A Phase 1/2 interventional study of Mesenchymal Stem Cells and Placebo in Rheumatoid Arthritis, Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis, sponsored by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan. Enrolling by invitation at 2 sites in Pakistan. Open to participants aged 18 Years to 50 Years. Per ClinicalTrials.gov, last updated 2025-03-21.
Sponsored by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan · Phase 1/2, Interventional, and Treatment
The goal of this study is to learn if mesenchymal stem cell therapy (treatment group) can effectively treat autoimmune diseases, when compared to normal saline (given to placebo group). The primary outcome measures will be clinical improvement based on the respective disease specific clinical scores, normalization of T-lymphocyte subsets and > 50% reduction in disease specific antibody titres. The study will also document the type and frequency of any adverse event or side effects, reported by or seen in any of the trial participants.
Patients in treatment group will receive single session of MSC therapy and placebo group will receive 0.9% saline solution. The participants will be followed at 3 and 6 months.
The autoimmune disorders are a spectrum of diseases ranging from organ-specific, in which antibodies and T cells react to self-antigens localized in a specific tissue, to systemic, which are characterized by reactivity against a specific antigen(s) spread throughout various tissues in the body. The incidence of different autoimmune diseases is variable, according to the Centers for Disease Control and Prevention, 22.7% have rheumatic diseases. Although no population-based epidemiological study has been carried out in Pakistan purely on autoimmune diseases; a study by Mohsin Z et al. reports the combined prevalence of rheumatic diseases at a tertiary hospital in Karachi to be 17.3%. Autoimmune diseases and their treatment modalities are the prime cause of disability in developing countries and have the highest incapacitating rates in health-related quality of life (HRQoL) and daily functioning.
The 21st century has seen rapid advances in the treatment of diseases of immune dysfunction. One such treatment modality is stem cell therapy which is believed to repair and regenerate tissues. In particular, mesenchymal stem cells (MSCs) have been applied to treat diseases associated with age, changing lifestyle, immune dysfunction and stroke. MSC therapy has promise to treat various autoimmune disorders like refractory systemic lupus erythematosus (SLE), Crohn's disease, systemic sclerosis (SS), rheumatoid arthritis (RA), multiple sclerosis (MS), graft versus host disease, diabetes mellitus, thyroiditis and even different types of neurological disorders. At present, nearly a thousand clinical trials have used MSC-based therapies. Among those around one hundred trials have been conducted for treatment of immune-mediated disorders, the first one being more than fifteen years ago (table 1).
The interest surrounding field of MSCs was initially based on their inherent capacity for self-renewal and regeneration with a potential to form cells of mesodermal origin (adipocytes, osteocytes, chondrocytes, hepatocytes, neurons, muscle cells and epithelial cells) depending on the surrounding microenvironment. Later on, due to their abilities to home to inflamed areas and exert immunomodulatory effects, therapies with MSCs extended to treatment of autoimmune and chronic inflammatory processes. Multiple studies have also demonstrated that MSCs have intrinsic immunomodulatory and anti-inflammatory properties.
Table 1: Clinical trials in which mesenchymal stem cells are being used as therapeutic modality.
Immune mediated disorder Number of Clinical trials Year of first Clinical trial Reference Graft vs. host disease 49 2004 Inflammatory bowel disease 23 2006 Multiple sclerosis 29 2006 Systemic lupus erythematosus 10 2007 Type I diabetes 26 2008 Primary Sjögren syndrome 1 2009 Type II diabetes 13 2010 Autoimmune hepatitis 2 2011 Ankylosing spondylitis 2 2011 Chronic urticarial 1 2017 Refractory autoimmune thrombocytopenia 1 2019
Source of MSCs MSCs were initially identified in the BM and are commonly isolated by gradient centrifugation to separate nucleated cells, followed by in vitro culture and serial passages. The Mesenchymal and Tissue Stem Cell Committee of the International Society for Cellular Therapy (ISCT) has designated the term 'multipotent mesenchymal stromal cells' for the plastic-adherent cells found under standard culture conditions. The immunophenotype of these cells as per ISCT criteria is positive for cell-surface markers CD73, CD90 and CD105 and negative for surface CD14 or CD11b, CD45, CD34, CD79 or CD19, and HLA-DR. MSCs have also been obtained from adipose tissue, placenta, amniotic fluid, umbilical cord blood (UCB), connective tissues of skeletal muscle and dermis,dental tissue, and fetal tissues such as lung and blood. Mobilized peripheral blood cells have also been reported as a source of MScs. Although gene expression studies demonstrate that MSC populations obtained from different tissue sources are highly heterogeneous, their ability to renew, differentiate, and major functional properties, such as regulation of immunological tolerance, wound healing, inflammation and fibrosis, are common to all MSCs.
