ICTRP Search Portal

Main

Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	12 December 2020
Main ID:	NCT00114530
Date of registration:	15/06/2005
Prospective Registration:	No
Primary sponsor:	National Institute of Allergy and Infectious Diseases (NIAID)
Public title:	Scleroderma: Cyclophosphamide or Transplantation (SCOT)
Scientific title:	A Randomized, Open-Label, Phase II Multicenter Study of High-Dose Immunosuppressive Therapy Using Total Body Irradiation, Cyclophosphamide, ATGAM, and Autologous Transplantation With Auto-CD34+HPC Versus Intravenous Pulse Cyclophosphamide for the Treatment of Severe Systemic Sclerosis (SCSSc-01)
Date of first enrolment:	June 2005
Target sample size:	75
Recruitment status:	Completed
URL:	https://clinicaltrials.gov/show/NCT00114530
Study type:	Interventional
Study design:	Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Phase:	Phase 2/Phase 3

Countries of recruitment
Canada	United States

Contacts

Name:	Richard Nash, MD
Address:
Telephone:
Email:
Affiliation:	Fred Hutchinson Cancer Research Center

Name:	Daniel Furst, MD
Address:
Telephone:
Email:
Affiliation:	Rheumatology Division, UCLA Medical School

Name:	Leslie Crofford, MD
Address:
Telephone:
Email:
Affiliation:	University of Kentucky

Name:	Maureen Mayes, MD, MPH
Address:
Telephone:
Email:
Affiliation:	The University of Texas Health Science Center, Houston

Name:	Keith Sullivan, MD
Address:
Telephone:
Email:
Affiliation:	Division of Cellular Therapy, Duke University

Name:	Peter McSweeney, MD
Address:
Telephone:
Email:
Affiliation:	Blood and Marrow Transplant Program, Presbyterian/St. Luke's Medical Center, Rocky Mountain Cancer Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- Severe systemic sclerosis (SSc) as defined by the American College of Rheumatology
(ACR);

- SSc, including extensive skin and internal organ involvement involving either the
lungs or the kidneys, that threatens participant's life; and

- Willingness to use accepted methods of contraception for at least 15 months after
starting study treatment.

Exclusion Criteria:

- Lung, heart, liver, or kidney impairment that would interfere with the study or
compromise participant's survival;

- Active blood vessel dilation in the stomach (Active Gastric Antral Vascular
Ectasia/GAVE, also known as "watermelon stomach"). Patients found to have this
disorder at study screening can receive treatment outside the study and then be
re-screened. For more information about this study criterion, refer to the study
protocol.

- Previous treatment with cyclophosphamide, as defined by: a) prior IV cyclophosphamide
administration for more than 6 months OR a total cumulative IV dose greater than 3
g/m^2; b) prior oral cyclophosphamide administration for more than 4 months,
regardless of dose; or c) combination of prior oral and IV cyclophosphamide
administration for more than 6 months, independent of dose.

- Steroid therapy at doses of greater than 10 mg/day, or more than 2 pulses for
concurrent illnesses within prior 12 months;

- Unwillingness or inability to discontinue certain disease-modifying antirheumatic
drugs (DMARDs) for the treatment of SSc;

- Presence of clinically significant rheumatic diseases other than scleroderma requiring
significant immunosuppression;

- Any active uncontrolled infection that would interfere with high-dose therapy or pulse
cyclophosphamide regimens:

- Hepatitis B virus infected

- Hepatitis C virus infected or

- HIV infected.

- Blood abnormalities;

- Diagnosis of cancer within 2 years prior to study entry. Participants with adequately
treated squamous cell skin cancer, basal cell carcinoma, and carcinoma in situ are not
excluded.

- Other comorbid illnesses with an estimated life expectancy of less than 5 years;

- Defective formation of bone marrow cells (myelodysplasia);

- Uncontrolled hypertension;

- History of hypersensitivity to murine or Escherichia coli (e.g., E. coli) proteins;
History of noncompliance with prior medical care;

- History of substance abuse within 5 years prior to study entry; or

- Pregnancy.

