Anemia, AplasticPhase 2NCT00944749
Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment
A Phase 2 study testing h-ATG (ATGAM ) and Cyclosporine (Gengraf ) in people with Anemia, Aplastic and Anemia, Hypoplastic, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions to improve blood clotting. A standard treatment for SAA involves injections of rabbit-antithymocyte globulin (r-ATG). Read more about this study
- Who is it for?
- Ages 2 to 82 years
- Conditions the study is for
- Anemia, Aplastic, Anemia, Hypoplastic
- What is being tested?
- h-ATG (ATGAM ) and Cyclosporine (Gengraf )
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2009to Dec 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 82 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosed with SAA characterized by:
- Bone marrow cellularity < 30% (excluding lymphocytes)
- At least two of the following:
- Absolute neutrophil count <500/ microL
- Platelet count <20,000/ microL
- Reticulocyte count <60,000/ microL
- Failure to respond to an initial course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment or a suboptimal response to initial therapy defined by both platelet and reticulocyte count < 50,000 /microL at 3 months post-treatment
- Age greater than or equal to 2 years of age
Who cannot join
- Diagnosis of Fanconi anemia. Patients with very severe neutropenia (ANC < 200 /microL) will not be excluded initially if results of Fanconi anemia testing are not available or pending. If evidence of Fanconi anemia is later identified, the subject will go off study.
- Evidence of a clonal disorder on cytogenetics. Patients with very severe neutropenia (ANC < 200/uL) will not be excluded initially if results of cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the subject will go off study.
- Patients who received prior course(s) of alemtuzumab will not be excluded.
- Infection not adequately responding to appropriate therapy
- HIV seropositivity
- Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy or that death within 7-10 days is likely.
Show all 10Show fewer
- Subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
- Serum creatinine > 2.5 mg/dL
- Current pregnancy, breast-feeding or unwillingness to refrain from pregnancy if of child bearing potential
- Inability to understand the investigational nature of the study or give informed consent
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
Background:
- Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions to improve blood clotting.
- A standard treatment for SAA involves injections of rabbit-antithymocyte globulin (r-ATG). r-ATG is developed by injecting horses with a type of human white blood cells called thymocytes. The horse's immune system reacts against these cells and makes antibodies that can destroy them. These antibodies are collected and purified to make r-ATG. Horses can also be used for this procedure to make horse-antithymocyte globulin (h-ATG).
- h-ATG is approved by the Food and Drug Administration for the treatment of aplastic anemia. h-ATG is a standard first-line method to treat aplastic anemia, but researchers do not know how effective it is in patients who were first treated unsuccessfully with r-ATG.
Read more
Objectives:
- To evaluate the effectiveness and safety of horse-ATG (with cyclosporine) in increasing blood counts and reducing the need for transfusions in aplastic anemia patients who have failed to respond to prior immunosuppressive treatment with rabbit-ATG and cyclosporine.
Eligibility:
- Patients 2 years of age and older who have consistently low blood platelet counts related to aplastic anemia that has not responded to conventional treatment with rabbit-ATG.
Design:
- After initial screening, medical history, and blood tests, patients will be admitted to the inpatient unit at the National Institutes of Health Clinical Center. Researchers will perform a skin test with h-ATG to check for allergic or other adverse reaction.
- After the skin test, h-ATG will be given into a vein continuously over 4 days.
- Cyclosporine will also be given to improve the response rate of ATG treatment. Treatment with cyclosporine will start the same day as the h-ATG, either in liquid or capsule form, and continued for 6 months. The dose of cyclosporine will be monitored and adjusted based on blood levels and signs of side effects in the kidney and liver.
- To prevent or treat infections that may result from cyclosporine s effect on the immune system, patients will also take inhaled or capsule doses of pentamidine.
- After the study is completed, patients will have followup evaluations every 3 months, 6 months, and annually for 5 years. Evaluations will include blood samples and periodic bone marrow biopsies.
Read the detailed description
Severe aplastic anemia (SAA), characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggests that patients with minimal blood count responses to a single course of ATG, even when transfusion independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement.
