Safety and Efficacy of Adipose Derived Stem Cells in Refractory Rheumatoid Arthritis, Systemic Lupus Erythematosus or Sharp's Syndrome
| Status: | Terminated |
|---|---|
| Conditions: | Arthritis, Lupus, Rheumatoid Arthritis |
| Therapuetic Areas: | Immunology / Infectious Diseases, Rheumatology |
| Healthy: | No |
| Age Range: | 18 - 99 |
| Updated: | 3/4/2017 |
| Start Date: | December 2015 |
| End Date: | December 31, 2016 |
A Phase I-II Open- Label Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Autologous Adipose Stem Cell (Adult Human) to Subjects With Refractory Rheumatoid Arthritis, Relapsing Systemic Lupus Erythematosus or Sharp's Syndrome
This is a Phase I-II open- label single-dose study in subjects with significant refractory
Rheumatoid Arthritis (RA), relapsing Systemic Lupus Erythematosus (SLE) or Sharp's Syndrome
(SS).
This study will enroll a minimum of 20 subjects for RA, 20 subjects for SLE and 20 patients
for SS. 6 week data of serum Tumor Necrosis Factor- alpha (TNFa), Interleukin- 6 (IL-6), C-
Reactive Protein (CRP), Erythrocyte Sedimentation Rate (ESR), Cluster of Differentiation
(CD)4 +CD25 + Forkhead box P3(Foxp3) + regulatory T cells, Disease Activity Score for 28
joints (DAS-28) score and pain score will be collected in all patients who are enrolled in
the study for the RA group (Baseline and 6 weeks after). For the SLE group, Transforming
Growth Factor- beta (TGF-β), TNFa, IL-6, Interleukin- 17 (IL-17), CD3+CD8-IL17A+ T helper-17
(Th17) cells, CD4+CD25+Foxp3+ regulatory T cells and the Systemic Lupus Erythematosus
Quality of Life Questionnaire (SLEQoL) score will be collected in all the subjects of this
group. SS group will undergo the assessments of RA and SLE. Prior to the stem cell
treatment, the patient will be assessed for 6 weeks by all the previously mentioned markers.
Then, patients will receive the infusion of stromal vascular fraction cells containing the
adult adipose derived stem cells 'aADSC' (single intravenous dose). The disease- modifying
anti-rheumatic drugs (DMARDs) or the standard SLE treatment will not be interrupted with the
exception of systemic steroids (excluding minimal maintenance dose of one steroid) during
the duration of the study. Follow up visits will take place at 6 weeks, 3 Months and 6
Months after the cell infusion. Safety will be monitored on an ongoing basis, and an interim
safety review will be conducted by the Investigator(s) and Sponsor after the first 10
patients have been enrolled and treated in each group.
Rheumatoid Arthritis (RA), relapsing Systemic Lupus Erythematosus (SLE) or Sharp's Syndrome
(SS).
This study will enroll a minimum of 20 subjects for RA, 20 subjects for SLE and 20 patients
for SS. 6 week data of serum Tumor Necrosis Factor- alpha (TNFa), Interleukin- 6 (IL-6), C-
Reactive Protein (CRP), Erythrocyte Sedimentation Rate (ESR), Cluster of Differentiation
(CD)4 +CD25 + Forkhead box P3(Foxp3) + regulatory T cells, Disease Activity Score for 28
joints (DAS-28) score and pain score will be collected in all patients who are enrolled in
the study for the RA group (Baseline and 6 weeks after). For the SLE group, Transforming
Growth Factor- beta (TGF-β), TNFa, IL-6, Interleukin- 17 (IL-17), CD3+CD8-IL17A+ T helper-17
(Th17) cells, CD4+CD25+Foxp3+ regulatory T cells and the Systemic Lupus Erythematosus
Quality of Life Questionnaire (SLEQoL) score will be collected in all the subjects of this
group. SS group will undergo the assessments of RA and SLE. Prior to the stem cell
treatment, the patient will be assessed for 6 weeks by all the previously mentioned markers.
Then, patients will receive the infusion of stromal vascular fraction cells containing the
adult adipose derived stem cells 'aADSC' (single intravenous dose). The disease- modifying
anti-rheumatic drugs (DMARDs) or the standard SLE treatment will not be interrupted with the
exception of systemic steroids (excluding minimal maintenance dose of one steroid) during
the duration of the study. Follow up visits will take place at 6 weeks, 3 Months and 6
Months after the cell infusion. Safety will be monitored on an ongoing basis, and an interim
safety review will be conducted by the Investigator(s) and Sponsor after the first 10
patients have been enrolled and treated in each group.
Inclusion Criteria:
1. Males and Females ≥18 years
2. Cognitive competitiveness.
3. Diagnosis of refractory RA (patient's poor response of 3 months of DMARDs determined
and documented by a rheumatologist) ,diagnosis of relapsing SLE (more than 2 episodes
in a year during optimal medical SLE therapy) or diagnosis of Sharp syndrome
(Alarcon-Segovia's criteria: patients with U1 Ribonucleic protein (RNP) ≥ 1:1600, and
three of the following: Swollen hands, Synovitis, Myositis, Raynauld's phenomenon,
Acrosclerosis)
4. Life expectancy greater than 8 months
5. Ability to understand and provide signed informed consent, or have a designated legal
guardian or spouse legally able and willing to make such decisions on the subject's
behalf
6. Reasonable expectation that patient will receive standard post-treatment care and
attend all scheduled safety follow-up visits
7. No changes in active pulmonary medications for heart failure during the two weeks
prior enrollment.
8. Written informed consent
Exclusion Criteria:
1. Currently on systemic steroids (if chronic steroid therapy cannot be stopped, the
patient will qualify if he/she is on a minimum maintenance dose per SLE, RA or SS
guidelines of only one systemic steroid and constituting a bioequivalent to normal
endogenous levels of that hormone).
2. Dementia.
3. Presence of immune deficiency.
4. Currently on NSAIDs, TNFa inhibitors, Calcium channel blockers or P2Y12 inhibitors
5. Complicated type 2 diabetes mellitus (presence of diabetic foot, advanced kidney
disease or retinopathy).
6. History of cancer within the past 5 years.
7. Presence of any other clinically-significant medical condition, psychiatric
condition, or laboratory abnormality, that in the judgment of the Investigator or
Sponsor for which participation in the study would pose a safety risk to the subject
8. Participation in another study with an investigational drug or device within 3 weeks
prior to treatment
9. History within the past year of drug or alcohol abuse.
10. Females known to be pregnant, lactating or having a positive pregnancy test (will be
tested during screening) or planning to become pregnant during the study.
11. Inability to comply with the conditions of the protocol.
12. Porphyria.
13. Allergy to sodium citrate or any "caine" type of local anesthetic.
14. Patient scheduled for hospice care.
We found this trial at
1
site
Little Rock, Arkansas 72211
Principal Investigator: Gerardo Rodriguez, MD PhD
Phone: 501-614-3641
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