Phase 1/2a Clinical Trial to Assess the Safety of HB-adMSCs for the Treatment of Rheumatoid Arthritis

Study Overview
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Brief Summary
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Hope Biosciences is conducting a research study of an investigational product called Hope Biosciences autologous adipose-derived mesenchymal stem cells (abbreviated as HB-adMSCs) as a possible treatment for Rheumatoid Arthritis (RA). The study purpose is to evaluate the safety profile of a single IV infusion of HB-adMSCs in subjects with clinical diagnosis of RA.

Detailed Description
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This is a Phase 1/2a, open-label, single-dose study in subjects with active Rheumatoid Arthritis (RA). 12-15 patients will be enrolled for the study. The overall objective of this study is to evaluate the safety profile of a single IV infusion of autologous adipose-derived mesenchymal stem cells (HB-adMSCs) in subjects with clinical diagnosis of RA. The primary endpoint of this study is to measure the number and frequency of adverse event(s) and/or severe adverse event(s) throughout the study duration. The second endpoint of this study is to evaluate the ability of HB-adMSCs to alter RA-related inflammation via measuring levels of Tumor Necrosis Factor alpha (TNF-a), Interleukin-6 (IL-6), C-Reactive Protein (CRP), Erythrocyte Sedimentation Rate (ESR) and Joint Count 66/68 after a single infusion of autologous HB-adMSCs for up to 12-month post-infusion.

Official Title
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A Phase 1/2a Clinical Trial to Assess Safety of a Single IV Infusion of Autologous Adipose-derived Mesenchymal Stem Cells in Adults With Active Rheumatoid Arthritis

Conditions
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Rheumatoid Arthritis

Intervention / Treatment
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* Biological: HB-adMSCs


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult male or female between the ages of 18 and 65 Patients have active RA as confirmed by the following criteria: ≥ 6 swollen joints and ≥ 6 tender joints at screening (68-joint count) Abnormal CRP result OR abnormal ESR result at screening. Abnormal CRP result at screening OR abnormal ESR defined as: CRP > 4.9 mg/L or ESR > 10mm/hr for men, > 20mm/hr for women Patients without current established treatment, or if being treated, patients who are on a stable dose of RA therapy regimen for ≥ 4 weeks prior to screening Exclusion Criteria: Inability to understand and provide signed informed consent Pregnancy, lactation, or, if female of childbearing potential, positive serum β-hCG at screening. Currently diagnosed any malignant neoplasm. Any patient who was successfully treated for cancer and has been disease-free, with no recurrence, for at least 5 years, will be considered. Uncontrolled systemic illness, including, but not limited to: hypertension (systolic >150 mm Hg or diastolic >95 mm Hg); diabetes; renal, hepatic, or cardiac failure or any laboratory abnormality that poses a safety risk to the subject such as: Hemoglobin ≤8.5 g/dL White blood cells (WBCs) ≤3,500/mm3 (3.5 G/L) Any other illness which, in the opinion of the investigator, characterizes the subject as not being a good candidate for the study Participation in another study with an investigational drug or device within 4 weeks prior to treatment or 5 half-lives of the investigational product used (whichever is longer). Positive results of hepatitis B surface antigen (HBsAg), hepatitis B surface antibody (HBsAb), hepatitis B core antibody (HBcAb), hepatitis C antibody (HCV Ab), and/or human immunodeficiency virus antibody (HIV Ab) tests at screening (excluding patients who are tested positive for HBsAb alone due to a hepatitis B vaccination). Positive history of Treponema pallidum.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: A single IV infusion of autologous adipose-derived mesenchymal stem cells (HB-adMSCs) in subjects with clinical diagnosis of RA.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment Arm<br>Single IV administration of autologous adipose-derived mesenchymal stem cells Baseline laboratory data will be collected prior to infusion; follow up data will be compared against baseline at 1, 3, 6 and 12 months. Joint Assessment 68 will be administered at 1, 3, 6 and 12 months. | Biological: HB-adMSCs<br>* Hope Biosciences autologous adipose-derived mesenchymal stem cells<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total Number of Adverse Events and Serious Adverse Events | Total number of Adverse Events and Serious Adverse Events across all subjects over 12 months. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Effect of Single Injection of HB-adMSCs on TNF-a in Patients With Acute RA | Efficacy is a secondary endpoint for this study and will be assessed by comparing the levels of Tumor Necrosis factor (TNF-a) (pg/mL) measured during trial. | 12 months | 
| The Effect of Single Injection of HB-adMSCs on IL-6 in Patients With Acute RA | Efficacy is a secondary endpoint for this study and will be assessed by comparing the levels of interleukin 6 (IL-6) (pg/mL) during the trial | 12 months | 
| The Effect of Single Injection of HB-adMSCs on CRP in Patients With Acute RA | Efficacy is a secondary endpoint for this study and will be assessed by comparing the levels of C-reactive protein (CRP) (mg/L) during the trial | 12 months | 
| The Effect of Single Injection of HB-adMSCs on ESR in Patients With Acute RA | Efficacy is a secondary endpoint for this study and will be assessed by comparing the levels of erythrosedimentation rate (ESR) (mm/hr) during the trial. | 12 months | 
| The Effect of Single Injection of HB-adMSCs on Joint Count in Patients With Acute RA. | Efficacy is a secondary endpoint for this study and will be assessed by comparing the levels of Joint Count 66/68 (# joints - tender and swollen) during the trial. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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arthritis, stem cells, MSCs, adipose MSCs