New combo therapy aims to boost blood counts in bone marrow failure
NCT ID NCT01624805
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 trial tests a combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can improve blood counts and reduce transfusion needs. Participants must be 18 or older and not eligible for a stem cell transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- horse anti-thymocyte globulin (hATG), cyclosporine, methylprednisolone, and filgrastim or pegfilgrastim
- What this could lead to
- If successful, this combination could improve blood cell counts and reduce the need for transfusions in people with aplastic anemia or low-risk MDS.
- What could go wrong
- This is an early Phase 2 trial with only 140 participants, so results may not apply to everyone. The treatment suppresses the immune system, which can increase the risk of serious infections.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 140 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2012
- Expected to finish
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Jun 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Patients with the diagnosis of MDS (Low, Int-1 by IPSS, or hypocellular) who are either previously treated or untreated are eligible for this trial. 2. Patients with the diagnosis of aplastic anemia who are either previously treated or untreated are eligible if they are not currently candidates for an allogeneic stem cell transplant. 3. Patients ages 18 years and older are eligible 4. Patients must have been off of cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks prior to entering this study, and have recovered from the toxic effects of that therapy to grade 1 or less. 5. Adequate organ function as defined below: * liver function (bilirubin \< 2mg/dL, AST \<3 x ULN) * kidney function (creatinine \< 2.5 x ULN ). 6. ECOG performance status of ≤ 2. 7. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. 8. A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial. 9. Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol. 10. Patients should have an indication for therapy for their disease such as transfusion dependence or morbidity associated with their cytopenia(s) such as bleeding, severe fatigue, or frequent/multiple infections (eg. neutropenia). Exclusion Criteria: 1. Pregnant women are excluded from this study. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated on this study. 2. Known HIV infection. 3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. 4. Patient with documented hypersensitivity to any of the component medications.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
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Other studies related to the condition(s) this trial covers.
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- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
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- A nationwide effort to map a rare blood Cancer's toll