Translation of MSC knowledge into clinical application Cell therapy indeed appears to be an applicable translational strategy for autoimmune diseases.
In another study, 81 patients with PM/DM were randomly divided into two groups: 44 patients in the control group were individually treated with glucocorticoids and immunosuppressants for 6 months, while 37 patients in the transplantation group were injected intravenously with 3.5-5.2 × 107 UC-MSCs. The results of that study showed that the creatine kinase values in both groups were significantly decreased; however, the transplantation group had better results than the control group at several time points, and the lung function was significantly improved in the transplantation group. One patient died after transplantation and no transplantation-related complications occurred. Currently, there are only a few studies investigating PM/DM, and large-scale and randomized clinical studies are needed to evaluate the long-term effectiveness and safety of MSCT in PM/DM patients, including the risks of tumors and infections, as well as the optimal transplantation dose and schedule.
Since the clinical outcome in case reports and phase I-II trials seem occasionally striking, there exists a dire need to perform structured and preferably controlled multicenter trials and document results in our own population.
3,332 studies on the registry are indexed under Arthritis, Rheumatoid; 443 are open to participants now.
This study's planned enrollment of 200 is above the median of 96 across 2,220 interventional studies indexed under Arthritis, Rheumatoid.
Browse Arthritis, Rheumatoid studies →National Institute of Blood and Marrow Transplant (NIBMT), Pakistan is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
A- RA
Exclusion Criteria:
Allergy to methotrexate (MTX)
Inclusion criteria for SLE
Disease activity that is to hydroxychoroquine and one DMARD, or patients unable to take hydroxychoroquine due to contra-indication or prior toxicity Exclusion criteria for SLE Severe "critical" SLE flare defined as: (i) BILAG 2004 A flare in CNS system; (ii) BILAG 2004 A flare in the renal system; or (iii) any other SLE manifestation requiring more immunosuppression than allowed within the protocol in the physician's opinion
Diagnosis of moderate to severe AS with prior documented radiologic evidence (x-ray or radiologist's report) fulfilling the Modified New York criteria for AS:
Total back pain as measured by VAS ≥ 40 mm (0-100 mm) at Baseline Patients should have had inadequate response or failure to respond to at least 2 NSAIDs at an approved dose for a minimum of 4 weeks in total and a minimum of 2 weeks for each NSAID prior to randomization, or less than 4 weeks if therapy had to be withdrawn due to intolerance, toxicity or contraindications Patients who are regularly taking NSAIDs (including COX-1 or COX-2 inhibitors) as part of their AS therapy are required to be on a stable dose for at least 2 weeks before randomization patients who have been on a TNFα inhibitor (not more than one) must have experienced an inadequate response to previous or current treatment given at an approved dose for at least 3 months prior to randomization or have been intolerant to at least one administration of an anti-TNFα agent Exclusion criteria for ankylosing spondlitis
Subjects with a diagnosis of Juvenile Dermatomyositis (JDM) must meet either of the following criteria:
An MMT-8 (Manual Muscle Test) score that is no greater than 125/150 in conjunction with 2 other abnormal core set measures.
OR
If MMT (Manual Muscle Test) score is greater than 125/150 the patient MUST meet at least 3 abnormal core set measures.
Willing to forgo immunization with a live vaccine for the duration of the study Exclusion for dermatomyositis
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
MSC therapy group
Biological: Mesenchymal Stem Cells
Placebo (0.9% saline)
Other: Placebo
allogeneic bone marrow-derived mesenchymal stem cells therapy
0.9% Saline as placebo
Primary Outcome Measures for RA
Change From Baseline in the Disease Activity Score- Erythrocyte Sedimentation Rate (DAS28-ESR) at Week 48 The DAS28-ESR is a score on a scale (0 to 10) that is a measure of the subject's disease activity. It is based on the tender joint count (28 joints), swollen joint count (28 joints), patient's global assessment of disease activity (mm), and ESR (mm/hour). Lower score indicates less disease activity. Flares in disease activity are defined as an increase in this score of greater than 1.2 and remission is defined as achieving a DAS28-ESR score of less than 2.6.
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for RA
Change From Baseline in Tender Joint Count Score at Week 48 Tender Joint Count (TJC) is calculated based on tenderness response of 28 joints. TJC possible values range from 0 to 28. A lower TJC indicates less joint tenderness. Change from baseline is computed as Week 48 value minus baseline value. A negative value in change from baseline indicates an improvement.
