Aplastic AnemiaNCT00767650
Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
An observational study of Aplastic Anemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
This study will use neuropsychological tests to look at nervous system side effects of Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an immunosuppressive regimen in treating severe aplastic anemia. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Aplastic Anemia
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Sep 2008to 28 Jun 2011
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- Prior use of cyclosporine within 6 months to next line of treatment.
- History of learning disability (i.e. dyslexia).
- Unable to read and speak English (the neuropsychological testing tools are validated for use in English speaking subjects only).
- Life expectancy less than six months or when clinical status prevents full performance with testing.
- Either adult patients or guardians for the minor patient, unable to comprehend the investigational nature of the study and provide informed consent.
- Inability or unwillingness to come to Clinical Center for the 6-month and 12-month follow-up appointments.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
This study will use neuropsychological tests to look at nervous system side effects of Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an immunosuppressive regimen in treating severe aplastic anemia. The drug can produce nervous system side effects, such as tremor and, less commonly, insomnia, anxiety, headache, confusion or seizures. This study will look at effects of CsA on intellectual ability, depression, anxiety, attention, concentration, memory, perception, coordination, and thought processing in patients
Patients 15 years of age or older who have severe aplastic anemia may be eligible for this study if they:
Read more
- are co-enrolled in a Clinical Center protocol in which they will receive CsA
- have not taken CsA for 6 months before enrolling in this study
Participants undergo neuropsychological testing. In addition, they provide blood samples and their clinical data are reviewed for things that may influence the interpretation of findings from the testing, such as results of blood tests, types of medications taken, number of transfusions required, etc. The procedures are as follows:
Before first dose of cyclosporine:
- Patients are asked about prior problems with their nervous system, prior treatment for their aplastic anemia (including transfusions), prior infections, and current medications. They then complete the following sets of tests:
- Battery 1: A set of three tests that measure intellectual ability, level of depression (if any) and level of anxiety (if any).
- Battery 2: A set of seven tests that measure changes in the central nervous system and how these changes affect attention, concentration, memory, perception, coordination, and thought processing.
- Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol. 6 months and 12 months after starting cyclosporine
- Patients are asked about treatment for their aplastic anemia (including transfusions), infections, and changes in medications that have occurred since they started taking cyclosporine. They then repeat the set of tests in Battery 2.
- Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol.
Read the detailed description
Cyclosporine (CsA) is widely used in the treatment of aplastic anemia as well as to provide immunosuppression after transplantation. CsA has a spectrum of neurologic and more subtle, poorly investigated neuropsychological functional effects. In the NHLBI, a unique opportunity exists to evaluate changes in neuropsychological function following CsA in a large cohort of aplastic anemia patients accrued to NHLBI treatment protocols.
With increased success in treating blood diseases with immunosuppressive therapy or stem cell transplantation that utilize CsA in the treatment regimen, patients are enjoying prolonged survival during which quality of life becomes an increasingly relevant concern. We therefore propose this natural history protocol designed to evaluate neuropsychological functioning before, during, and after CsA administration in severe aplastic anemia patients.
The primary objective is to evaluate changes in attention, language, memory, spatial/motor and executive function domains following CsA therapy using well established neuropsychological tests including the Neuropsychological Assessment Battery (NAB) Screening Module, Revised Hopkins Verbal Learning Test, Revised Brief Visual-spatial Memory Test, Judgment of Line Orientation test, Grooved Pegboard, Trail Making Test and the two performance subsets in Wechsler Adult Intelligence Scale-III. IQ test by Wechsler Abbreviated Scale of Intelligence and depression/anxiety level will be screened once prior to formal testing. Measures will also be correlated with clinical status, standard CsA side effects, CsA drug levels and relevant biochemical lab values at each time point to control the testing accuracy and explore potential correlation.
- Conditions
- Aplastic Anemia
- Keywords
- Aplastic Anemia, Cyclosporine, Campath, Neurophyschological Side Effect
- Medical subject headings
- Anemia, Aplastic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2008 (estimated)
- Study start23 Sep 2008
- Study completion28 Jun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- Time perspective
- Prospective
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00767650
- Sponsor's protocol ID
- 080222
- Other IDs
- 08-H-0222
- Status last verified
- 28 Jun 2011
- First submitted
- 6 Oct 2008
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19. doi: 10.1182/blood-2006-03-010777. Epub 2006 Jun 15.
PubMed 16778145Gijtenbeek JM, van den Bent MJ, Vecht CJ. Cyclosporine neurotoxicity: a review. J Neurol. 1999 May;246(5):339-46. doi: 10.1007/s004150050360.
PubMed 10399863Syrjala KL, Dikmen S, Langer SL, Roth-Roemer S, Abrams JR. Neuropsychologic changes from before transplantation to 1 year in patients receiving myeloablative allogeneic hematopoietic cell transplant. Blood. 2004 Nov 15;104(10):3386-92. doi: 10.1182/blood-2004-03-1155. Epub 2004 Jul 13.
