Hematologic DiseasesNCT00001397
Evaluation, Treatment, and Training for Patients With Blood Disorders
An observational study of Hematologic Diseases, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
This study is designed to allow the evaluation, follow-up, and medical care of patients with blood disorders not currently participating in a research study being conducted by the Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI) or not being screened for participation in a study. Read more about this study
- Who is it for?
- Ages 2 to 110 years
- Conditions the study is for
- Hematologic Diseases
- How many people
- 1,452 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Nov 1993to 5 Oct 2020
- 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 2 to 110 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All subjects age 2 or older and weighing greater than 12 kg, who meet entry criteria will be considered for participation on this protocol at the discretion of the Principal or Associate Investigator.
Who may be able to join
- Patients (and when appropriate the patient's stem cell donor) may be entered on this protocol at the discretion of the Principal and/or Associate Investigators if :
- An investigator decides that it is in the best interest of the patient/donor and the Branch for the patient to be enrolled in this protocol while awaiting a decision on eligibility for other protocols; or after enrollment on another protocol, or to allow careful evaluation of patients with unique or rare disorders of direct interest to the Branch; or with more common disorders for fellowship teaching purposes.
- The patient or the patient's guardian is capable of informed consent and signs the consent form. The consent form will be signed by parents or guardians of patients under the age of 18.
- Age greater than or equal to 2 and weight greater than 12 kg.
Who cannot join
None listed.
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
Official title: Evaluation, Treatment and Training Protocol for Patients With Bone Marrow Failure States, Isolated or Multilineage Cytopenias, Metastatic Solid Tumors, or Hematologic Malignancies
This study is designed to allow the evaluation, follow-up, and medical care of patients with blood disorders not currently participating in a research study being conducted by the Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI) or not being screened for participation in a study.
The purpose of this study is to allow investigation into the blood disorders of patients in order to teach, learn, and gather more information about diseases of the blood.
In addition, this study allows researchers the opportunity to evaluate patients referred to the Hematology Branch of the NHLBI with rare or undiagnosed diseases of the blood. This may be potentially beneficial to the patient and at the same time contribute to the development of new research ideas.
Read the detailed description
This protocol is designed to allow the evaluation, follow up, and standard medical care of patients (and when appropriate, their stem cell donor) with bone marrow failure states, cytopenias, metastatic solid tumors, or hematologic malignancies not currently entered on an active NHLBI protocol or not being screened for an active NHLBI research protocol. Its purpose is to allow investigation into the hematologic problems of these patients for the primary purpose of teaching and furthering general hematologic knowledge. The ability to evaluate and treat patients with a wide variety of hematologic disease is critical to maintaining our accreditation as a hematology fellowship program and training our fellows to be competent hematologists, as well as for keeping all staff, including senior staff, up-to-date and familiar with the evaluation and treatment of patients with a wide spectrum of hematologic disease. It also allows the NHLBI investigators to evaluate patients referred with rare or as yet undiagnosed hematologic problems, both potentially benefiting the patient and stimulating new research directions for the NHLBI in the future. Periodic follow-up and treatment of patients previously entered on NHLBI protocols in order to monitor the long-term course of the underlying hematologic state and the consequences of experimental treatments is also required for fellowship training, and for maintenance of good referral relationships with patients and their physicians.
- Conditions
- Hematologic Diseases
- Keywords
- Hematopoiesis, Thrombocytopenia, Anemia, Neutropenia, Dysplasia, Hematologic Malignancies
- Medical subject headings
- Hematologic Diseases, Thrombocytopenia, Anemia, Neutropenia, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | treated according to the guidelines of standard medical evaluation and care. No investigational treatments or procedures will be administered on this protocol. |
What is being measured
The primary outcome is the main question the study is designed to answer.
To provide a mechanism for the evaluation, follow-up, and standard hematological care of patients by the Hematology Branch
To provide a mechanism for the evaluation, follow-up, and standard hematological care of patients by the Hematology Branch
Measured: 20 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 1999 (estimated)
- Study start8 Nov 1993
- Primary completion5 Oct 2020
- Study completion5 Oct 2020
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 Hematologic DiseasesStudies 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
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 1,452 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Georg Aue, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001397
- Sponsor's protocol ID
- 940010
- Other IDs
- 94-H-0010
- Status last verified
- Oct 2020
- First submitted
- 3 Nov 1999
- Last updated
- 8 Oct 2020
- Results posted
- No
Publications
Giudice V, Wu Z, Kajigaya S, Fernandez Ibanez MDP, Rios O, Cheung F, Ito S, Young NS. Circulating S100A8 and S100A9 protein levels in plasma of patients with acquired aplastic anemia and myelodysplastic syndromes. Cytokine. 2019 Jan;113:462-465. doi: 10.1016/j.cyto.2018.06.025. Epub 2018 Jun 27.
PubMed 29958797Giudice V, Banaszak LG, Gutierrez-Rodrigues F, Kajigaya S, Panjwani R, Ibanez MDPF, Rios O, Bleck CK, Stempinski ES, Raffo DQ, Townsley DM, Young NS. Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes. Haematologica. 2018 Jul;103(7):1150-1159. doi: 10.3324/haematol.2017.182824. Epub 2018 Apr 19.
PubMed 29674506Giudice V, Feng X, Lin Z, Hu W, Zhang F, Qiao W, Ibanez MDPF, Rios O, Young NS. Deep sequencing and flow cytometric characterization of expanded effector memory CD8+CD57+ T cells frequently reveals T-cell receptor Vbeta oligoclonality and CDR3 homology in acquired aplastic anemia. Haematologica. 2018 May;103(5):759-769. doi: 10.3324/haematol.2017.176701. Epub 2018 Feb 1.
