A Phase 2 interventional study of Bone marrow transplant and Thymoglobulin in Severe Aplastic Anemia and Bone Marrow Failure Syndromes, sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Completed at 2 sites in United States. Open to participants aged Up to 73 Years. Per ClinicalTrials.gov, last updated 2023-03-10.
Sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Phase 2, Interventional, and Treatment
Our primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.
This research is being done to find out if bone marrow transplantation (BMT) followed by chemotherapy will help people with aplastic anemia who have failed other treatments.
You have a severe, life threatening disease (severe aplastic anemia) in your bone marrow. Your disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like yours but complications from Graft vs. Host disease (GVHD) and graft failure have limited the survival for those people.
A small study done at Johns Hopkins has shown that in subjects with other diseases (blood cancers) some immunosuppressive drugs given after the BMT have decreased how often subjects had complications of GVHD and engraftment failure.
People with aplastic anemia who have refractory disease (not responding to standard treatment) may join.
371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.
This study's enrollment of 18 is below the median of 37 across 281 interventional studies indexed under Anemia, Aplastic.
Browse Anemia, Aplastic studies →Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins is the lead sponsor of 582 studies on the registry; 71 are open to participants now.
Of its 114 completed or terminated interventional studies of FDA-regulated products, 68 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients with SAA or very SAA defined:
Peripheral cytopenias (at least 2 of 3)
Adequate end-organ function as measured by:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Thymoglobulin on days -9 to -7 Fludarabine on days -6 to -2 Cyclophosphamide on days -6, -5, 3, 4 TBI on day -1 BMT on day 0 Mesna on days 3, 4 Tacrolimus on days 5-365 Mycophenolic acid mofetil on days 5-35
Procedure: Bone marrow transplant · Drug: Thymoglobulin · Drug: Fludarabine · Drug: Cyclophosphamide · Radiation: TBI · Drug: Mesna · Drug: Tacrolimus · Drug: Mycophenolic acid mofetil
Day 0
0.5 mg/kg IV on Day -9 2 mg/kg IV on Days -8, -7
30 mg/M2 IV on days -6 to -2
14.5 mg/kg IV on days -6, -5, 3, 4
Also known as: CTX
200 cGy on day -1
40 mg/kg IV on days 3, 4
For patients 18 years or older, tacrolimus will be given per institutional standards; may be increased or later changed to a PO BID schedule. Treatment to continue until Day 365 or longer if GVHD present
15 mg/kg PO/IV TID beginning on day 5 through day 35
Also known as: MMF
Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe?
Feasibility will be met with the following conditions: the patient has the transplant, is assessed for the safety endpoint, and survives one year. The safety monitoring plan is included to monitor graft failure (day 60), grade 2-4 acute graft versus host disease (day100), 6 month mortality (day 180), and chronic graft versus host disease (day 180).
Time frame: 1 year
Number of Patients That Have Survived at One Year
Time frame: 1 year
Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant
Donor chimerism will be measured in the peripheral blood around day 30 and day 60. Patients with \>5% donor chimerism around day 60 will be considered as having engrafted.
Time frame: 60 days
Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant
Time frame: 1 year
Number of Participants With Major Toxicities Related to Transplant
Time frame: 1 year
Number of Patients That Expired Due to Transplant Related Mortality
Time frame: 1 year
Number of Patients With Primary or Secondary Graft Failure Following Transplant
Graft failure: \< 5% donor chimerism in blood and/or bone marrow on \~Day 30 or after and on all subsequent measurements. Primary graft failure: \< 5% donor chimerism in blood and/or bone marrow by \~ Day 56 Secondary graft failure: achievement of \> 5% donor chimerism, followed by sustained \<5% donor chimerism in blood and/or bone marrow.
Time frame: 1 year
Number of Participants With Grade II-IV or Grade III-IV Acute GVHD
Participants were graded during clinical visits based on evidence and extent of skin rash, liver involvement, and GI tract involvement
Time frame: 1 year
Participants With Chronic GVHD at One Year
Time frame: 1 year
Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant
CBC drawn daily with a WBC differential once the total WBC is greater than 100 until ANC \> 500 for three days or two consecutive measurements over a three day period; then CBC drawn weekly with differential.