PubMed 15251983
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00767650
- Acronym / sponsor ID
- 080222
- Conditions
- Aplastic Anemia
- Keywords
- Aplastic Anemia, Cyclosporine, Campath, Neurophyschological Side Effect
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 23 Sep 2008
- Study completion
- 28 Jun 2011
- First posted
- 7 Oct 2008 (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.
This study will use neuropsychological tests to look at nervous system side effects of Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an immunosuppressive regimen in treating severe aplastic anemia. The drug can produce nervous system side effects, such as tremor and, less commonly, insomnia, anxiety, headache, confusion or seizures. This study will look at effects of CsA on intellectual ability, depression, anxiety, attention, concentration, memory, perception, coordination, and thought processing in patients
Patients 15 years of age or older who have severe aplastic anemia may be eligible for this study if they:
- are co-enrolled in a Clinical Center protocol in which they will receive CsA
- have not taken CsA for 6 months before enrolling in this study
Participants undergo neuropsychological testing. In addition, they provide blood samples and their clinical data are reviewed for things that may influence the interpretation of findings from the testing, such as results of blood tests, types of medications taken, number of transfusions required, etc. The procedures are as follows:
Before first dose of cyclosporine:
- Patients are asked about prior problems with their nervous system, prior treatment for their aplastic anemia (including transfusions), prior infections, and current medications. They then complete the following sets of tests:
- Battery 1: A set of three tests that measure intellectual ability, level of depression (if any) and level of anxiety (if any).
- Battery 2: A set of seven tests that measure changes in the central nervous system and how these changes affect attention, concentration, memory, perception, coordination, and thought processing.
- Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol. 6 months and 12 months after starting cyclosporine
- Patients are asked about treatment for their aplastic anemia (including transfusions), infections, and changes in medications that have occurred since they started taking cyclosporine. They then repeat the set of tests in Battery 2.
- Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol.
Detailed description
Cyclosporine (CsA) is widely used in the treatment of aplastic anemia as well as to provide immunosuppression after transplantation. CsA has a spectrum of neurologic and more subtle, poorly investigated neuropsychological functional effects. In the NHLBI, a unique opportunity exists to evaluate changes in neuropsychological function following CsA in a large cohort of aplastic anemia patients accrued to NHLBI treatment protocols.
With increased success in treating blood diseases with immunosuppressive therapy or stem cell transplantation that utilize CsA in the treatment regimen, patients are enjoying prolonged survival during which quality of life becomes an increasingly relevant concern. We therefore propose this natural history protocol designed to evaluate neuropsychological functioning before, during, and after CsA administration in severe aplastic anemia patients.
The primary objective is to evaluate changes in attention, language, memory, spatial/motor and executive function domains following CsA therapy using well established neuropsychological tests including the Neuropsychological Assessment Battery (NAB) Screening Module, Revised Hopkins Verbal Learning Test, Revised Brief Visual-spatial Memory Test, Judgment of Line Orientation test, Grooved Pegboard, Trail Making Test and the two performance subsets in Wechsler Adult Intelligence Scale-III. IQ test by Wechsler Abbreviated Scale of Intelligence and depression/anxiety level will be screened once prior to formal testing. Measures will also be correlated with clinical status, standard CsA side effects, CsA drug levels and relevant biochemical lab values at each time point to control the testing accuracy and explore potential correlation.
Study design
- Time perspective
- Prospective
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (6 listed)
- Prior use of cyclosporine within 6 months to next line of treatment.
- History of learning disability (i.e. dyslexia).
- Unable to read and speak English (the neuropsychological testing tools are validated for use in English speaking subjects only).
- Life expectancy less than six months or when clinical status prevents full performance with testing.
- Either adult patients or guardians for the minor patient, unable to comprehend the investigational nature of the study and provide informed consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
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 2 Jul 2017.
- Background: PubMed 16778145Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19. doi: 10.1182/blood-2006-03-010777. Epub 2006 Jun 15.
- Background: PubMed 10399863Gijtenbeek JM, van den Bent MJ, Vecht CJ. Cyclosporine neurotoxicity: a review. J Neurol. 1999 May;246(5):339-46. doi: 10.1007/s004150050360.
- Background: PubMed 15251983Syrjala KL, Dikmen S, Langer SL, Roth-Roemer S, Abrams JR. Neuropsychologic changes from before transplantation to 1 year in patients receiving myeloablative allogeneic hematopoietic cell transplant. Blood. 2004 Nov 15;104(10):3386-92. doi: 10.1182/blood-2004-03-1155. Epub 2004 Jul 13.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Aplastic Anemia, run by National Heart, Lung, and Blood Institute (NHLBI) 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?
The sponsor lists 0 inclusion and 6 exclusion criteria. 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 2 Jul 2017. 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?
On the ClinicalTrials.gov record for NCT00767650, which also lists 3 linked publications.
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 2 Jul 2017.