Age minimum: 18 Years
Age maximum: 69 Years
Gender: All

Health Condition(s) or Problem(s) studied

Scleroderma, Systemic

Autoimmune Disease

Sclerosis

Intervention(s)

Drug: cyclophosphamide

Biological: mHSCT

Primary Outcome(s)

Global Rank Composite Score (GRCS) (Month 54, ITT) [Time Frame: 54 Months Post-Randomization]

Secondary Outcome(s)

Change From Baseline to Month 54 in Short Form 36 Health Survey (SF-36) (PP) [Time Frame: 54 Months Post-Randomization]

Documented Myositis (PP) [Time Frame: 54 Months Post-Randomization]

Regimen-Related Toxicities [Time Frame: Randomization through end of study follow-up (up to Month 72 post-randomization)]

All-Cause Mortality (Month 48, PP) [Time Frame: 48 Months Post-Randomization]

All-Cause Mortality (Month 54, ITT) [Time Frame: 54 Months Post-Randomization]

All-Cause Mortality (Month 54, PP) [Time Frame: 54 Months Post-Randomization]

Event-Free Survival (EFS) (Month 54, ITT) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Modified Rodnan Skin Score (mRSS) (ITT) [Time Frame: 54 Months Post-Randomization]

Global Rank Composite Score (GRCS) (Month 48, ITT) [Time Frame: 48 Months Post-Randomization]

Event-Free Survival (EFS) (Month 54, PP) [Time Frame: 54 Months Post-Randomization]

New or Worsening Pulmonary Hypertension (PP) [Time Frame: 54 Months Post-Randomization]

All-Cause Mortality (Month 48, ITT) [Time Frame: 48 Months Post-Randomization]

Event-Free Survival (EFS) (Month 48, ITT) [Time Frame: 48 Months Post-Randomization]

Global Rank Composite Score (GRCS) (Month 54, PP) [Time Frame: 54 Months Post-Randomization]

New or Worsening Pulmonary Hypertension (ITT) [Time Frame: 54 Months Post-Randomization]

Number of Subjects With Regimen-Related Toxicities [Time Frame: Randomization through end of study follow-up (up to Month 72 post-randomization).]

Change From Baseline to Month 54 in Modified Rodnan Skin Score (mRSS) (PP) [Time Frame: 54 Months Post-Randomization]

Event-Free Survival (EFS) (Month 48, PP) [Time Frame: 48 Months Post-Randomization]

Time to Absolute Neutrophil Count Engraftment [Time Frame: 28 days post-transplant]

Treatment-Related Mortality (Month 48, ITT) [Time Frame: 48 Months Post-Randomization]

Infectious Complications [Time Frame: Randomization through end of study follow-up (up to Month 72 post-randomization).]

Change From Baseline to Month 54 in Diffusion in Liters of Carbon Monoxide (DLCO) (PP) [Time Frame: 54 Months Post-Randomization]

Occurrence of Scleroderma Renal Crisis (ITT) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Health Assessment Questionnaire - Disability Index (HAQ-DI) (PP) [Time Frame: 54 Months Post-Randomization]

Initiating Use of Disease-Modifying Antirheumatic Drugs (DMARDs) by Month 54 (ITT) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Forced Vital Capacity (FVC) (PP) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Health Assessment Questionnaire - Disability Index (HAQ-DI) (ITT) [Time Frame: 54 Months Post-Randomization]

Global Rank Composite Score (GRCS) (Month 48, PP) [Time Frame: 48 Months Post-Randomization]

Initiating Use of Disease-Modifying Antirheumatic Drugs by Month 54 (DMARDs) (PP) [Time Frame: 54 Months Post-Randomization]

Treatment-Related Mortality (Month 54, ITT) [Time Frame: 54 Months Post-Randomization]

Number of Subjects With Infectious Complications [Time Frame: Randomization through end of study follow-up (up to Month 72 post-randomization).]

New or Worsening Arrhythmias, Congestive Heart Failure, or Pericardial Effusion (ITT) [Time Frame: 54 Months Post-Randomization]

New or Worsening Arrhythmias, Congestive Heart Failure, or Pericardial Effusion (PP) [Time Frame: 54 Months Post-Randomization]

Occurrence of Scleroderma Renal Crisis (PP) [Time Frame: 54 Months Post-Randomization]

Treatment-Related Mortality (Month 54, PP) [Time Frame: 54 Months Post-Randomization]

Treatment-Related Mortality (Month 48, PP) [Time Frame: 48 Months Post-Randomization]

Change From Baseline to Month 54 in Diffusion in Liters of Carbon Monoxide (DLCO) (ITT) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Forced Vital Capacity (FVC) (ITT) [Time Frame: 54 Months Post-Randomization]

Change From Baseline to Month 54 in Short Form 36 Health Survey (SF-36) (ITT) [Time Frame: 54 Months Post-Randomization]

Documented Myositis (ITT) [Time Frame: 54 Months Post-Randomization]

Secondary ID(s)

DAIT SCSSc-01

SCOT

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:	Yes
Date Posted:	14/07/2017
Date Completed:
URL:	https://clinicaltrials.gov/ct2/show/results/NCT00114530

Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.