Completed

A Phase II Trial Of Intravenous Pentostatin For The Treatment Of Patients With Refractory Chronic Graft-Versus-Host Disease

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What is being tested

pentostatin

Drug
Who is being recruted

Bronchiolitis Obliterans Syndrome
+8

+ Organizing Pneumonia
+ Bronchial Diseases
Over 18 Years
See all eligibility criteria
How is the trial designed

Supportive Care Study

Phase 2
Interventional
Study Start: December 2003
See protocol details

Summary

Principal SponsorAlliance for Clinical Trials in Oncology
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: December 1, 2003Actual date on which the first participant was enrolled.

OBJECTIVES: Primary * Determine the response rate in patients with refractory chronic graft-versus-host disease treated with pentostatin. Secondary * Determine the time to next immunosuppressive agent (i.e., the time to progression from best response) in patients treated with this drug. * Determine the toxicity of this drug in these patients. * Determine the infection rate in patients treated with this drug. * Determine the pharmacokinetics of this drug in these patients. * Determine the changes in lymphocyte populations in patients treated with this drug. * Determine the survival of patients treated with this drug. OUTLINE: This is a multicenter study. Patients receive pentostatin IV over 20-30 minutes on day 1. Treatment repeats every 14 days for 6 courses in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response after 6 courses receive 4 additional courses. Patients who achieve a partial response, minor response, or stable disease after 6 courses may receive up to 6 additional courses. Patients are followed every 4 weeks for 1 year, every 3 months for 2 years, and then annually for 5 years. PROJECTED ACCRUAL: Approximately 37 patients will be accrued for this study.

Official TitleA Phase II Trial Of Intravenous Pentostatin For The Treatment Of Patients With Refractory Chronic Graft-Versus-Host Disease 
NCT00074035
Principal SponsorAlliance for Clinical Trials in Oncology
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
39 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Supportive Care Study
These studies explore ways to improve comfort and daily life for people living with a condition. They may focus on easing symptoms, reducing treatment side effects, or supporting overall well-being.

How participants are assigned to different groups/arms
In this clinical study, all participants receive the same treatment. Since there is only one group, there is no need for randomization or assignment to different arms. This type of study is often used to test a new treatment without comparing it to another.

Other Ways to Assign Participants
Randomized allocation
: Participants are assigned randomly, like flipping a coin, to ensure fairness and reduce bias.

Non-randomized allocation
: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

How treatments are given to participants
In this study, all participants receive the same treatment. This approach is often used to evaluate the effects of a single intervention without comparing it to another.

Other Ways to Assign Treatments
Parallel assignment
: Participants are split into separate groups, each receiving a different treatment.

Cross-over assignment
: Participants switch between treatments during the study.

Factorial assignment
: Participants receive different combinations of treatments.

Sequential assignment
: Participants receive treatments one after another in a specific order, possibly based on individual responses.

Other assignment
: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a non placebo-controlled study, no participants receive an inert substance (placebo) to compare outcomes. Instead, all participants receive either the experimental treatment or an alternative treatment (often the Standard of Care). This method allows researchers to compare the effects of the experimental treatment with those of a different active intervention, rather than a placebo.

Other Options
Placebo-Controlled
: A placebo is used to compare the effects of the experimental treatment with those of an inert substance, isolating the true treatment effect.

How the interventions assigned to participants is kept confidential
Everyone involved in the study knows which treatment is being given. This is typically used when it's not possible or necessary to hide the treatment details from participants or researchers.

Other Ways to Mask Information
Single-blind
: Participants do not know which treatment they are receiving, but researchers do.

Double-blind
: Neither participants nor researchers know which treatment is given.

Triple-blind
: Participants, researchers, and outcome assessors do not know which treatment is given.

