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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.
Must not have
Estimated CrCl <40 mL/min, using Cockcroft-Gault formula based on actual body weight
Active, uncontrolled infection that would be a contraindication to safe use of high-dose immunosuppressive therapy or cyclophosphamide
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
No Placebo-Only Group
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.
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.
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.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 8 and 24 years old.
Select...
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.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My kidney function is reduced, with a filtration rate under 40 mL/min.
Select...
I do not have any serious infections that would make immune-suppressing therapy unsafe for me.
Select...
I have an ongoing, untreated kidney crisis due to scleroderma.
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
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Death
High Dose Immunoablative therapy-Safety
High Dose Immunoablative therapy-Treatment Effect
+4 moreSecondary study objectives
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 • NCT0010903158%
Diarrhoea
50%
Nausea
50%
Vomiting
42%
Fatigue
42%
Cough
33%
Constipation
33%
Oedema
33%
Anorexia
33%
Rash
17%
Pruritus
17%
Dyspepsia
17%
Asthenia
17%
Bacteraemia
17%
Folliculitis
17%
Dizziness
17%
Headache
17%
Neuropathy
17%
Anxiety
17%
Confusional state
17%
Depression
17%
Insomnia
17%
Haematuria
17%
Flushing
8%
Pollakiuria
8%
Petechiae
8%
Photophobia
8%
Engraftment syndrome
8%
Febrile neutropenia
8%
Eye pruritus
8%
Eye swelling
8%
Abdominal pain
8%
Haematochezia
8%
Oesophagitis
8%
Tongue coated
8%
Tongue disorder
8%
Tongue geographic
8%
Axillary pain
8%
Chest discomfort
8%
Chills
8%
Face oedema
8%
Feeling jittery
8%
Non-cardiac chest pain
8%
Oedema peripheral
8%
Pyrexia
8%
Hyperbilirubinaemia
8%
Herpes simplex
8%
Staphylococcal bacteraemia
8%
Upper respiratory tract infection
8%
Delayed engraftment
8%
Skin laceration
8%
Alanine aminotransferase increased
8%
Aspartate aminotransferase increased
8%
Intraocular pressure increased
8%
Weight decreased
8%
Arthralgia
8%
Bone pain
8%
Chest wall pain
8%
Joint stiffness
8%
Dysgeusia
8%
Migraine
8%
Peripheral sensory neuropathy
8%
Syncope
8%
Agitation
8%
Mental disorder
8%
Dysuria
8%
Urinary retention
8%
Prostatitis
8%
Dyspnoea
8%
Epistaxis
8%
Hiccups
8%
Lung infiltration
8%
Dermatitis acneiform
8%
Dermatitis allergic
8%
Drug eruption
8%
Erythema
8%
Rash erythematous
8%
Hypertension
8%
Hypotension
8%
Jugular vein thrombosis
8%
Localised oedema
8%
Atrial fibrillation
8%
Transfusion reaction
8%
Palmar-plantar erythrodysaesthesia syndrome
8%
Orthostatic hypotension
100%
80%
60%
40%
20%
0%
Study treatment Arm
Palifermin 180 μg/kg on Day -3 (D)
Palifermin 60 µg/kg for 3 Days (A)
Palifermin 180 μg/kg on Day -1 (B)
Palifermin 180 μg/kg on Day -2 (C)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
~2120
GM-CSF
2011
Completed Phase 4
~1280
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
~2990
Alemtuzumab
2004
Completed Phase 4
~1880
Cyclophosphamide
2010
Completed Phase 4
~2310
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.