The majority of the experience in the US and worldwide has been with horse ATG (h-ATG) plus CsA as initial therapy in SAA. Rabbit ATG (r-ATG) plus CsA has been employed successfully in about 1/3 of cases in those who are refractory to initial h-ATG/CsA (current NHLBI Protocol 03-H-0249). In recent years, h-ATG and r-ATG have been used interchangeably in treatment-naive patients, and initial therapy with r-ATG/CsA is now frequent in the US and the only option in Europe and Japan, where h-ATG is no longer available. An active NHLBI randomized study is comparing the efficacy of h- and r-ATG as initial therapy in SAA, and the results from a recently completed interim analysis suggest that the hematologic response rate ultimately may not be comparable between these two agents (Protocol 06-H-0034). There is no published report on the outcome of repeat immunosuppressive therapy in those patients refractory to initial r-ATG/CsA, and thus the management of these patients is uncertain. We therefore propose this study of h-ATG/CsA in SAA patients who are refractory or have a suboptimal response to r-ATG.
The primary endpoint will be the response rate at 3 months where response is defined as no longer meeting criteria for SAA.
The primary objective is to evaluate the effectiveness (response rate) at 3 months of a second course of immunosuppression with h-ATG/CsA in subjects refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post treatment.
Secondary objectives include robustness of hematologic recovery, relapse, response rate at 6 months, clonal evolution and overall survival.
The primary endpoint will be changes in absolute neutrophil count, platelet count, reticulocyte count at 3 months.
Secondary endpoints will include time to relapse, changes in cytogenetics, time to death.
- Conditions
- Anemia, Aplastic, Anemia, Hypoplastic
- Being tested
- h-ATG (ATGAM ), Cyclosporine (Gengraf )
- Keywords
- H- ATG, Anti -Thymocyte Globulin, Lymphocyte Immune Globulin, Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
h-ATG (ATGAM )Drug
Cyclosporine (Gengraf )Drug
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Not described Receives: Drug: h-ATG (ATGAM ); Drug: Cyclosporine (Gengraf ) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Complete Response at 3 Months.
The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA) defined as bone marrow cellularity of less than 30% and severe pancytopenia with at least two of the following peripheral blood count criteria: (i) absolute neutrophil count less than 0.5×109/L; (ii) absolute reticulocyte count less than 60×109/L; and (iii) platelet count less than 20×109/L.
A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb > 10 g/dL, and platelet count > 100×109/L.
A partial response was defined as a hematologic response that was not sufficient for a complete response.
Measured: 3-months
Number of Participants With Complete Response at 3 Months.
The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA).
A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb > 10 g/dL, and platelet count > 100×109/L.
A partial response was defined as a hematologic response that was not sufficient for a complete response.
In subjects with a non-robust hematologic response at 3 months, improvement in one or more of the listed peripheral blood parameter (a,b,c) were recorded as a response: a) ANC - if baseline ANC below 0.5×109/L, increase in ANC by > 0.3×109/L, if baseline ANC above 0.5×109/L, any increase in ANC by > 0.5×109/L of blood; (b) platelets - if baseline platelet count < 50×109/L, any increase in platelet count by > 20×109/L of blood; c) hemoglobin - any increase in hemoglobin by 1.5 g/dl of blood in transfusion-independent patients and in absolute reticulocyte count to > 60×109/L of blood in transfusion-dependent patients.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2009 (estimated)
- Study start31 Aug 2009
- Primary completionDec 2015
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Anemia, AplasticStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Danielle M Townsley, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00944749
- Sponsor's protocol ID
- 090183
- Other IDs
- 09-H-0183
- Status last verified
- Sep 2020
- First submitted
- 22 Jul 2009
- Last updated
- 23 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Mendez G Jr, Russell E. Gastrointestinal varices: percutaneous transheptic therapeutic embolization in 54 patients. AJR Am J Roentgenol. 1980 Nov;135(5):1045-50. doi: 10.2214/ajr.135.5.1045.
PubMed 6778145Maciejewski JP, Selleri C, Sato T, Anderson S, Young NS. Increased expression of Fas antigen on bone marrow CD34+ cells of patients with aplastic anaemia. Br J Haematol. 1995 Sep;91(1):245-52. doi: 10.1111/j.1365-2141.1995.tb05277.x.
PubMed 7577642Risitano AM, Maciejewski JP, Green S, Plasilova M, Zeng W, Young NS. In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing. Lancet. 2004 Jul 24-30;364(9431):355-64. doi: 10.1016/S0140-6736(04)16724-X.
PubMed 15276395Zaimoku Y, Patel BA, Adams SD, Shalhoub R, Groarke EM, Lee AAC, Kajigaya S, Feng X, Rios OJ, Eager H, Alemu L, Quinones Raffo D, Wu CO, Flegel WA, Young NS. HLA associations, somatic loss of HLA expression, and clinical outcomes in immune aplastic anemia. Blood. 2021 Dec 30;138(26):2799-2809. doi: 10.1182/blood.2021012895.
