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Chemotherapy
Stem Cell Transplant for Scleroderma (SSc Trial)
Phase 2
Recruiting
Led By Paul Szabolcs, MD
Research Sponsored by Paul Szabolcs
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Age 8-24, inclusive, at time of consent.
Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up disease response will be defined as subject improvement. this will be assessed for both skin and interstitial lung disease at 12 and 24 months post-hsct.
Awards & highlights
SSc Trial Summary
This trial will test a new immunotherapy treatment for scleroderma, to see if it is safe and more effective than other treatments.
Who is the study for?
This trial is for young individuals aged 8-24 with Systemic Sclerosis diagnosed before age 19, who haven't improved after trying at least two DMARDs. They should have skin thickening or worsening lung disease and be free from HIV, hepatitis B/C, and untreated renal crisis. Participants must not be pregnant, agree to birth control if applicable, and have clearance for stem cell transplantation.Check my eligibility
What is being tested?
The study tests a high-dose immunoablative therapy regimen using drugs like Thiotepa and Cyclophosphamide combined with treatments such as Total Body Irradiation in patients with Systemic Sclerosis. The goal is to assess safety compared to other regimens while looking for treatment effectiveness.See study design
What are the potential side effects?
Potential side effects include increased risk of infections due to immune system suppression, organ damage from high-dose medications or radiation, allergic reactions to the drugs used, blood disorders like anemia or low platelet counts, and possible infertility.
SSc Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 8 and 24 years old.
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I was diagnosed with Systemic Sclerosis before I turned 20.
Select...
My lung condition has worsened in the last 18 months.
Select...
I have sores on the tips of my fingers or toes.
Select...
My arthritis hasn't improved after trying 2 different treatments.
Select...
My skin has gotten thicker recently or my skin score is 20 or more.
Select...
My lung scans show changes or my lung function tests are worse by 10% or more.
Select...
My muscle enzyme levels are high or my MRI shows muscle inflammation.
Select...
I am not pregnant.
Select...
My heart is cleared by a cardiologist for stem cell transplantation.
SSc Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ disease response will be defined as subject improvement. this will be assessed for both skin and interstitial lung disease at 12 and 24 months post-hsct.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~disease response will be defined as subject improvement. this will be assessed for both skin and interstitial lung disease at 12 and 24 months post-hsct.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Death
High Dose Immunoablative therapy-Safety
High Dose Immunoablative therapy-Treatment Effect
+4 moreSecondary outcome measures
An increase of mRSS (modified Rodnan skin score ) by ≥5 points for skin score
Decrease in DLCO sustained for 3 months or longer on pulmonary function tests (PFT)
Development of scleroderma renal crisis (hypertensive or non-hypertensive)
+3 moreSide effects data
From 2010 Phase 3 trial • 47 Patients • NCT0010903160%
Fatigue
60%
Nausea
60%
Diarrhoea
60%
Vomiting
40%
Rash
30%
Insomnia
20%
Febrile neutropenia
20%
Abdominal pain
20%
Pyrexia
20%
Constipation
20%
Asthenia
20%
Dysgeusia
20%
Anorexia
20%
Pain in extremity
20%
Headache
20%
Flushing
20%
Chills
10%
Hiccups
10%
Pneumonia
10%
Anxiety
10%
Lipase increased
10%
Muscle spasms
10%
Blood amylase increased
10%
Dyspnoea
10%
Back pain
10%
Alopecia
10%
Dry mouth
10%
Catheter site pain
10%
Folliculitis
10%
Excoriation
10%
Arthralgia
10%
Dizziness
10%
Joint stiffness
10%
Gastritis
10%
Oral pain
10%
Rhinorrhoea
10%
Oedema peripheral
10%
Staphylococcal bacteraemia
10%
Dyspepsia
10%
Bacteraemia
10%
Dehydration
10%
Colitis
10%
Gastrointestinal pain
10%
Hypoaesthesia oral
10%
Oesophageal pain
10%
Tongue coated
10%
Oedema
10%
Confusional state
10%
Genital rash
10%
Pharyngolaryngeal pain
10%
Dry skin
10%
Erythema
10%
Urticaria
10%
Hypotension
10%
Abdominal distension
10%
Clostridium colitis
10%
Catheter site related reaction
10%
Sunburn
10%
Weight increased
10%
Crackles lung
100%
80%
60%
40%
20%
0%
Study treatment Arm
Palifermin 60 µg/kg for 3 Days (A)
Palifermin 180 μg/kg on Day -1 (B)
Palifermin 180 μg/kg on Day -2 (C)
Palifermin 180 μg/kg on Day -3 (D)
SSc Trial Design
1Treatment groups
Experimental Treatment
Group I: Autologous Stem Cell TransplantationExperimental Treatment9 Interventions
CD34-selected autologous stem cell being performed on CliniMACS depletion device. Conditioning regimen will not start sooner than 3 weeks, and ideally no more than 90 days, after cyclophosphamide dose in the mobilization regimen.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Thiotepa
2008
Completed Phase 3
~2210
GM-CSF
2014
Completed Phase 4
~1340
Intravenous immunoglobulin
2017
Completed Phase 3
~330
Total Body Irradiation
2006
Completed Phase 3
~820
Anti Thymocyte Globulin
2009
Completed Phase 4
~120
Rituximab
1999
Completed Phase 4
~1880
Alemtuzumab
2004
Completed Phase 4
~1890
Cyclophosphamide
1995
Completed Phase 3
~3770
Mesna
2003
Completed Phase 2
~1380
Find a Location
Who is running the clinical trial?
