Systemic SclerodermaEarly Phase 1NCT04380831
TBI Using IMRT and Cyclophosphamide Prior to Stem Cell Transplant for the Treatment of Severe Systemic Sclerosis
A Early Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Intensity-Modulated Radiation Therapy in people with Systemic Scleroderma, run by City of Hope Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Scleroderma
- What is being tested?
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Intensity-Modulated Radiation Therapy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Feb 2022to 22 Dec 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: Pilot Study of Total Body Irradiation Using Intensity Modulated Radiation Therapy (IMRT) and Cyclophosphamide Conditioning Regimen Prior to Autologous Hematopoietic Cell Transplantation in Patients With Severe Systemic Sclerosis
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.
Read the detailed description
PRIMARY OBJECTIVES:
- To assess the feasibility of using IMRT to deliver total body irradiation (TBI) to 800 cGy while keeping mean lung and kidney doses to 200 cGy.
II. To assess the safety/feasibility of total body irradiation using IMRT (IMRT TBI) in systemic sclerosis patients undergoing autologous hematopoietic stem cell transplantation.
SECONDARY OBJECTIVES:
- To evaluate dose homogeneity and dose sparing to lung and kidneys. II. To evaluate transplant-related mortality at 30 days and 100 days post IMRT TBI.
OUTLINE:
Patients undergo TBI using IMRT twice daily (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.
After completion of study treatment, patients are followed up on days 30 and 100.
- Conditions
- Systemic Scleroderma
- Being tested
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Intensity-Modulated Radiation Therapy, Total-Body Irradiation
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called Allogeneic Hematopoietic Cell Transplantation, allogeneic stem cell transplantation, HSC, HSCT
Undergo HSCT
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida
Undergo HSCT
Intensity-Modulated Radiation TherapyRadiation
Also called IMRT, Intensity Modulated RT, Intensity-Modulated Radiotherapy, Radiation, Intensity-Modulated Radiotherapy
Undergo TBI using IMRT
Total-Body IrradiationProcedure or surgery
Also called TBI, Total Body Irradiation, Whole Body Irradiation, Whole-Body Irradiation
Undergo TBI using IMRT
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Cyclophosphamide; Radiation: Intensity-Modulated Radiation Therapy; Procedure: Total-Body Irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to day 100
Secondary outcomes (2)
Dose homogeneity for lungs, kidneys, and total body
"Dose homogeneity for lung, kidney and total body will use the DVH (dose volume histograms) generated by the treatment plan for each of these regions. The goal is to keep the lung and kidney dose homogeneity to with +/- 15% of the mean dose to that organ. The goal is to keep total body dose homogeneity to within +/- 15% of the prescribed total body dose of 8 Gy."
Measured: Up to day 100
Transplant-related mortality
Measured by comparing the transplant-related mortality rates on day 30 compared to day 100.
Measured: Day 30 and day 100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2020
- Study start24 Feb 2022
- Primary completion22 Dec 2026 (estimated)
- Study completion22 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerodermaStudies of the same condition, open and under way.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Jeffrey Y Wong, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04380831
- Sponsor's protocol ID
- 20006
- Other IDs
- NCI-2020-02744, 20006
- Status last verified
- Aug 2026
- First submitted
- 30 Apr 2020
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Total Body Irradiation Using Intensity Modulated Radiation Therapy (IMRT) and Cyclophosphamide Conditioning Regimen Prior to Autologous Hematopoietic Cell Transplantation in Patients With Severe Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT04380831
- Acronym / sponsor ID
- 20006
- Conditions
- Systemic Scleroderma
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 24 Feb 2022 (actual)
- Primary completion
- 22 Dec 2026 (estimated)
- Study completion
- 22 Dec 2026 (estimated)
- First posted
- 8 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PRIMARY OBJECTIVES:
- To assess the feasibility of using IMRT to deliver total body irradiation (TBI) to 800 cGy while keeping mean lung and kidney doses to 200 cGy.
II. To assess the safety/feasibility of total body irradiation using IMRT (IMRT TBI) in systemic sclerosis patients undergoing autologous hematopoietic stem cell transplantation.
SECONDARY OBJECTIVES:
- To evaluate dose homogeneity and dose sparing to lung and kidneys. II. To evaluate transplant-related mortality at 30 days and 100 days post IMRT TBI.
OUTLINE:
Patients undergo TBI using IMRT twice daily (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.
After completion of study treatment, patients are followed up on days 30 and 100.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (TBI using IMRT, cyclophosphamide, HSCT) | Experimental | Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Cyclophosphamide, Radiation: Intensity-Modulated Radiation Therapy, Procedure: Total-Body IrradiationPatients 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Hematopoietic Stem Cell TransplantationAlso: Allogeneic Hematopoietic Cell Transplantation, allogeneic stem cell transplantation, HSC, HSCTUndergo HSCT | Procedure or surgery | Treatment (TBI using IMRT, cyclophosphamide, HSCT) |
| CyclophosphamideAlso: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, CiclofosfamidaUndergo HSCT | Drug | Treatment (TBI using IMRT, cyclophosphamide, HSCT) |
| Intensity-Modulated Radiation TherapyAlso: IMRT, Intensity Modulated RT, Intensity-Modulated Radiotherapy, Radiation, Intensity-Modulated RadiotherapyUndergo TBI using IMRT | Radiation | Treatment (TBI using IMRT, cyclophosphamide, HSCT) |
| Total-Body IrradiationAlso: TBI, Total Body Irradiation, Whole Body Irradiation, Whole-Body IrradiationUndergo TBI using IMRT | Procedure or surgery | Treatment (TBI using IMRT, cyclophosphamide, HSCT) |
Primary outcome
Mean lung dose
Time frame: Up to day 100
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.
Mean kidney dose
Time frame: Up to day 100
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.
2 secondary outcomes
Dose homogeneity for lungs, kidneys, and total body
Time frame: Up to day 100
Transplant-related mortality
Time frame: Day 30 and day 100
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Y Wong principal investigator
City of Hope Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Intensity-Modulated Radiation Therapy in people with Systemic Scleroderma, run by City of Hope Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean lung dose (time frame: Up to day 100). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026.