Aplastic AnemiaPhase 2NCT01624805

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

A Phase 2 study testing Anti-Thymocyte Globulin, Cyclosporine and Filgrastim in people with Aplastic Anemia and de Novo Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Aplastic Anemia, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome
What is being tested?: Anti-Thymocyte Globulin, Cyclosporine and Filgrastim
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 140 participants
Where: 1 siteUnited States
Study dates: 25 Jun 2012to 30 Jun 2029
Phase: Phase 2
Are there results?: Not yet: the study is under way

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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.
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.
Patients ages 18 years and older are eligible
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.
Adequate organ function as defined below:
liver function (bilirubin < 2mg/dL, AST <3 x ULN)

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kidney function (creatinine < 2.5 x ULN ).
ECOG performance status of ≤ 2.
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.
A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.
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.
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).

Who cannot join

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.
Known HIV infection.
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.
Patient with documented hypersensitivity to any of the component medications.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Tapan Kadia, MD contact

713-563-3534

tkadia@mdanderson.org

United States1 site, 1 recruiting

M D Anderson Cancer Center
Houston, Texas, 77030
Tapan M. Kadia, 713-563-3534, tkadia@mdanderson.org
Tapan M. Kadia
Recruiting

Other trials in United States

About this study

Official title: Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)

This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine, filgrastim, and/or pegfilgrastim or pegfilgrastim biosimilar in treating patients with aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte globulin is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in aplastic anemia and in some cases of myelodysplastic syndromes, killing these cells may help treat the disease. Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in aplastic anemia and myelodysplastic syndromes. Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count. Giving methylprednisolone and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and/or pegfilgrastim may be an effective treatment for patients with aplastic anemia or myelodysplastic syndrome.

Read the detailed description

PRIMARY OBJECTIVES:

To evaluate the efficacy of the combination of hATG (horse anti-thymocyte globulin), methylprednisolone, cyclosporine, and GCSF (filgrastim) in achieving response (complete response [CR], partial response [PR], or hematologic improvement [HI]) in patients with aplastic anemia, or myelodysplastic syndromes (MDS).

SECONDARY OBJECTIVES:

To assess the safety, tolerability, and toxicities of the combination of hATG, methylprednisolone, cyclosporine, and GCSF in patients with aplastic anemia, or MDS. II. To assess time to response, response duration, and overall survival of patients with aplastic anemia, or MDS being treated with the combination of hATG, methylprednisolone, cyclosporine, and GCSF.

OUTLINE:

Patients receive methylprednisolone intravenously (IV) over 10 minutes on days 1-4 and IV or orally (PO) with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV over 8 hours daily on days 1-4, cyclosporine PO twice daily (BID) on days 1-180, and pegfilgrastim or pegfilgrastim biosimilar subcutaneously (SC) on day 5 and/or filgrastim SC beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6-12 months.

Conditions: Aplastic Anemia, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome
Being tested: Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, Methylprednisolone, Pegfilgrastim
Medical subject headings: Anemia, Aplastic, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Anti-Thymocyte GlobulinBiological
Also called Antithymocyte Globulin, Antithymocyte Serum, ATG, ATGAM
Given IV
CyclosporineDrug
Also called 27-400, Ciclosporin, CsA, Cyclosporin
Given PO
FilgrastimBiological
Also called FILGRASTIM, LICENSE HOLDER UNSPECIFIED, G-CSF, Neupogen, r-metHuG-CSF
Given SC
MethylprednisoloneDrug
Also called Adlone, Caberdelta M, DepMedalone, Depo Moderin
Given IV or PO
PegfilgrastimBiological
Also called Filgrastim SD-01, filgrastim-SD/01, Fulphila, HSP-130
Given SC

Study groups in NCT01624805
Study group	Role	What they receive
Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)	Experimental	Patients receive methylprednisolone IV over 10 minutes on days 1-4 and IV or PO with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV over 8 hours daily on days 1-4, cyclosporine PO BID on days 1-180, and pegfilgrastim or pegfilgrastim biosimilar SC on day 5 and/or filgrastim SC beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity. Receives: Biological: Anti-Thymocyte Globulin; Drug: Cyclosporine; Biological: Filgrastim; Drug: Methylprednisolone; Biological: Pegfilgrastim

What is being measured

The primary outcome is the main question the study is designed to answer.

