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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 below

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I am between 8 and 24 years old.

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I was diagnosed with Systemic Sclerosis before I turned 20.

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My lung condition has worsened in the last 18 months.

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I have sores on the tips of my fingers or toes.

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My arthritis hasn't improved after trying 2 different treatments.

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My skin has gotten thicker recently or my skin score is 20 or more.

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My lung scans show changes or my lung function tests are worse by 10% or more.

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My muscle enzyme levels are high or my MRI shows muscle inflammation.

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I am not pregnant.

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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:

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My kidney function is reduced, with a filtration rate under 40 mL/min.

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I do not have any serious infections that would make immune-suppressing therapy unsafe for me.

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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.

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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. for reporting.

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 more

Secondary 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 more

Side effects data

From 2010 Phase 3 trial • 47 Patients • NCT00109031

58%

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

Petechiae

Pollakiuria

Photophobia

Engraftment syndrome

Febrile neutropenia

Eye pruritus

Eye swelling

Abdominal pain

Haematochezia

Oesophagitis

Tongue coated

Tongue disorder

Tongue geographic

Axillary pain

Chest discomfort

Chills

Face oedema

Feeling jittery

Non-cardiac chest pain

Oedema peripheral

Pyrexia

Hyperbilirubinaemia

Herpes simplex

Staphylococcal bacteraemia

Upper respiratory tract infection

Delayed engraftment

Skin laceration

Alanine aminotransferase increased

Aspartate aminotransferase increased

Intraocular pressure increased

Weight decreased

Arthralgia

Bone pain

Chest wall pain

Joint stiffness

Dysgeusia

Migraine

Peripheral sensory neuropathy

Syncope

Agitation

Mental disorder

Dysuria

Urinary retention

Prostatitis

Dyspnoea

Epistaxis

Hiccups

Lung infiltration

Dermatitis acneiform

Dermatitis allergic

Drug eruption

Erythema

Rash erythematous

Hypertension

Hypotension

Jugular vein thrombosis

Localised oedema

Atrial fibrillation

Transfusion reaction

Palmar-plantar erythrodysaesthesia syndrome

Orthostatic hypotension

100%

80%

60%

40%

20%

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

~1290