Metabolism, Inborn ErrorsNCT00010361
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
A study testing cyclosporine, fludarabine and mycophenolate mofetil in people with Metabolism, Inborn Errors and Granulomatous Disease, Chronic, run by Fred Hutchinson Cancer Center at 1 site in United States.
Trial summary
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with inherited disorders. II. Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Metabolism, Inborn Errors, Granulomatous Disease, Chronic
- What is being tested?
- cyclosporine, fludarabine and mycophenolate mofetil
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2000to Apr 2007
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 Up to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Inherited disorders treatable with allogeneic peripheral blood or bone marrow transplantation At high risk for regimen related toxicity with a conventional transplant
- No severe CNS involvement of disease, defined by IQ score less than 70
- HLA matched donor Sibling donors must be a confirmed match at HLA-A, B, and DRB1 Other related and non-related donors must be matched at HLA-A, B, C, DRB1, and DQB1 A donor homozygous for one allele only at HLA-A, B, C, DRB1, or DQB1 allowed (1 antigen mismatch for graft-versus-host disease, 0 antigen mismatch for graft-rejection)
--Prior/Concurrent Therapy--
- No concurrent growth factors with mycophenolate mofetil
--Patient Characteristics--
- Age: Under 55
- Performance status: Not specified
- Life expectancy: At least 100 days
- Hematopoietic: Not specified
- Hepatic: No evidence of synthetic dysfunction No severe cirrhosis
- Renal: Not specified
- Cardiovascular: LVEF at least 30% No poorly controlled hypertension on multiple antihypertensives
- Other: No organ dysfunction that would preclude survival Not pregnant or nursing Fertile patients must use effective contraception during and for 12 months following study
Trial locations and contacts
1 site in 1 country.
United States1 site
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
About this study
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with inherited disorders.
II. Determine whether this regimen can establish mixed chimerism in these patients.
III. Determine whether mixed chimerism is sufficient to reverse disease symptoms in these patients.
IV. Determine the safety of donor lymphocyte infusions to eliminate persistent disease in these patients with mixed chimerism.
Read the detailed description
PROTOCOL OUTLINE: Patients receive fludarabine IV over 2 hours on days -4 to -2 followed by total body irradiation and peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive oral or IV cyclosporine 2-3 times daily on days -3 to 50 (related donor) or 100 (unrelated donor) and oral mycophenolate mofetil twice daily on days 0 to 28 (related donor) or 40 (unrelated donor).
Patients may also receive donor lymphocyte infusion for continued treatment of symptoms in the event of mixed chimerism and in the absence of graft-versus-host disease.
Patients are followed weekly for 1 month, monthly for 2 years, and then annually thereafter.
- Being tested
- cyclosporine, fludarabine, mycophenolate mofetil
- Medical subject headings
- Metabolism, Inborn Errors, Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclosporineDrug
fludarabineDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2001 (estimated)
- Study startNov 2000
- Primary completionApr 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metabolism, Inborn ErrorsStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer 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
- Phase N/A
- Primary purpose
- Treatment
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Investigators
- Ann Woolfrey, Study chair, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00010361
- Sponsor's protocol ID
- 199/15577
- Other IDs
- FHCRC-1475.00
- Status last verified
- Aug 2006
- First submitted
- 2 Feb 2001
- Last updated
- 9 Dec 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
- ClinicalTrials.gov ID
- NCT00010361
- Acronym / sponsor ID
- 199/15577
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Study start
- Nov 2000
- Primary completion
- Apr 2007 (actual)
- First posted
- 2 Feb 2001 (estimated)
- 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.
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with inherited disorders.
II. Determine whether this regimen can establish mixed chimerism in these patients.
III. Determine whether mixed chimerism is sufficient to reverse disease symptoms in these patients.
IV. Determine the safety of donor lymphocyte infusions to eliminate persistent disease in these patients with mixed chimerism.
Detailed description
PROTOCOL OUTLINE: Patients receive fludarabine IV over 2 hours on days -4 to -2 followed by total body irradiation and peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive oral or IV cyclosporine 2-3 times daily on days -3 to 50 (related donor) or 100 (unrelated donor) and oral mycophenolate mofetil twice daily on days 0 to 28 (related donor) or 40 (unrelated donor).
Patients may also receive donor lymphocyte infusion for continued treatment of symptoms in the event of mixed chimerism and in the absence of graft-versus-host disease.
Patients are followed weekly for 1 month, monthly for 2 years, and then annually thereafter.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 55 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 |
|---|---|---|
| cyclosporine | Drug | – |
| fludarabine | Drug | – |
| mycophenolate mofetil | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2014.
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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.
Ann Woolfrey study chair
Fred Hutchinson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing cyclosporine, fludarabine and mycophenolate mofetil in people with Metabolism, Inborn Errors and Granulomatous Disease, Chronic, run by Fred Hutchinson Cancer 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?
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 9 Dec 2014. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
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 9 Dec 2014.