Time frame: 1 year
Participants That Were GVHD Free, Relapse Free Survival (GRFS)
Time frame: 1 year
| Milestone | Bone Marrow Transplant |
|---|---|
| Started | 18 |
| Completed | 16 |
| Not completed | 2 |
Feasibility will be met with the following conditions: the patient has the transplant, is assessed for the safety endpoint, and survives one year. The safety monitoring plan is included to monitor graft failure (day 60), grade 2-4 acute graft versus host disease (day100), 6 month mortality (day 180), and chronic graft versus host disease (day 180).
| Participants | Bone Marrow Transplant |
|---|---|
| Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe? | 18 |
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Patients That Have Survived at One Year | 18 |
Donor chimerism will be measured in the peripheral blood around day 30 and day 60. Patients with \>5% donor chimerism around day 60 will be considered as having engrafted.
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant | 7 |
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant | 0 |
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Participants With Major Toxicities Related to Transplant | 0 |
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Patients That Expired Due to Transplant Related Mortality | 0 |
Graft failure: \< 5% donor chimerism in blood and/or bone marrow on \~Day 30 or after and on all subsequent measurements. Primary graft failure: \< 5% donor chimerism in blood and/or bone marrow by \~ Day 56 Secondary graft failure: achievement of \> 5% donor chimerism, followed by sustained \<5% donor chimerism in blood and/or bone marrow.
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Patients With Primary or Secondary Graft Failure Following Transplant | 17 |
Participants were graded during clinical visits based on evidence and extent of skin rash, liver involvement, and GI tract involvement
| Participants | Bone Marrow Transplant |
|---|---|
| Number of Participants With Grade II-IV or Grade III-IV Acute GVHD | 17 |
| Participants | Bone Marrow Transplant |
|---|---|
| Participants With Chronic GVHD at One Year | 3 |
CBC drawn daily with a WBC differential once the total WBC is greater than 100 until ANC \> 500 for three days or two consecutive measurements over a three day period; then CBC drawn weekly with differential.
| days | Bone Marrow Transplant |
|---|---|
| Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant | 18.85 (14 to 48) |
| Participants | Bone Marrow Transplant |
|---|---|
| Participants That Were GVHD Free, Relapse Free Survival (GRFS) | 14 |
Collected over Adverse event data will be collected from day 0 of transplant through day 60 post transplant. All-Cause mortality is reported up to one year after transplant. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Bone Marrow Transplant | 0/18 (0%) | 7/18 (38.9%) | 0/18 (0%) |
| Event | Bone Marrow Transplant |
|---|---|
| Acute Respiratory Distress SyndromRespiratory, thoracic and mediastinal disorders | 1/18 |
| Acute Hypoxic Respiratory FailureRespiratory, thoracic and mediastinal disorders | 1/18 |
| Intracranial HemorrhageVascular disorders | 1/18 |
| Retroperitoneal HemorrhageVascular disorders | 1/18 |
| Hemorrhagic ShockVascular disorders | 1/18 |
| VomitingGastrointestinal disorders | 1/18 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/18 |
| Persistent FeversInfections and infestations | 1/18 |
| dysuria/hematuriaRenal and urinary disorders | 1/18 |
| Age, Categorical(Participants) | Bone Marrow Transplant |
|---|---|
| <=18 years | 5 |
| Between 18 and 65 years | 12 |
| >=65 years | 1 |
| Age, Continuous(years) | Bone Marrow Transplant |
|---|---|
| Mean | 30 (5 to 69) |
| Sex: Female, Male(Participants) | Bone Marrow Transplant |
|---|---|
| Female | 6 |
| Male | 12 |
| Race (NIH/OMB)(Participants) | Bone Marrow Transplant |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 13 |
| More than one race | 2 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | Bone Marrow Transplant |
|---|---|
| United States | 18 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Mar 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Thymoglobulin · Fludarabine · Cyclophosphamide · Mesna · Tacrolimus · Mycophenolic Acid
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins →