Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00767650
Other study ID # 080222
Secondary ID 08-H-0222
Status Completed
Phase N/A
First received October 6, 2008
Last updated June 30, 2017
Start date September 23, 2008
Est. completion date June 28, 2011

Study information

Verified date June 28, 2011
Source National Institutes of Health Clinical Center (CC)
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical 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. 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 7
Est. completion date June 28, 2011
Est. primary completion date
Accepts healthy volunteers No
Gender All
Age group 15 Years and older
Eligibility - INCULSION CRITERIA:

- Diagnosed with aplastic anemia.

- Co-enrolled on a Clinical Center CsA treatment protocol that prescribes CsA (included but not limited to initial treatment cohort in both rabbit and horse ATG arm for the aplastic anemia patients).

- Age greater than or equal to 15 years old.

EXCLUSION CRITERIA:

- 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.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda Maryland

Sponsors (1)

Lead Sponsor Collaborator
National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

References & Publications (3)

Gijtenbeek JM, van den Bent MJ, Vecht CJ. Cyclosporine neurotoxicity: a review. J Neurol. 1999 May;246(5):339-46. Review. — View Citation

Syrjala 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. Epub 2004 Jul 13. — View Citation

Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19. Epub 2006 Jun 15. Review. — View Citation

See also
  Status Clinical Trial Phase
Active, not recruiting NCT03025698 - A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia Phase 2
Completed NCT00987480 - Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine Phase 2
Not yet recruiting NCT02833493 - Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload N/A
Completed NCT02833805 - NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Phase 2
Recruiting NCT02028416 - Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia N/A
Completed NCT00004474 - Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia Phase 3
Recruiting NCT05031897 - Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell Transplant Phase 2
Completed NCT04439006 - Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization Phase 1
Not yet recruiting NCT05996393 - CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly Phase 4
Completed NCT02462252 - Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome Phase 2
Completed NCT00513175 - Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia N/A
Completed NCT01272817 - Nonmyeloablative Allogeneic Transplant N/A
Completed NCT00001398 - Stem Cell Factor Medication for Aplastic Anemia Phase 1
Recruiting NCT01861093 - Safety Study of Cord Blood Units for Stem Cell Transplants Phase 2
Not yet recruiting NCT05018936 - Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia Phase 2/Phase 3
Completed NCT00065260 - Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia Phase 2
Recruiting NCT02007811 - Open-label Clinical Trial to Investigate the Safety and Tolerability of Allogeneic B-cell Concentrates for Immune Reconstitution After Allogeneic Stem Cell Transplantation Measured as Response to a Antedated Single Vaccination Phase 1/Phase 2
Recruiting NCT01758042 - Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders N/A
Terminated NCT01500161 - Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match Phase 2
Recruiting NCT00882323 - Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA) Phase 2