Paul SzabolcsLead Sponsor
7 Previous Clinical Trials
224 Total Patients Enrolled
Paul Szabolcs, MDPrincipal Investigator - University of Pittsburgh
University of Pittsburgh
7 Previous Clinical Trials
221 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My kidney function is reduced, with a filtration rate under 40 mL/min.I am between 8 and 24 years old.I was diagnosed with Systemic Sclerosis before I turned 20.My lung condition has worsened in the last 18 months.My lung function test shows less than 35% capacity.I have sores on the tips of my fingers or toes.Your liver enzyme levels are more than four times the normal limit.Your ANC (a type of white blood cell) count is less than 1500 cells per microliter of blood.You have less than 100,000 platelets per microliter of blood.I haven't had cancer, except for certain skin cancers or in situ carcinoma, in the last 2 years.My arthritis hasn't improved after trying 2 different treatments.My skin has gotten thicker recently or my skin score is 20 or more.You do not have HIV, hepatitis B, or hepatitis C, confirmed by a special blood test.I do not have any serious infections that would make immune-suppressing therapy unsafe for me.I, or my guardian, have agreed to participate in this study.My lung scans show changes or my lung function tests are worse by 10% or more.My muscle enzyme levels are high or my MRI shows muscle inflammation.I am not pregnant.I have an ongoing, untreated kidney crisis due to scleroderma.Abnormalities in the number of blood cells in a blood test.Your childhood myositis assessment score is less than 30.Your oxygen level is less than 92% when you're resting without using extra oxygen.I have arthritis.My heart is cleared by a cardiologist for stem cell transplantation.Your hemoglobin level is less than 9.0 grams per deciliter.
Research Study Groups:
This trial has the following groups:- Group 1: Autologous Stem Cell Transplantation
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any vacancies left in this clinical experiment?
"According to the official record at clinicaltrials.gov, this trial is in active recruitment mode. It was first announced on July 31st 2018 and last edited on September 7th 2022."
Answered by AI
What criteria should a person meet in order to be qualified for participating in this clinical study?
"Patients with systemic sclerosis and diffuse symptoms between the ages of 8 to 60 are eligible for admission into this clinical trial. At present, there is capacity to accept around 8 such individuals."
Answered by AI
In what circumstances is Total Body Irradiation commonly employed?
"Total Body Irradiation has proven to be a viable form of treatment for multiple sclerosis, B-cell lymphomas, and polyangium."
Answered by AI
How many individuals are enrolled in this trial?
"Indeed. According to clinicaltrials.gov, this research endeavour was first announced on July 31st 2018 and the details were most recently refreshed on September 7th 2022. 8 individuals must be recruited from 2 separate sites for the study's completion."
Answered by AI
Does the FDA sanction Total Body Irradiation as a viable treatment option?
"Since the clinical trial is currently in Phase 2, which indicates that while there are data points supporting safety but no evidence on efficacy yet, we have assigned Total Body Irradiation a score of 2."
Answered by AI
Could you provide information about additional experiments involving Total Body Irradiation?
"At the present moment, 1230 clinical trials concerning Total Body Irradiation are currently underway with 253 studies in Phase 3. A significant number of these experiments for Total Body Irradiation are concentrated within Philadelphia, Pennsylvania; however, there is a total of 38 749 sites hosting related research initiatives."
Answered by AI
What is the ultimate goal of this experimental initiative?
"This medical trial will observe its participants over a 36-month period post HSCT to measure the prevalence of Respiratory Failure. Secondary objectives include tracking Worsening Cardiac Involvement (new or worsening arrhythmias that require long-term medical treatment, a decline in ejection fraction), Decrease in DLCO on PFTs (15% increase sustained for three months), and an increase in mRSS by 5 points for skin score - all used as qualitative metrics for cutaneous response."
Answered by AI
Does this examination accept participants who are older than 35 years of age?
"Patient eligibility for this trial is restricted to those between 8 and 60 years of age. Separately, there are 505 studies that cater to individuals below the age of consent and 2261 trials catering to seniors above 65."
Answered by AI
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