PubMed 34724566Scheinberg P, Townsley D, Dumitriu B, Scheinberg P, Weinstein B, Rios O, Wu CO, Young NS. Horse antithymocyte globulin as salvage therapy after rabbit antithymocyte globulin for severe aplastic anemia. Am J Hematol. 2014 May;89(5):467-9. doi: 10.1002/ajh.23669. Epub 2014 Mar 7.
PubMed 24415649
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment
- ClinicalTrials.gov ID
- NCT00944749
- Acronym / sponsor ID
- 090183
- Conditions
- Anemia, Aplastic, Anemia, Hypoplastic
- Keywords
- H- ATG, Anti -Thymocyte Globulin, Lymphocyte Immune Globulin, Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 31 Aug 2009 (actual)
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 23 Jul 2009 (estimated)
- Results
- Posted 23 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions to improve blood clotting.
- A standard treatment for SAA involves injections of rabbit-antithymocyte globulin (r-ATG). r-ATG is developed by injecting horses with a type of human white blood cells called thymocytes. The horse's immune system reacts against these cells and makes antibodies that can destroy them. These antibodies are collected and purified to make r-ATG. Horses can also be used for this procedure to make horse-antithymocyte globulin (h-ATG).
- h-ATG is approved by the Food and Drug Administration for the treatment of aplastic anemia. h-ATG is a standard first-line method to treat aplastic anemia, but researchers do not know how effective it is in patients who were first treated unsuccessfully with r-ATG.
Objectives:
- To evaluate the effectiveness and safety of horse-ATG (with cyclosporine) in increasing blood counts and reducing the need for transfusions in aplastic anemia patients who have failed to respond to prior immunosuppressive treatment with rabbit-ATG and cyclosporine.
Eligibility:
- Patients 2 years of age and older who have consistently low blood platelet counts related to aplastic anemia that has not responded to conventional treatment with rabbit-ATG.
Design:
- After initial screening, medical history, and blood tests, patients will be admitted to the inpatient unit at the National Institutes of Health Clinical Center. Researchers will perform a skin test with h-ATG to check for allergic or other adverse reaction.
- After the skin test, h-ATG will be given into a vein continuously over 4 days.
- Cyclosporine will also be given to improve the response rate of ATG treatment. Treatment with cyclosporine will start the same day as the h-ATG, either in liquid or capsule form, and continued for 6 months. The dose of cyclosporine will be monitored and adjusted based on blood levels and signs of side effects in the kidney and liver.
- To prevent or treat infections that may result from cyclosporine s effect on the immune system, patients will also take inhaled or capsule doses of pentamidine.
- After the study is completed, patients will have followup evaluations every 3 months, 6 months, and annually for 5 years. Evaluations will include blood samples and periodic bone marrow biopsies.
Detailed description
Severe aplastic anemia (SAA), characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggests that patients with minimal blood count responses to a single course of ATG, even when transfusion independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement.
The majority of the experience in the US and worldwide has been with horse ATG (h-ATG) plus CsA as initial therapy in SAA. Rabbit ATG (r-ATG) plus CsA has been employed successfully in about 1/3 of cases in those who are refractory to initial h-ATG/CsA (current NHLBI Protocol 03-H-0249). In recent years, h-ATG and r-ATG have been used interchangeably in treatment-naive patients, and initial therapy with r-ATG/CsA is now frequent in the US and the only option in Europe and Japan, where h-ATG is no longer available. An active NHLBI randomized study is comparing the efficacy of h- and r-ATG as initial therapy in SAA, and the results from a recently completed interim analysis suggest that the hematologic response rate ultimately may not be comparable between these two agents (Protocol 06-H-0034). There is no published report on the outcome of repeat immunosuppressive therapy in those patients refractory to initial r-ATG/CsA, and thus the management of these patients is uncertain. We therefore propose this study of h-ATG/CsA in SAA patients who are refractory or have a suboptimal response to r-ATG.
The primary endpoint will be the response rate at 3 months where response is defined as no longer meeting criteria for SAA.
The primary objective is to evaluate the effectiveness (response rate) at 3 months of a second course of immunosuppression with h-ATG/CsA in subjects refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post treatment.
Secondary objectives include robustness of hematologic recovery, relapse, response rate at 6 months, clonal evolution and overall survival.
The primary endpoint will be changes in absolute neutrophil count, platelet count, reticulocyte count at 3 months.