Quadruple-blind
: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
Over 18 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Bronchiolitis Obliterans Syndrome
Organizing Pneumonia
Bronchial Diseases
Bronchiolitis
Bronchiolitis Obliterans
Bronchitis
Graft vs Host Disease
Immune System Diseases
Lung Diseases
Lung Diseases, Obstructive
Respiratory Tract Diseases
Criteria

1. Histologic documentation of chronic GvHD following allogeneic HCT or donor lymphocyte infusion. 2. Patients may have progressive, quiescent, or de novo onset chronic GvHD. 3. Patients with extensive stage chronic GvHD requiring systemic immunosuppressive therapy are eligible. Patients with limited stage disease are excluded. Extensive stage is defined according to Seattle criteria (9) as either: * Generalized skin involvement or * Limited skin involvement or hepatic involvement with any one of the following: * Liver histology showing chronic progressive hepatitis, bridging necrosis or cirrhosis * Eye involvement (Schirmer's test with \< 5 mm wetting) * Involvement of minor salivary glands or oral mucosa * Involvement of any other organ 4. Patients must have failed treatment with, or experience progression after, prior corticosteroids for extensive stage chronic GvHD, as defined below. 4.1 Patients will be considered to have failed corticosteroids if they have any one of the following criteria: * Progressive disease or less than a minor response in any organ system despite 2 weeks on corticosteroid treatment at least 1 mg/kg methylprednisolone or equivalent. * Failure to achieve at least a minor response after at least 4 weeks of treatment with a dose of ≥ 0.5 mg/kg methylprednisolone or equivalent. * Achievement of less than a partial response at 8 weeks of corticosteroid treatment despite use of a dose ≥ 0.5 mg/kg methylprednisolone or equivalent. * Requirement of ≥ 0.5 mg/kg methylprednisolone or equivalent to maintain a partial response or better at 12 weeks of corticosteroid treatment. * Requirement of \> 10 mg/kg methylprednisolone or equivalent to maintain a partial response or better at 18 weeks of corticosteroid treatment. 4.2 Patients with progression of extensive stage chronic GvHD after a prior history of treatment with at least 18 weeks of corticosteroids, now requiring the reintroduction of corticosteroids (\> 10 mg/day methylprednisolone or equivalent) or an additional agent (including photopheresis, PUVA) for treatment. 5. Patients with established chronic GvHD not improving or progressing on other immunosuppressive agents are also eligible if steroid refractoriness has been established previously. 6. Age ≥ 18 years 7. Performance Status 0-3 8. Patients on mechanical ventilation are excluded. 9. No active infection. Patients with active infection requiring antibiotic therapy are not eligible until infection is controlled. 10. No HIV infection. Patients with HIV infection are excluded because of safety concerns in this patient population. 11. Non-pregnant and non-nursing. Women and men of reproductive potential should agree to use an appropriate method of birth control throughout their participation in this study due to the teratogenic potential of the therapy utilized in this trial (although it is unlikely that successful pregnancy will occur in patients with chronic GvHD). Appropriate methods of birth control include oral contraceptives, implantable hormonal contraceptives (Norplant®), or double barrier method (diaphragm plus condom). 12. Required Initial Laboratory Values: * Calc. Creatinine Clearance ≥ 30 mL/min/1.73 m\^2 * ANC \> 1000/μL * Platelets \> 50,000/μL without transfusion


Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
One single intervention group 

is designated in this study

This study does not include a placebo group 

Treatment Groups
Group I
Experimental
treatment of pts with refractory graft vs host disease

4 mg/sq m IV infusion over 20-30 min q 2 weeks
Study Objectives
Primary Objectives

Percentage of participants who had a complete or partial response defined by the Hopkins scoring system. A complete response is defined as the disappearance of signs and symptoms of chronic GVHD in all involved systems that is sustained for at lest 4 weeks. A partial response is an improvement by 2 or more points in at least one system score, which is sustained for at least 4 weeks, with no signs of worsening in others.
Secondary Objectives

Number of participants experiencing a grade 3, 4 or 5 clinically significant non-hematologic adverse events, at least possibly related to treatment.

Percentage of patients who were alive at 1 year.

Percentage of patients who were alive at 2 years.

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 16 locations
Suspended
Tunnell Cancer Center at Beebe Medical CenterLewes, United StatesSee the location
Suspended
CCOP - Christiana Care Health ServicesNewark, United States
Suspended
University of Illinois Cancer CenterChicago, United States
Suspended
University of Chicago Cancer Research CenterChicago, United States

Completed16 Study Centers
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