Achievement of response
Measured by complete response (CR), partial response, or hematologic improvement.
Measured: Up to 6 years

Secondary outcomes (4)

Time to response
Estimated according to the Kaplan-Meier method.
Measured: The interval between treatment start and the date of response, assessed up to 6 years
Duration of CR
Estimated according to the Kaplan-Meier method.
Measured: Time interval between the date of CR to the date of first evidence of disease recurrence or death, assessed up to 6 years
Overall survival
Estimated according to the Kaplan-Meier method.
Measured: Time from treatment start until the death or last follow-up time, assessed up to 6 years
Incidence of adverse events
Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. Tabulated by grade and course of therapy. Numbers of required dose reductions will be included in the toxicity report.
Measured: Up to 6 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 Jun 201230 Jun 2029

First posted21 Jun 2012 (estimated)
Study start25 Jun 2012
Primary completion30 Jun 2029 (estimated)
Study completion30 Jun 2029 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
More trials run by M.D. Anderson Cancer CenterThe 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: 140 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01624805
Sponsor's protocol ID: 2012-0334
Other IDs: NCI-2012-01096, 2012-0334
Status last verified: Jun 2026
First submitted: 19 Jun 2012
Last updated: 22 Jun 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Interested in taking part?

Tapan Kadia, MD

713-563-3534

tkadia@mdanderson.org

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See sites and contacts

RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

All Aplastic Anemia trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)
ClinicalTrials.gov ID: NCT01624805
Acronym / sponsor ID: 2012-0334
Conditions: Aplastic Anemia, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Recruiting
Enrolment: 140 participants (estimated)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 25 Jun 2012 (actual)
Primary completion: 30 Jun 2029 (estimated)
Study completion: 30 Jun 2029 (estimated)
First posted: 21 Jun 2012 (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.

Detailed description

PRIMARY OBJECTIVES:

To evaluate the efficacy of the combination of hATG (horse anti-thymocyte globulin), methylprednisolone, cyclosporine, and GCSF (filgrastim) in achieving response (complete response [CR], partial response [PR], or hematologic improvement [HI]) in patients with aplastic anemia, or myelodysplastic syndromes (MDS).

SECONDARY OBJECTIVES:

To assess the safety, tolerability, and toxicities of the combination of hATG, methylprednisolone, cyclosporine, and GCSF in patients with aplastic anemia, or MDS. II. To assess time to response, response duration, and overall survival of patients with aplastic anemia, or MDS being treated with the combination of hATG, methylprednisolone, cyclosporine, and GCSF.

OUTLINE:

After completion of study treatment, patients are followed up every 6-12 months.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)	Experimental	Biological: Anti-Thymocyte Globulin, Drug: Cyclosporine, Biological: Filgrastim, Drug: Methylprednisolone, Biological: PegfilgrastimPatients receive methylprednisolone IV over 10 minutes on days 1-4 and IV or PO with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV over 8 hours daily on days 1-4, cyclosporine PO BID on days 1-180, and pegfilgrastim or pegfilgrastim biosimilar SC on day 5 and/or filgrastim SC beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (12 listed)

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.
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.
Patients ages 18 years and older are eligible
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.
Adequate organ function as defined below:

Key exclusion criteria (4 listed)

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.
Known HIV infection.
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.
Patient with documented hypersensitivity to any of the component medications.

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
Anti-Thymocyte GlobulinAlso: Antithymocyte Globulin, Antithymocyte Serum, ATG, ATGAMGiven IV	Biological	Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
CyclosporineAlso: 27-400, Ciclosporin, CsA, CyclosporinGiven PO	Drug	Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
FilgrastimAlso: FILGRASTIM, LICENSE HOLDER UNSPECIFIED, G-CSF, Neupogen, r-metHuG-CSFGiven SC	Biological	Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
MethylprednisoloneAlso: Adlone, Caberdelta M, DepMedalone, Depo ModerinGiven IV or PO	Drug	Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
PegfilgrastimAlso: Filgrastim SD-01, filgrastim-SD/01, Fulphila, HSP-130Given SC	Biological	Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)

Primary outcome

Achievement of response
Time frame: Up to 6 years
Measured by complete response (CR), partial response, or hematologic improvement.

4 secondary outcomes

Time to response
Time frame: The interval between treatment start and the date of response, assessed up to 6 years
Duration of CR
Time frame: Time interval between the date of CR to the date of first evidence of disease recurrence or death, assessed up to 6 years
Overall survival
Time frame: Time from treatment start until the death or last follow-up time, assessed up to 6 years
Incidence of adverse events
Time frame: Up to 6 years

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
MD Anderson Cancer CenterLink provided by the sponsor.

Related trials for Aplastic Anemia

RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

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.

Tapan Kadia, MD contact

713-563-3534

tkadia@mdanderson.org

Tapan M Kadia principal investigator

M.D. Anderson Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 12 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 22 Jun 2026. 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Tapan Kadia, MD (713-563-3534, tkadia@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Achievement of response (time frame: Up to 6 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 NCT01624805.

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 22 Jun 2026.