Fanconi's AnemiaPhase 1NCT00006127
Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
A Phase 1 study testing amifostine in people with Fanconi's Anemia, run by Dana-Farber Cancer Institute at 1 site in United States.
Trial summary
OBJECTIVES: I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia. II. Determine the efficacy of this treatment regimen in this patient population. III. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Fanconi's Anemia
- What is being tested?
- amifostine
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2000to not given
- Phase
- Phase 1
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of Fanconi's anemia by conventional diepoxybutane or mitomycin sensitivity assays, or molecular testing
Bone marrow failure verified on at least 3 occasions in the preceding 3 months by any one of the following: hemoglobin less than 8 g/dL; absolute neutrophil count less than 1,000/mm3; platelet count less than 30,000/mm3; symptomatic bone marrow failure (e.g., exercise limitation from anemia or spontaneous bleeding from thrombocytopenia)
Evidence consistent with myelodysplastic syndrome allowed if less than 5% blasts on bone marrow aspiration; clonality on bone marrow cytogenetic analysis OR morphological changes on bone marrow aspirate
Refusal of or unsuccessful with prior conventional therapies
--Prior/Concurrent Therapy--
Biologic therapy: No prior bone marrow transplantation; no concurrent hematopoietic growth factors
Endocrine therapy: No concurrent androgens
--Patient Characteristics--
Hepatic: Bilirubin no greater than 2 times normal AST no greater than 3 times normal ALT no greater than 6 times normal
Renal: Creatinine no greater than 2 times normal
Other: No prior malignancy; no active bacterial, viral, or fungal infection requiring therapy other than prophylaxis; not pregnant; negative pregnancy test
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
OBJECTIVES:
- Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia.
II. Determine the efficacy of this treatment regimen in this patient population.
III. Evaluate the effect of this treatment regimen on bone marrow progenitor cell proliferation and peripheral blood mononuclear cell apoptosis in these patients.
Read the detailed description
PROTOCOL OUTLINE:
This is a dose escalation study.
Patients receive amifostine IV over 3-5 minutes three times a week for three weeks.
Cohorts of 3 patients receive one of three dose levels of amifostine. The maximum tolerated dose is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.
Patients are followed weekly for 3 weeks.
- Conditions
- Fanconi's Anemia
- Being tested
- amifostine
- Keywords
- Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease
- Medical subject headings
- Fanconi Anemia, Anemia, Aplastic, Hematologic Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
amifostineDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2000 (estimated)
- Study startApr 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi's AnemiaStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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 1
- Primary purpose
- Treatment
- Enrolment
- 21 participants
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Investigators
- W. Nicholas Haining, Study chair, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00006127
- Sponsor's protocol ID
- 199/15281
- Other IDs
- DFCI-9910170, ALZA-99-004-ii
- Status last verified
- Oct 2003
- First submitted
- 3 Aug 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
- ClinicalTrials.gov ID
- NCT00006127
- Acronym / sponsor ID
- 199/15281
- Conditions
- Fanconi's Anemia
- Keywords
- Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Study start
- Apr 2000
- First posted
- 4 Aug 2000 (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:
- Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia.
II. Determine the efficacy of this treatment regimen in this patient population.
III. Evaluate the effect of this treatment regimen on bone marrow progenitor cell proliferation and peripheral blood mononuclear cell apoptosis in these patients.
Detailed description
PROTOCOL OUTLINE:
This is a dose escalation study.
Patients receive amifostine IV over 3-5 minutes three times a week for three weeks.
Cohorts of 3 patients receive one of three dose levels of amifostine. The maximum tolerated dose is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.
Patients are followed weekly for 3 weeks.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older 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 |
|---|---|---|
| amifostine | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
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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.
W. Nicholas Haining study chair
Dana-Farber Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing amifostine in people with Fanconi's Anemia, run by Dana-Farber Cancer Institute 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: status not confirmed, as of the sponsor's last update on 24 Jun 2005. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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 24 Jun 2005.