Secondary endpoints will include time to relapse, changes in cytogenetics, time to death.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: h-ATG (ATGAM ), Drug: Cyclosporine (Gengraf ) |
Who may qualify
- Ages
- 2 to 82 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Diagnosed with SAA characterized by:
- Failure to respond to an initial course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment or a suboptimal response to initial therapy defined by both platelet and reticulocyte count < 50,000 /microL at 3 months post-treatment
- Age greater than or equal to 2 years of age
Key exclusion criteria (10 listed)
- Diagnosis of Fanconi anemia. Patients with very severe neutropenia (ANC < 200 /microL) will not be excluded initially if results of Fanconi anemia testing are not available or pending. If evidence of Fanconi anemia is later identified, the subject will go off study.
- Evidence of a clonal disorder on cytogenetics. Patients with very severe neutropenia (ANC < 200/uL) will not be excluded initially if results of cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the subject will go off study.
- Patients who received prior course(s) of alemtuzumab will not be excluded.
- Infection not adequately responding to appropriate therapy
- HIV seropositivity
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 |
|---|---|---|
| h-ATG (ATGAM ) | Drug | Single Arm |
| Cyclosporine (Gengraf ) | Drug | Single Arm |
Primary outcome
Number of Participants With Complete Response at 3 Months.
Time frame: 3-months
The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA) defined as bone marrow cellularity of less than 30% and severe pancytopenia with at least two of the following peripheral blood count criteria: (i) absolute neutrophil count less than 0.5×109/L; (ii) absolute reticulocyte count less than 60×109/L; and (iii) platelet count less than 20×109/L. A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb > 10 g/dL, and platelet count > 100×109/L. A partial response was defined as a hematologic response that was not sufficient for a complete response.
Number of Participants With Complete Response at 3 Months.
Time frame: 3 months
The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA). A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb > 10 g/dL, and platelet count > 100×109/L. A partial response was defined as a hematologic response that was not sufficient for a complete response. In subjects with a non-robust hematologic response at 3 months, improvement in one or more of the listed peripheral blood parameter (a,b,c) were recorded as a response: a) ANC - if baseline ANC below 0.5×109/L, increase in ANC by > 0.3×109/L, if baseline ANC above 0.5×109/L, any increase in ANC by > 0.5×109/L of blood; (b) platelets - if baseline platelet count < 50×109/L, any increase in platelet count by > 20×109/L of blood; c) hemoglobin - any increase in hemoglobin by 1.5 g/dl of blood in transfusion-independent patients and in absolute reticulocyte count to > 60×109/L of blood in transfusion-dependent patients.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 6778145Mendez G Jr, Russell E. Gastrointestinal varices: percutaneous transheptic therapeutic embolization in 54 patients. AJR Am J Roentgenol. 1980 Nov;135(5):1045-50. doi: 10.2214/ajr.135.5.1045.
- Background: PubMed 7577642Maciejewski JP, Selleri C, Sato T, Anderson S, Young NS. Increased expression of Fas antigen on bone marrow CD34+ cells of patients with aplastic anaemia. Br J Haematol. 1995 Sep;91(1):245-52. doi: 10.1111/j.1365-2141.1995.tb05277.x.
- Background: PubMed 15276395Risitano AM, Maciejewski JP, Green S, Plasilova M, Zeng W, Young NS. In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing. Lancet. 2004 Jul 24-30;364(9431):355-64. doi: 10.1016/S0140-6736(04)16724-X.
- Derived from this study: PubMed 34724566Zaimoku Y, Patel BA, Adams SD, Shalhoub R, Groarke EM, Lee AAC, Kajigaya S, Feng X, Rios OJ, Eager H, Alemu L, Quinones Raffo D, Wu CO, Flegel WA, Young NS. HLA associations, somatic loss of HLA expression, and clinical outcomes in immune aplastic anemia. Blood. 2021 Dec 30;138(26):2799-2809. doi: 10.1182/blood.2021012895.
- Derived from this study: PubMed 24415649Scheinberg P, Townsley D, Dumitriu B, Scheinberg P, Weinstein B, Rios O, Wu CO, Young NS. Horse antithymocyte globulin as salvage therapy after rabbit antithymocyte globulin for severe aplastic anemia. Am J Hematol. 2014 May;89(5):467-9. doi: 10.1002/ajh.23669. Epub 2014 Mar 7.
- 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.
Danielle M Townsley, 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?
A Phase 2 study testing h-ATG (ATGAM ) and Cyclosporine (Gengraf ) in people with Anemia, Aplastic and Anemia, Hypoplastic, 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 8 inclusion and 10 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 23 Mar 2021. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Feb 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Complete Response at 3 Months. (time frame: 3-months). The study lists 2 primary outcomes in all. 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 NCT00944749, which also lists 5 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 23 Mar 2021.