Chronic Myeloproliferative DisordersNCT00900055
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
An observational study of Chronic Myeloproliferative Disorders and Fanconi Anemia, run by OHSU Knight Cancer Institute at 1 site in United States.
Trial summary
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of blood cancers. Read more about this study
- Who is it for?
- Ages 1 year to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm
- What is being tested?
- microarray analysis, polyacrylamide gel electrophoresis and polymerase chain reaction
- How many people
- 213 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 1975to 23 Aug 2016
- Phase
- Observational
- 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 1 year to 55 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: The Center for Hematologic Malignancies, the Knight Cancer Institute at OHSU, and the Bone Marrow Failure clinic at Doernbecher Children's Hospital at OHSU will be centers for recruitment.
DISEASE CHARACTERISTICS:
- Meets 1 of the following criteria:
- Diagnosis of one of the following:
- Fanconi's anemia requiring bone marrow biopsy as part of standard care (adults and children)
- Myeloproliferative disorder or myeloma (adults)
- Healthy volunteer, meeting 1 of the following criteria:
- Over 18 years of age
- Bone marrow transplant donor (children)
PATIENT CHARACTERISTICS:
- Hemoglobin > 13 g/dL
- White blood cells (WBC) > 4,000/mm³
- Platelet count > 150,000/mm³
- No clinical signs or symptoms of acute or subacute infections (viral, bacterial, or fungal)
- No known blood abnormality (healthy volunteers)
- No allergies to lidocaine or xylocaine
PRIOR CONCURRENT THERAPY:
- Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
Knight Cancer Institute at Oregon Health and Science University
Portland, Oregon, 97239-3098
About this study
Official title: Dysregulation of Hematopoiesis in Fanconi Anemia
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of blood cancers.
PURPOSE: The purpose of this study is to analyze in the laboratory blood and bone marrow cells from healthy volunteers or patients with Fanconi anemia, myeloproliferative disorders, or myeloma.
Read the detailed description
OBJECTIVES:
- Identify the specific molecular function of the Fanconi anemia (FA) complementing gene products in hematopoietic progenitor cells from patients and normal volunteers.
- Identify functional defects in hematopoietic stromal cells, including macrophages, from patients with FA, and selected blood cancers as well as normal volunteers.
OUTLINE: Peripheral blood mononuclear leukocytes, skin fibroblasts, and marrow fibroblasts are collected for loss-of-function and gain-of-function analysis related to the Fanconi anemia complementing gene.
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Being tested
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction, protein expression analysis, reverse transcriptase-polymerase chain reaction
- Keywords
- Fanconi anemia, multiple myeloma and other plasma cell neoplasms, chronic myeloproliferative disorders, myelodysplastic/myeloproliferative neoplasms
- Medical subject headings
- Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic-Myeloproliferative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
microarray analysisGenetic
polyacrylamide gel electrophoresisGenetic
polymerase chain reactionGenetic
protein expression analysisGenetic
reverse transcriptase-polymerase chain reactionGenetic
western blottingGenetic
chromatographyOther
high performance liquid chromatographyOther
immunoenzyme techniqueOther
What is being measured
The primary outcome is the main question the study is designed to answer.
