Fanconi's AnemiaPhase 1NCT00005896
Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
A Phase 1 study testing filgrastim, Autologous stem cells transduced with FACC retroviral vector and Autologous Stem Cell Transplantation in people with Fanconi's Anemia, run by University of Minnesota.
Trial summary
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C. II. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Fanconi's Anemia
- What is being tested?
- filgrastim, Autologous stem cells transduced with FACC retroviral vector and Autologous Stem Cell Transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Mar 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Fanconi's anemia, complementation group C (FACC) Confirmed by diepoxybutane or mitomycin testing AND DNA analysis indicating FACC mutations
- Patients at least 25 kg weight
- No acute leukemia OR Bone marrow aspirate with greater than 10% blasts
- No patients who elect bone marrow transplantation
--Prior/Concurrent Therapy--
- At least 14 days since prior therapy for any acute viral, bacterial, or fungal infection
--Patient Characteristics--
- Performance status: Karnofsky 40-100%
- Hepatic: SGOT, SGPT, and alkaline phosphatase no greater than 5 times upper limit of normal (ULN) PT/PTT no greater than 1.5 times ULN Serum amylase no greater than 1.5 times ULN Bilirubin no greater than 2.5 mg/dL Triglyceride less than 400 mg/dL
- Renal: Creatinine clearance greater than 50 mL/min
- Cardiovascular: Normal cardiac function No ischemic heart disease that may be considered an anesthetic or operative risk
- Pulmonary: No lung disease that may be considered an anesthetic or operative risk Resting transcutaneous oxygen saturation greater than 90% on room air
- Other: HIV negative Hepatitis B surface antigen negative No underlying condition that may preclude study therapy (e.g., allergies to study reagents)
- No acute viral, bacterial, or fungal infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception
About this study
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C.
II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients.
III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.
Read the detailed description
PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every 2 months for up to 4 courses total.
Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for the next year, and then yearly thereafter.
- Conditions
- Fanconi's Anemia
- Being tested
- filgrastim, Autologous stem cells transduced with FACC retroviral vector, Autologous Stem Cell Transplantation
- 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.
filgrastimDrug
Autologous stem cells transduced with FACC retroviral vectorGenetic
Autologous Stem Cell TransplantationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2000 (estimated)
- Study startMar 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 University of MinnesotaThe 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
- 6 participants
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Investigators
- John E. Wagner, Jr., Study chair, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00005896
- Sponsor's protocol ID
- 199/15107
- Other IDs
- UMN-MT-1997-10, UNM-MT-9710
- Status last verified
- Mar 2001
- First submitted
- 2 Jun 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 Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
- ClinicalTrials.gov ID
- NCT00005896
- Acronym / sponsor ID
- 199/15107
- 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
- 6 participants
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Study start
- Mar 2000
- First posted
- 5 Jun 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: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C.
II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients.
III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.
Detailed description
PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every 2 months for up to 4 courses total.
Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for the next year, and then yearly thereafter.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 5 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 |
|---|---|---|
| filgrastim | Drug | – |
| Autologous stem cells transduced with FACC retroviral vector | Genetic | – |
| Autologous Stem Cell Transplantation | Procedure or surgery | – |
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.
John E. Wagner, Jr. study chair
Masonic Cancer Center, University of Minnesota
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing filgrastim, Autologous stem cells transduced with FACC retroviral vector and Autologous Stem Cell Transplantation in people with Fanconi's Anemia, run by University of Minnesota.
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.
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.