (% Difference in Volume and Size of RBC)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (% Difference in Volume and Size of RBC)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Complete Blood Count (% of Total Blood Cell Count)

  Clinically significant changes from Baseline in laboratory values results - Complete Blood Count (% of Total Blood Cell Count)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (g/dL)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (g/dL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (Ratio of Albumin to Calc. Globulin)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (Ratio of Albumin to Calc. Globulin)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (U/L)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (U/L)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mg/dL)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mg/dL)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mEq/L)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mEq/L)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mL/Min/1.73m^2)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (mL/Min/1.73m\^2)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (Ratio of Calc BUN/Creatinine)

  Clinically significant changes from Baseline in laboratory values results - Comprehensive Metabolic Panel (Ratio of Calc BUN/Creatinine)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Coagulation Panel (Seconds)

  Clinically significant changes from Baseline in laboratory values results - Coagulation Panel (Seconds)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in laboratory values results - Coagulation Panel (Ratio of Prothrombin Time/Mean Prothrombin Time)

  Clinically significant changes from Baseline in laboratory values results - Coagulation Panel (Ratio of Prothrombin Time/Mean Prothrombin Time)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - Respiratory Rate (Breaths per minute)

  Clinically significant changes from baseline in Respiratory Rate (Breaths per minute)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - Heart Rate (Breaths per minute)

  Clinically significant changes from baseline in Heart Rate (Breaths per minute)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - Body Temperature (Celsius)

  Clinically significant changes from baseline in Body Temperature (Celsius)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - Systolic Blood Pressure (mmHg)

  Clinically significant changes from baseline in Systolic Blood Pressure (mmHg)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - Diastolic Blood Pressure (mmHg)

  Clinically significant changes from baseline in Diastolic Blood Pressure (mmHg)

  Baseline (Week 0) up to Week 72

• Changes from Baseline in Vital Signs - SPO2 (%)

  Clinically significant changes from baseline in SPO2 (%)

  Baseline (Week 0) up to Week 72

• Clinically significant changes in weight results (in kg)

  Changes from Baseline in weight results in patients (in kg)

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Abdomen

  Number of participants with abnormal physical examination results - Abdomen

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Cardiovascular

  Number of participants with abnormal physical examination results - Cardiovascular

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Head, Eyes, Ears, Nose, and Throat

  Number of participants with abnormal physical examination results - Head, Eyes, Ears, Nose, and Throat

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Lymph Node

  Number of participants with abnormal physical examination results - Lymph Node

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Musculoskeletal

  Number of participants with abnormal physical examination results - Musculoskeletal

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Neurological

  Number of participants with abnormal physical examination results - Neurological

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Respiratory

  Number of participants with abnormal physical examination results - Respiratory

  Baseline (Week 0) up to Week 72

• Number of participants with abnormal physical examination results - Skin

  Number of participants with abnormal physical examination results - Skin

  Baseline (Week 0) up to Week 72

• Clinically significant changes in ACR Pedi 30

  Changes from Baseline in ACR Pedi 30 in patients

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

Secondary Outcomes (5)

• Clinically significant changes in ACR Pedi 50

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

• Clinically significant changes in ACR Pedi 70

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

• Clinically significant changes in C-reactive protein values (mg/L)

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

• Clinically significant changes in erythrocyte sedimentation rate values (mm/h)

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

• Clinically significant changes in PedsQL

  Baseline (Week 0) up to Week 52 for Group AB or Week 72 for Group BA

Study Arms (3)

Cohort 2: Group AB

EXPERIMENTAL

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.

Biological: allogeneic Hope Biosciences adipose-derived mesenchymal stem cells; Other: Normal Saline Solution 0.9%

Cohort 2: Group BA

EXPERIMENTAL

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.

Biological: allogeneic Hope Biosciences adipose-derived mesenchymal stem cells; Other: Normal Saline Solution 0.9%

Cohort 1: Group A

EXPERIMENTAL

Cohort 1 - Group A will receive allogeneic HB-adMSCs (Hope Biosciences adipose-derived mesenchymal stem cells) at study weeks 0, 4, and 8.

Biological: allogeneic Hope Biosciences adipose-derived mesenchymal stem cells

Interventions

allogeneic Hope Biosciences adipose-derived mesenchymal stem cells BIOLOGICAL

Product: Allogeneic HB-adMSCs (Hope Biosciences adipose derived mesenchymal stem cells) Dose determined by body weight: * 50 million cells in 50mL saline: ≥ 10 kg to \< 22 kg * 100 million cells in 100mL saline: ≥ 22 kg to \< 45 kg * 200 million cells in 250mL saline: ≥ 45 kg Route: Intravenous Regimen: Weeks 0, 4, and 8 (Cohort 1: Group A and Cohort 2: Group AB) or Weeks 20, 24, and 28 (Cohort 2: Group BA)

Cohort 1: Group A; Cohort 2: Group AB; Cohort 2: Group BA

Normal Saline Solution 0.9% OTHER

Product: Normal Saline Solution 0.9% Route: Intravenous Regimen: Weeks 0, 4, and 8 (Cohort 2: Group BA) or Weeks 20, 24, and 28 (Cohort 2: Group AB)

Cohort 2: Group AB; Cohort 2: Group BA

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• Must be diagnosed with Oligoarticular or Polyarticular Juvenile Idiopathic Arthritis by a Pediatric Rheumatologist.
• Must have rheumatoid factor (RF) factor test result documented in medical records.
• Must have at least 3 affected joints at the screening visit.
• Must have a body weight of \> 10 kg at the screening visit.
• 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.
• 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.
• 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.).
• 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).

You may not qualify if:

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

Sponsors & Collaborators

• Hope Biosciences Research Foundation: lead

Study Sites (1)

Hope Biosciences Research Foundation

Sugar Land, Texas, 77478, United States

Location

MeSH Terms

Conditions

Arthritis, Juvenile; Arthritis

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Thanh Cheng, MD

  Hope Biosciences Research Foundation

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This clinical trial is a double-blinded study, meaning that the subjects and the researchers both do not know the group assignments. Research staff and subjects may be unblinded once all study data has been collected, all study-related procedures and follow-ups are completed, and all monitoring has been completed. (The first 6 patients (Cohort 1) will only receive active treatment; therefore, blinding is not applicable).
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This study is 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. Cohort 1 (first 6 patients) will undergo a screening period (up to 35 days) and an 8-week active treatment period, followed by a 64-week follow-up. Cohort 2 (remaining 60 patients) will undergo a screening period (up to 35 days), an 8-week active treatment period and an 8-week inactive treatment period (placebo) with a washout period of 12 weeks between the treatment periods. Subjects assigned to Cohort 2 - Group AB will receive allogeneic HB-adMSCs before the washout period, while subjects assigned to Cohort 2 - Group BA will receive allogeneic HB-adMSCs after the washout period. Group AB will undergo a safety follow-up of 64 weeks after their last infusion of HB-adMSCs, while Group BA will undergo a safety follow-up of 44 weeks after their last infusion of HB-adMSCs.

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 2, 2024
• Study Start: January 1, 2026
• Primary Completion: January 1, 2026
• Study Completion: January 1, 2026
• Last Updated: January 7, 2026

Record last verified: 2026-01

Locations

US (1)