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for RA
Change From Baseline in Swollen Joint Count at Week 48 Swollen Joint Count (SJC) is calculated based on swelling response of 28 joints. SJC possible values range from 0 to 28. A lower SJC indicates less joint swelling. Change from baseline is computed as Week 48 value minus baseline value. A negative value in change from baseline indicates an improvement
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for RA
Change From Baseline in Patient's Assessment of Arthritis Pain-Visual Analog Scale (PAAP-VAS) at Week 48 Change from Baseline in PAAP-VAS (0 to 100 millimeters visual analog scale, 0 being no pain and 100 being most severe pain) is computed as the value at Week 48 minus the Baseline value. A negative value in change from Baseline indicates an improvement.
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for RA
Change From Baseline in Patient's Global Assessment of Disease Activity- Visual Analog Scale (PtGADA-VAS) at Week 48 Change from Baseline in PtGADA-VAS (0 to 100 mm visual analog scale, 0 being no symptoms and 100 being severe symptoms) is computed as the value at Week 48 minus the Baseline value. A negative value in change from Baseline indicates an improvement.
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for RA
Change From Baseline in Physician's Global Assessment of Patient's Disease Activity- Visual Analog Scale (PhGADA-VAS) at Week 48
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for Systemic lupus erythematosus
Proportion of patients achieving BILAG-based Composite Lupus Assessment (BICLA)
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for systemic sclerosis
Change From Baseline in Modified Rodnan Skin Score (mRSS) at Week 48 Skin thickness was assessed by the mRSS. The mRSS was rated with scores ranging from 0 (normal) to 3 (severe skin thickening) across 17 different sites. The total score was the sum of the individual skin scores in the 17 body areas (e.g., face, hands, fingers; proximal area of the arms, distal area of the arms, thorax, abdomen; proximal area of the legs, and distal area of the legs, feet), giving a range of 0-51 units and had been validated for participants with systemic sclerosis (SSc). A negative change from baseline showed improvement.
Time frame: Day 0, Weeks 12, 24, 48
Primary Outcome Measures for systemic sclerosis
Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 0, Weeks 12, 24, 48
Primary outcome measures in ankylosing spondlitis
The Proportion of Participants Who Achieve an ASAS 20 Response (Assessment of SpondyloArthritis International Society Criteria) ASAS20 response is defined as an improvement of ≥20% and ≥1 units on a scale of 10 in at least three of the four ASAS main domains and no worsening of ≥20% and ≥1 unit on a scale of 10 in the remaining domain
Time frame: Week 16
Primary Outcome Measure for dermatomyositis
Comparison Between the Time to Improvement Between the Two Groups of Idiopathic Inflammatory Myopathy Patients at week 48 ASAS20 response is defined as an improvement of ≥20% and ≥1 units on a scale of 10 in at least three of the four ASAS main domains and no worsening of ≥20% and ≥1 unit on a scale of 10 in the remaining domain
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for RA
Change From Baseline in the Short Form 36 (SF-36) Physical and Mental Health Component Summary Scores (PCS and MCS) at Week 48 \[ Time Frame: Baseline (Day 0), Week 12,week 24,week48 \] It has 36 questions with 8 subscale scores and 2 summary scores: PCS=physical functioning, role-physical, bodily pain, and general health; MCS=vitality, social functioning, role-emotional, and mental health. Scoring is done for both subscores and summary scores. For both, 0=worst score (or quality of life) and 100=best score. Change from baseline is computed as the value at Week 48 minus the baseline value. A positive value in change from Baseline indicates an improvement and a negative value worsening.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for RA
Change From Baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at Week 48 \[ Time Frame: Each question is scored 0-3 (0 = without any difficulty, 1 = with some difficulty, 2 = with much difficulty, and 3 = unable to do). In addition, category scores are modified if an aid or device is used, for example, a walker or wheelchair, or help is received from another person in the daily living activities. If an aid or device is used or help is received then a category score of 0 or 1 increases to a category score of 2. A category score of 3 remains a 3 regardless of aids, devices, or help. Scores from each of the 8 categories are totaled. The total score can range from 0 to 24. Change from baseline is computed as the total score at Week 48 minus the baseline total score. A negative value in change from baseline indicates an improvement.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for RA
Change From Baseline in Erythrocyte Sedimentation Rate (ESR) at Week 48 ESR is a blood test used to monitor therapy in inflammatory diseases such as rheumatoid arthritis and reflects acute phase reactant levels. Active disease in RA is defined by an ESR greater than 30 mm/hr. Change from baseline is computed as the value at Week 48 minus the baseline value. A negative value in change from baseline indicates an improvement
Time frame: Day 0, Week 12, week 24, week48
Secondary Outcome Measures for SLE
Proportion of patients achieving SLEDAI responder Index (SRI)
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SLE
Number of serious adverse events
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Physical Function Assessed by Scleroderma Health Assessment Questionnaire Disability Index (SHAQ-DI) Each VAS item was rated separately (0-100 millimeters \[mm\]), with higher scores indicating more severe disease. The five items were: 1) intestinal disease, 2) breathing problem, 3) Raynaud syndrome, 4) finger ulcers, and 5) overall disease.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) There are four possible responses for each component: 0 = without any difficulty; 1 = with some difficulty; 2 = with much difficulty; 3 = unable to do. The HAQ-DI was the sum of the domain scores, divided by the number of domains that have a score (i.e. the average score), with total range of 0 to 3, higher scores showing larger functional limitation.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Clinician's Global Assessment The Clinician's Global Assessment evaluated the overall impact of SSc on the participant as assessed by the physician on a VAS with scores ranging from 0 to 100 mm, with higher scores indicating worse disease in terms of severity, damage, or overall disease, but there was no standardization for the scale.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Patient's Global Assessment VAS scale (0 mm to 100 mm), with higher scores indicating worsening disease.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score The endpoint measured was fatigue. On this scale, a numerical increase indicated an improvement in the participant's condition.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in 5-D Itch Scale Each domain was scored on a 5-point scale, the scores of each of the five domains were achieved separately and then summed together to obtain a total 5-D score. 5-D scores ranged between 5 (no pruritus) and 25 (most severe pruritus).
