Collection of Blood, Stool, Bone Marrow and/or Tissue Samples From Subjects With Hepatitis-Associated Aplastic Anemia
Collection of Biological Samples From Patients With Hepatitis-Associated Aplastic Anemia
Sponsors
Source
National Institutes of Health Clinical Center (CC)
Brief Summary
This study will collect samples of blood, stool, bone marrow, or other tissues from patients
with hepatitis-associated aplastic anemia to investigate a possible association between
exposure to viruses and the development of aplastic anemia in these patients. Cells from the
samples obtained may be grown in the laboratory for future studies. Patients samples may be
used to:
- Study abnormalities that occur in hepatitis-associated aplastic anemia;
- Test for various viruses;
- Test immune function;
- Measure factors related to the patients disease or diseases they may be at risk for;
- Evaluate the effectiveness of current therapies, refine treatment approaches, and
identify potential new therapies;
- Identify possible measures for disease prevention;
- Identify possible genetic factors associated with hepatitis-associated aplastic anemia.
Patients 2 years of age and older with severe aplastic anemia that developed within 6 months
of an episode of hepatitis may be eligible for this study.
Participants will complete questionnaires and provide tissue samples as described below.
Questionnaires
All patients (or another respondent for the patient) will fill out a questionnaire including
demographic information (age, gender, race, ethnic group, education level, state of
residence), current symptoms, medications, medical history, and history of possible exposures
to toxins or viruses. A second questionnaire, which includes questions related to mental
health, sexual behavior, alcohol and drug use, is optional for participants age 21 and older.
These questionnaires are designed to uncover features of hepatitis-associated aplastic anemia
and possibly reveal a common cause of the disease.
Sample Collections
- Blood- will be collected at the time of the patient s initial evaluation or upon
enrollment into the study and possibly periodically during the study. Blood will be
drawn through a needle in an arm vein.
- Bone marrow- may be collected as part of the patient s standard medical care or
specifically for research purposes of this study. For this procedure, the skin over the
hipbone and the outer surface of the bone itself are numbed with an injection of a local
anesthesia. Then, a larger needle is inserted into the hipbone and marrow is drawn into
a syringe. Marrow cells are suctioned two to six times during the 15-minute procedure.
- Stool- will be provided by the patient.
Liver- tissue may be biopsied as part of the patient s general medical care or for NIH
patients, as part of their enrollment in a treatment protocol.
Detailed Description
Our laboratory has a long-standing interest in viruses that affect the bone marrow,
especially those causing bone marrow failure. One specific syndrome, hepatitis-associated
aplastic anemia, suggests that the same agent is responsible for the severe and sometimes
fulminate hepatitis as well as the profound bone marrow failure. This study is designed to
collect clinical data, and samples from patients with hepatitis-associated aplastic anemia,
in order to learn more about the clinical features of the disease, as well as to collect
blood, liver, bone marrow and stool samples where possible for ongoing virological studies.
For liver and bone marrow samples, material will only be obtained when the liver or bone
marrow are biopsied for other medical indications, or during the removal of the liver at the
time of transplantation. No additional risk to the patient should result from either
procedure.
Overall Status
Completed
Start Date
2002-11-18
Completion Date
N/A
Primary Completion Date
N/A
Study Type
Observational
Enrollment
9
Condition
Eligibility
Criteria
- INCLUSION CRITERIA
Presumptive clinical diagnosis of hepatitis-associated aplastic anemia. That is, aplastic
anemia within 12 months of an episode of hepatitis
Age equal to or greater than 2 years old
Ability to comprehend the investigational nature of the study and provide informed consent.
EXCLUSION CRITERIA
Suspected cholestatic or obstructed liver disease
Current diagnosis or past history of myelodysplastic syndrome, Fanconi s anemia,
dyskeratosis congenita or other congenital forms of aplastic anemia.
Diagnosis of Diamond-Blackfan anemia or a constitutional marrow failure disease.
Underlying carcinoma, recent history of radiation or chemotherapy
Age less than 2 years old
Gender
All
Minimum Age
2 Years
Maximum Age
N/A
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
|
Neal S Young, M.D. |
Principal Investigator |
National Heart, Lung, and Blood Institute (NHLBI) |
Location
Facility |
|
National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda Maryland 20892 United States |
Location Countries
Country
United States
Verification Date
2018-03-21
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Has Expanded Access
No
Condition Browse
Secondary Id
03-H-0051
Firstreceived Results Date
N/A
Reference
Citation
Böttiger LE, Westerholm B. Aplastic anaemia. 3. Aplastic anaemia and infectious hepatitis. Acta Med Scand. 1972 Oct;192(4):323-6.
PMID
5081070
Citation
Mary JY, Baumelou E, Guiguet M. Epidemiology of aplastic anemia in France: a prospective multicentric study. The French Cooperative Group for Epidemiological Study of Aplastic Anemia. Blood. 1990 Apr 15;75(8):1646-53.
PMID
2183887
Citation
Young NS, Issaragrasil S, Chieh CW, Takaku F. Aplastic anaemia in the Orient. Br J Haematol. 1986 Jan;62(1):1-6.
PMID
3942690
Firstreceived Results Disposition Date
N/A
Study Design Info
Time Perspective
Prospective
Study First Submitted
November 21, 2002
Study First Submitted Qc
November 19, 2002
Study First Posted
November 20, 2002
Last Update Submitted
August 8, 2018
Last Update Submitted Qc
August 8, 2018
Last Update Posted
August 9, 2018
ClinicalTrials.gov processed this data on August 31, 2018
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.