Total Body Irradiation for Pulmonary Hypertension

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Pulmonary Hypertension+3 MoreTotal Body Irradiation - Radiation
Eligibility
8 - 60
All Sexes
What conditions do you have?
Select

Study Summary

This trial will test a new immunotherapy treatment for scleroderma, to see if it is safe and more effective than other treatments.

Eligible Conditions
  • Pulmonary Hypertension
  • Diffuse Systemic Sclerosis
  • Interstitial Lung Disease
  • Progressive Systemic Sclerosis

Treatment Effectiveness

Study Objectives

7 Primary · 6 Secondary · Reporting Duration: 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.

Year 1
Decrease in DLCO sustained for 3 months or longer on pulmonary function tests (PFT)
Worsening of > 10% of FVC (pulmonary function testing)
Month 24
An increase of mRSS (modified Rodnan skin score ) by ≥5 points for skin score
Increase by ≥25% if the baseline mRSS > 20.
Month 36
Treatment-related mortality (TRM)
Month 36
Death
Development of scleroderma renal crisis (hypertensive or non-hypertensive)
Renal Failure
Respiratory Failure
The occurrence of cardiomyopathy
Worsening of cardiac involvement
Month 36
High Dose Immunoablative therapy-Safety
Month 36
High Dose Immunoablative therapy-Treatment Effect

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Palifermin 180 μg/kg on Day -3 (D)
58%Diarrhoea
50%Nausea
50%Vomiting
42%Fatigue
42%Cough
33%Constipation
33%Oedema
33%Anorexia
33%Rash
17%Folliculitis
17%Dyspepsia
17%Headache
17%Asthenia
17%Bacteraemia
17%Neuropathy
17%Dizziness
17%Anxiety
17%Depression
17%Confusional state
17%Insomnia
17%Haematuria
17%Pruritus
17%Flushing
8%Hypertension
8%Engraftment syndrome
8%Febrile neutropenia
8%Hyperbilirubinaemia
8%Atrial fibrillation
8%Dysgeusia
8%Eye pruritus
8%Photophobia
8%Eye swelling
8%Abdominal pain
8%Pyrexia
8%Oesophagitis
8%Haematochezia
8%Tongue coated
8%Tongue disorder
8%Tongue geographic
8%Face oedema
8%Chills
8%Axillary pain
8%Chest discomfort
8%Oedema peripheral
8%Localised oedema
8%Feeling jittery
8%Agitation
8%Non-cardiac chest pain
8%Migraine
8%Peripheral sensory neuropathy
8%Syncope
8%Herpes simplex
8%Staphylococcal bacteraemia
8%Skin laceration
8%Upper respiratory tract infection
8%Delayed engraftment
8%Alanine aminotransferase increased
8%Aspartate aminotransferase increased
8%Transfusion reaction
8%Weight decreased
8%Intraocular pressure increased
8%Bone pain
8%Arthralgia
8%Chest wall pain
8%Joint stiffness
8%Mental disorder
8%Pollakiuria
8%Urinary retention
8%Dysuria
8%Prostatitis
8%Epistaxis
8%Dyspnoea
8%Hiccups
8%Lung infiltration
8%Dermatitis acneiform
8%Dermatitis allergic
8%Erythema
8%Drug eruption
8%Palmar-plantar erythrodysaesthesia syndrome
8%Petechiae
8%Rash erythematous
8%Hypotension
8%Jugular vein thrombosis
8%Orthostatic hypotension
This histogram enumerates side effects from a completed 2010 Phase 3 trial (NCT00109031) in the Palifermin 180 μg/kg on Day -3 (D) ARM group. Side effects include: Diarrhoea with 58%, Nausea with 50%, Vomiting with 50%, Fatigue with 42%, Cough with 42%.

Trial Design

1 Treatment Group

Autologous Stem Cell Transplantation
1 of 1

Experimental Treatment

8 Total Participants · 1 Treatment Group

Primary Treatment: Total Body Irradiation · No Placebo Group · Phase 2

Autologous Stem Cell TransplantationExperimental Group · 9 Interventions: Rituximab, Anti Thymocyte Globulin, GM-CSF, Thiotepa, Alemtuzumab, Intravenous immunoglobulin, Total Body Irradiation, Cyclophosphamide, Mesna · Intervention Types: Drug, Drug, Drug, Drug, Drug, Drug, Radiation, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Rituximab
FDA approved
Anti Thymocyte Globulin
2009
Completed Phase 4
~120
Filgrastim
FDA approved
Thiotepa
FDA approved
Alemtuzumab
FDA approved
Human immunoglobulin G
FDA approved
Total Body Irradiation
2006
Completed Phase 3
~890
Cyclophosphamide
FDA approved
Coenzyme M
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 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.

Who is running the clinical trial?

Paul SzabolcsLead Sponsor
6 Previous Clinical Trials
199 Total Patients Enrolled
Paul Szabolcs, MDPrincipal Investigator - University of Pittsburgh
University of Pittsburgh
6 Previous Clinical Trials
196 Total Patients Enrolled

Eligibility Criteria

Age 8 - 60 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Individuals must meet all the following criteria to be eligible for this study.
You must have given informed consent for this procedure.
You are aged 8-24 years at the time of consent.
You have skin thickening over the past 6 months or mRSS ≥ 20.
You have increased ground glass opacities or reticulations on a CT scan or decreased FVC% or DLCO% predicted value of ≥10% on pulmonary function testing.