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in mRSS
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Percentage of Participants who maintained or Improved in mRSS
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Change From Baseline in Tender Joint Count 10 proximal interphalangeal joint (PIP) joints, and both knees, were assessed. Joints were classified as not tender = 0 or tender = 1. Observed data was presented for this outcome measure.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measures for SC
Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measure for ALS
The Proportion of Participants Who Achieve an ASAS40 Response ASAS40 response is defined as an improvement of ≥40% and ≥2 units on a scale of 10 in at least three of the four ASAS main domains and no worsening at all in the remaining domain
Time frame: Week 16
Secondary Outcome Measure for ALS
The Proportion of Participants Who Achieve an ASAS40 Response
Time frame: Week 16
Secondary Outcome Measure for ALS
Change in high-sensitivity C-reactive protein (hsCRP) Over Time
Time frame: Week 16
Secondary Outcome Measure for ALS
Percentage of Participants Who Achieve an ASAS 5/6 The ASAS 5/6 improvement criteria is an improvement of ≥20% in at least five of all six domains
Time frame: Week 16
Secondary Outcome Measure for ALS
Participants With Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Response The BASDAI or Bath Ankylosing Spondylitis Disease Activity Index consists of a 0 through 10 scale (0 being no problem and 10 being the worst problem, captured as a continuous VAS), which is used to answer 6 questions pertaining to the 5 major symptoms of AS
Time frame: Week 16
Secondary Outcome Measure for ALS
Change in Short Form (36) Physical Component Summary (PCS) Responders (Improvement of \>= 2.5 Points) at Week 16
Time frame: Week 16
Secondary Outcome Measure for ALS
Change in Ankylosing Spondylitis Quality of Life (ASQoL) Score Over Time The Ankylosing Spondylitis Quality of Life (ASQoL) is an instrument to assess health-related quality of life among adult patients with Ankylosing Spondylitis. Each statement on the ASQoL is given a score of "1" or "0". A score of "1" is given where the item is affirmed, indicating adverse QoL. All item scores are summed to give a total score or index. Scores can range from 0 (good QoL) to 18 (poor QoL).
Time frame: Week 16
Secondary Outcome Measure for ankylosing spondlitis
The Proportion of Patients Who Achieve an The Assessment in SpondyloArthritis International Society (ASAS) Partial Remission The The Assessment in SpondyloArthritis International Society (ASAS) partial remission criteria are defined as a value not above 2 units in each of the four main domains on a scale of 10
Time frame: Week 16
Secondary Outcome Measure for dermatomyositis
Response Rates (Proportion of Improved Patients) The Definition of Improvement for both adult and pediatric patients will be: 3 of any of the 6 core set measures improved by ≥ 20%, with no more than 2 of the core set measures worsening by ≥25% (worsening measure cannot include the MMT) at two consecutive visits. Of note, the MMT could not be one of the worsening measures.
Time frame: Day 0, Weeks 12, 24, 48
Secondary Outcome Measure for dermatomyositis
20% Improvement in Manual Muscle Testing (MMT) Over Baseline on Two Consecutive Time Points (Muscle is the Primary Organ of Involvement, and MMT is the One Objective Measurement of the Definition of Improvement \[DOI\])
Time frame: Day 0, Weeks 12, 24, 48
Plan to share: No
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National Institute of Blood and Marrow Transplant (NIBMT), Pakistan