Loss of function analyses
Measured: Duration of the study
Proteins binding to Fanconi anemia, complementation group C (FACC) gene-product by affinity chromatography of nuclear and whole cell lysates of normal cells
Measured: Duration of the study
Screening of proteins binding to FACC gene-product using monoclonal antibodies specific to signal transduction and cell cycle proteins
Measured: Duration of the study
Microsequencing of unique proteins
Measured: Duration of the study
Location of specific downstream block point imposed by antisense molecules using antibodies specific to signal transduction, cell cycle, or repair proteins for the FACC protein
Measured: Duration of the study
Affirmation that the block points identified are recapitulated in progenitor cells from peripheral blood
Measured: Duration of the study
Identification of functional defects in Fanconi anemia hematopoietic stromal cells
Measured: Duration of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2009 (estimated)
- Study startJun 1975
- Primary completion23 Aug 2016
- Study completion23 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by OHSU Knight 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 213 participants (actual)
Sponsor and investigators
- Lead sponsor
- OHSU Knight Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Laura Newell, OHSU Knight Cancer Institute
- Investigators
- Laura Newell, MD, Principal investigator, OHSU Knight Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00900055
- Sponsor's protocol ID
- IRB00000823
- Other IDs
- P01HL048546, OHSU-HEM-98030-L
- Status last verified
- Jun 2022
- First submitted
- 9 May 2009
- Last updated
- 5 Jul 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dysregulation of Hematopoiesis in Fanconi Anemia
- ClinicalTrials.gov ID
- NCT00900055
- Acronym / sponsor ID
- IRB00000823
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Keywords
- Fanconi anemia, multiple myeloma and other plasma cell neoplasms, chronic myeloproliferative disorders, myelodysplastic/myeloproliferative neoplasms
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 213 participants (actual)
- Lead sponsor
- OHSU Knight Cancer InstituteUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Jun 1975
- Primary completion
- 23 Aug 2016 (actual)
- Study completion
- 23 Aug 2016 (actual)
- First posted
- 12 May 2009 (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.
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of blood cancers.
PURPOSE: The purpose of this study is to analyze in the laboratory blood and bone marrow cells from healthy volunteers or patients with Fanconi anemia, myeloproliferative disorders, or myeloma.
Detailed description
OBJECTIVES:
- Identify the specific molecular function of the Fanconi anemia (FA) complementing gene products in hematopoietic progenitor cells from patients and normal volunteers.
- Identify functional defects in hematopoietic stromal cells, including macrophages, from patients with FA, and selected blood cancers as well as normal volunteers.
OUTLINE: Peripheral blood mononuclear leukocytes, skin fibroblasts, and marrow fibroblasts are collected for loss-of-function and gain-of-function analysis related to the Fanconi anemia complementing gene.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- The Center for Hematologic Malignancies, the Knight Cancer Institute at OHSU, and the Bone Marrow Failure clinic at Doernbecher Children's Hospital at OHSU will be centers for recruitment.
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 |
|---|---|---|
| microarray analysis | Genetic | – |
| polyacrylamide gel electrophoresis | Genetic | – |
| polymerase chain reaction | Genetic | – |
| protein expression analysis | Genetic | – |
| reverse transcriptase-polymerase chain reaction | Genetic | – |
| western blotting | Genetic | – |
| chromatography | Other | – |
| high performance liquid chromatography | Other | – |
| immunoenzyme technique | Other | – |
Primary outcome
Loss of function analyses
Time frame: Duration of the study
Proteins binding to Fanconi anemia, complementation group C (FACC) gene-product by affinity chromatography of nuclear and whole cell lysates of normal cells
Time frame: Duration of the study
Screening of proteins binding to FACC gene-product using monoclonal antibodies specific to signal transduction and cell cycle proteins
Time frame: Duration of the study
Microsequencing of unique proteins
Time frame: Duration of the study
Location of specific downstream block point imposed by antisense molecules using antibodies specific to signal transduction, cell cycle, or repair proteins for the FACC protein
Time frame: Duration of the study
Affirmation that the block points identified are recapitulated in progenitor cells from peripheral blood
Time frame: Duration of the study
Identification of functional defects in Fanconi anemia hematopoietic stromal cells
Time frame: Duration of the study
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 5 Jul 2022.
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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.
Laura Newell, MD principal investigator
OHSU Knight Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Myeloproliferative Disorders and Fanconi Anemia, run by OHSU Knight 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: finished, as of the sponsor's last update on 5 Jul 2022. 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.
What does the primary outcome measure?
Loss of function analyses (time frame: Duration of the study). The study lists 7 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 5 Jul 2022.