TBI Using IMRT and Cyclophosphamide Prior to Stem Cell Transplant for the Treatment of Severe Systemic Sclerosis

NCT04380831 | Recruiting Early Phase 1 DMC FDA Drug

• 2 other identifiers: 20006NCI-2020-02744
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This early phase I trial studies the side effects and feasibility of total body irradiation using intensity modulation radiation therapy (IMRT) when given in combination with cyclophosphamide prior to stem cell transplant to treat severe systemic sclerosis. IMRT delivers total body radiation therapy more precisely and may reduce radiation exposure to sensitive normal organs. Giving chemotherapy, such as cyclophosphamide, and total body irradiation before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the bone marrow for new blood-forming cells (stem cells) to grow. Giving IMRT and cyclophosphamide prior to stem cell transplant may work better in treating severe systemic sclerosis and reduce radiation doses to lung and kidneys compared to cyclophosphamide alone.

Conditions

Systemic Scleroderma

Outcome Measures

Primary Outcomes (2)

• Mean lung dose

  The study will be deemed feasible if \>= 80% of patients complete the intensity modulation radiation therapy (IMRT) to the total dose of 800 cGy, mean lung and kidney doses are limited to 200 cGy or less.

  Up to day 100

• Mean kidney dose

  The study will be deemed feasible if \>= 80% of patients complete the IMRT to the total dose of 800 cGy, mean lung and kidney doses are limited to 200 cGy or less.

  Up to day 100

Secondary Outcomes (2)

• Dose homogeneity for lungs, kidneys, and total body

  Up to day 100

• Transplant-related mortality

  Day 30 and day 100

Study Arms (1)

Treatment (TBI using IMRT, cyclophosphamide, HSCT)

EXPERIMENTAL

Patients undergo TBI using IMRT BID on days -5 and -4 in the absence of disease progression or disease progression. Patients then receive cyclophosphamide on days -3 and -2 and undergo HSCT on day 0 in the absence of disease progression or unacceptable toxicity.

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Cyclophosphamide; Radiation: Intensity-Modulated Radiation Therapy; Procedure: Total-Body Irradiation

Interventions

Total-Body Irradiation PROCEDURE

Undergo TBI using IMRT

Also known as: TBI, Total Body Irradiation, Whole Body Irradiation, Whole-Body Irradiation

Treatment (TBI using IMRT, cyclophosphamide, HSCT)

Cyclophosphamide DRUG

Undergo HSCT

Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719

Treatment (TBI using IMRT, cyclophosphamide, HSCT)

Intensity-Modulated Radiation Therapy RADIATION

Undergo TBI using IMRT

Also known as: IMRT, Intensity Modulated RT, Intensity-Modulated Radiotherapy, Radiation, Intensity-Modulated Radiotherapy

Treatment (TBI using IMRT, cyclophosphamide, HSCT)

Allogeneic Hematopoietic Stem Cell Transplantation PROCEDURE

Undergo HSCT

Also known as: Allogeneic Hematopoietic Cell Transplantation, allogeneic stem cell transplantation, HSC, HSCT, Stem Cell Transplantation, Allogeneic

Treatment (TBI using IMRT, cyclophosphamide, HSCT)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Karnofsky performance status (KPS) \>= 70
• Systemic sclerosis patients evaluated within 30 days of start of conditioning regimen who would be candidates for TBI-cyclophosphamide (Cy) per City of Hope (COH) guidelines and standard operating procedures (SOP) for autologous hematopoietic cell transplant
• Patients must be suitable for TBI conditioning regimens as part of transplant per radiation the referring hematologist
• Patients must have adequate organ function for HCT as determined by the hematologist
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation
• All subjects must have the ability to understand the treatment and the willingness to sign a written informed consent

You may not qualify if:

• Patients should not have any uncontrolled illness including ongoing or active infection
• Prior history of radiation therapy must be presented to study principal investigator (PI) for eligibility determination
• Pregnant women are excluded from this study because total body irradiation is an agent with the potential for teratogenic or abortifacient effects
• Subjects, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study

Sponsors & Collaborators

• National Cancer Institute (NCI): collaborator
• City of Hope Medical Center: lead

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91010, United States

RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic

Interventions

Stem Cell Transplantation; Cyclophosphamide; Radiotherapy, Intensity-Modulated; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Radiotherapy, Conformal; Radiotherapy, Computer-Assisted; Radiotherapy; Investigative Techniques

Study Officials

• Jeffrey Y Wong

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2020
• First Posted: May 8, 2020
• Study Start: February 24, 2022
• Primary Completion (Estimated): December 22, 2026
• Study Completion (Estimated): December 22, 2026
• Last Updated: March 30, 2026

Record last verified: 2026-03

Locations

US (1)