PubMed 29419434
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation, Treatment and Training Protocol for Patients With Bone Marrow Failure States, Isolated or Multilineage Cytopenias, Metastatic Solid Tumors, or Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00001397
- Acronym / sponsor ID
- 940010
- Conditions
- Hematologic Diseases
- Keywords
- Hematopoiesis, Thrombocytopenia, Anemia, Neutropenia, Dysplasia, Hematologic Malignancies
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,452 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 8 Nov 1993 (actual)
- Primary completion
- 5 Oct 2020 (actual)
- Study completion
- 5 Oct 2020 (actual)
- First posted
- 4 Nov 1999 (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 is designed to allow the evaluation, follow-up, and medical care of patients with blood disorders not currently participating in a research study being conducted by the Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI) or not being screened for participation in a study.
The purpose of this study is to allow investigation into the blood disorders of patients in order to teach, learn, and gather more information about diseases of the blood.
In addition, this study allows researchers the opportunity to evaluate patients referred to the Hematology Branch of the NHLBI with rare or undiagnosed diseases of the blood. This may be potentially beneficial to the patient and at the same time contribute to the development of new research ideas.
Detailed description
This protocol is designed to allow the evaluation, follow up, and standard medical care of patients (and when appropriate, their stem cell donor) with bone marrow failure states, cytopenias, metastatic solid tumors, or hematologic malignancies not currently entered on an active NHLBI protocol or not being screened for an active NHLBI research protocol. Its purpose is to allow investigation into the hematologic problems of these patients for the primary purpose of teaching and furthering general hematologic knowledge. The ability to evaluate and treat patients with a wide variety of hematologic disease is critical to maintaining our accreditation as a hematology fellowship program and training our fellows to be competent hematologists, as well as for keeping all staff, including senior staff, up-to-date and familiar with the evaluation and treatment of patients with a wide spectrum of hematologic disease. It also allows the NHLBI investigators to evaluate patients referred with rare or as yet undiagnosed hematologic problems, both potentially benefiting the patient and stimulating new research directions for the NHLBI in the future. Periodic follow-up and treatment of patients previously entered on NHLBI protocols in order to monitor the long-term course of the underlying hematologic state and the consequences of experimental treatments is also required for fellowship training, and for maintenance of good referral relationships with patients and their physicians.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –treated according to the guidelines of standard medical evaluation and care. No investigational treatments or procedures will be administered on this protocol. |
Who may qualify
- Ages
- 2 to 110 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All subjects age 2 or older and weighing greater than 12 kg, who meet entry criteria will be considered for participation on this protocol at the discretion of the Principal or Associate Investigator.
Key inclusion criteria (4 listed)
- Patients (and when appropriate the patient's stem cell donor) may be entered on this protocol at the discretion of the Principal and/or Associate Investigators if :
- An investigator decides that it is in the best interest of the patient/donor and the Branch for the patient to be enrolled in this protocol while awaiting a decision on eligibility for other protocols; or after enrollment on another protocol, or to allow careful evaluation of patients with unique or rare disorders of direct interest to the Branch; or with more common disorders for fellowship teaching purposes.
- The patient or the patient's guardian is capable of informed consent and signs the consent form. The consent form will be signed by parents or guardians of patients under the age of 18.
- Age greater than or equal to 2 and weight greater than 12 kg.
Key exclusion criteria (0 listed)
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
To provide a mechanism for the evaluation, follow-up, and standard hematological care of patients by the Hematology Branch
Time frame: 20 years
To provide a mechanism for the evaluation, follow-up, and standard hematological care of patients by the Hematology Branch
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 8 Oct 2020.
- Derived from this study: PubMed 29958797Giudice V, Wu Z, Kajigaya S, Fernandez Ibanez MDP, Rios O, Cheung F, Ito S, Young NS. Circulating S100A8 and S100A9 protein levels in plasma of patients with acquired aplastic anemia and myelodysplastic syndromes. Cytokine. 2019 Jan;113:462-465. doi: 10.1016/j.cyto.2018.06.025. Epub 2018 Jun 27.
- Derived from this study: PubMed 29674506Giudice V, Banaszak LG, Gutierrez-Rodrigues F, Kajigaya S, Panjwani R, Ibanez MDPF, Rios O, Bleck CK, Stempinski ES, Raffo DQ, Townsley DM, Young NS. Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes. Haematologica. 2018 Jul;103(7):1150-1159. doi: 10.3324/haematol.2017.182824. Epub 2018 Apr 19.
- Derived from this study: PubMed 29419434Giudice V, Feng X, Lin Z, Hu W, Zhang F, Qiao W, Ibanez MDPF, Rios O, Young NS. Deep sequencing and flow cytometric characterization of expanded effector memory CD8+CD57+ T cells frequently reveals T-cell receptor Vbeta oligoclonality and CDR3 homology in acquired aplastic anemia. Haematologica. 2018 May;103(5):759-769. doi: 10.3324/haematol.2017.176701. Epub 2018 Feb 1.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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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.
Georg Aue, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
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 Hematologic Diseases, 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 4 inclusion and 0 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 8 Oct 2020. 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?
To provide a mechanism for the evaluation, follow-up, and standard hematological care of patients by the Hematology Branch (time frame: 20 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001397, 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 8 Oct 2020.