DiarrheaPhase 2NCT00258180
Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
A Phase 2 study testing filgrastim and cyclophosphamide in people with Diarrhea and Gastrointestinal Complications, run by Johns Hopkins University at 1 site in United States.
Trial summary
RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy . PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe autoimmune enteropathy. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Diarrhea, Gastrointestinal Complications, Unspecified Childhood Solid Tumor, Protocol Specific
- What is being tested?
- filgrastim and cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Aug 2005to 24 Feb 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of severe autoimmune enteropathy
- Condition is resistant to conventional therapy
- Histologic evidence of severe villous atrophy with intense lymphocytic infiltrate of the lamina propria by small intestinal biopsy within the past 3 months
- Disease failed to respond after ≥ 2 months of corticosteroid therapy at a dose of ≥ 0.5 mg/kg/day or ≥ 40 mg/day for patients > 20 kg AND 1 of the following therapies:
- Cyclosporine resulting in ≥ 1 whole blood level of > 200 ng/mL
- Tacrolimus resulting in ≥ 1 whole blood level of 5 ng/mL
- At least 50% estimated caloric needs provided by parenteral nutrition
- History of intractable diarrhea, defined as frequent watery stools for > 3 months that does not respond to dietary restriction
- No celiac disease, defined by a history of positive antiendomysial antibody or tissue transglutaminase antibody
- No primary immunodeficiency or x-linked autoimmunity-allergy dysregulation
PATIENT CHARACTERISTICS:
Performance status
- Lansky 60-100%
Life expectancy
- Not specified
Hematopoietic
- Not specified
Hepatic
- Not specified
Renal
- Not specified
Cardiovascular
- Ejection fraction ≥ 40% OR shortening fraction ≥ 20%
Pulmonary
- FVC or FEV_1 ≥ 50% of predicted (for patients > 8 years of age)
- No clinically abnormal pulmonary function or abnormal pulse oximetry (for patients ≤ 8 years of age)
Other
- Not pregnant
- Negative pregnancy test
- Fertile patients must use effective contraception during and for at least 9 months after completion of study treatment
- No known chromosomal abnormality
PRIOR CONCURRENT THERAPY:
Biologic therapy
- No immunizations for at least 6 months after completion of study treatment
Endocrine therapy
- See Disease Characteristics
- At least 5 days since prior corticosteroids
- No concurrent dexamethasone as an anti-emetic
Other
- At least 5 days since other prior immunosuppressive medications (e.g., tacrolimus or cyclosporine)
Trial locations and contacts
1 site in 1 country.
United States1 site
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21231-2410
About this study
Official title: High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy .
PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe autoimmune enteropathy.
Read the detailed description
OBJECTIVES:
Primary
- Determine the rate of treatment-free remission in young patients with severe autoimmune enteropathy treated with high-dose cyclophosphamide.
Secondary
- Determine the toxic effects of this drug in these patients.
OUTLINE: Patients receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
After completion of study treatment, patients are followed periodically for up to 1½ years.
PROJECTED ACCRUAL: A total of 7-11 patients will be accrued for this study.
- Conditions
- Diarrhea, Gastrointestinal Complications, Unspecified Childhood Solid Tumor, Protocol Specific
- Being tested
- filgrastim, cyclophosphamide
- Keywords
- unspecified childhood solid tumor, protocol specific, gastrointestinal complications, diarrhea
- Medical subject headings
- Diarrhea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Also called G-CSF
Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover
cyclophosphamideDrug
Administered IV over 1 hour on days 1-4
| Study group | Role | What they receive |
|---|---|---|
| severe autoimmune enteropathy | Experimental | Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover Receives: Biological: filgrastim; Drug: cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-free Remission at 1 Year After Study Completion
Number of participants off therapy 1 year after study completion without relapse.
Measured: 1 year
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2005 (estimated)
- Study start15 Aug 2005
- Primary completion24 Feb 2009
- Study completion24 Feb 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for DiarrheaStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe 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
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- David M. Loeb, MD, PhD, Principal investigator, Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsMaria Oliva-Hemker, MD, Principal investigator, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00258180
- Sponsor's protocol ID
- 03-07-08-04
- Other IDs
- P30CA006973, JHOC-J0326, J0326, CDR0000441133
- Status last verified
- Mar 2019
- First submitted
- 22 Nov 2005
- Last updated
- 16 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
- ClinicalTrials.gov ID
- NCT00258180
- Acronym / sponsor ID
- 03-07-08-04
- Conditions
- Diarrhea, Gastrointestinal Complications, Unspecified Childhood Solid Tumor, Protocol Specific
- Keywords
- unspecified childhood solid tumor, protocol specific, gastrointestinal complications, diarrhea
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 15 Aug 2005 (actual)
- Primary completion
- 24 Feb 2009 (actual)
- Study completion
- 24 Feb 2009 (actual)
- First posted
- 24 Nov 2005 (estimated)
- Results
- Posted 16 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy .
PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe autoimmune enteropathy.
Detailed description
OBJECTIVES:
Primary
- Determine the rate of treatment-free remission in young patients with severe autoimmune enteropathy treated with high-dose cyclophosphamide.
Secondary
- Determine the toxic effects of this drug in these patients.
OUTLINE: Patients receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
After completion of study treatment, patients are followed periodically for up to 1½ years.
PROJECTED ACCRUAL: A total of 7-11 patients will be accrued for this study.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| severe autoimmune enteropathy | Experimental | Biological: filgrastim, Drug: cyclophosphamideYoung patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| filgrastimAlso: G-CSFAdministered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover | Biological | severe autoimmune enteropathy |
| cyclophosphamideAdministered IV over 1 hour on days 1-4 | Drug | severe autoimmune enteropathy |
Primary outcome
Number of Participants With Treatment-free Remission at 1 Year After Study Completion
Time frame: 1 year
Number of participants off therapy 1 year after study completion without relapse.
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month
Time frame: 1 month
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
David M. Loeb, MD, PhD principal investigator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Maria Oliva-Hemker, MD principal investigator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim and cyclophosphamide in people with Diarrhea and Gastrointestinal Complications, run by Johns Hopkins University 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?
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: finished, as of the sponsor's last update on 16 Apr 2019. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Treatment-free Remission at 1 Year After Study Completion (time frame: 1 year). 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 16 Apr 2019.