A Phase 2 interventional study of CD34+ in Waning Donor Chimerism, Waning Immune Function and Primary Immunodeficiency Disease(s), sponsored by Children's Hospital Medical Center, Cincinnati. Terminated at 1 site in United States. Open to participants aged Up to 35 Years. Per ClinicalTrials.gov, last updated 2019-11-22.
Sponsored by Children's Hospital Medical Center, Cincinnati · Phase 2, Interventional, and Treatment
Among 67 studies in Bone Marrow Failure Disorders with a site in United States — 21 open to participants now, 29 under way, none of them phase 3. Bone Marrow Failure Disorders · United States.
Among the 67 studies in Bone Marrow Failure Disorders with a site in United States, this one is the 28th largest by enrollment of the 55 interventional ones that give a count, is the only one that started in 2010, is one of 25 in phase 2, and was the 23rd registered.
91 studies on the registry are indexed under Bone Marrow Failure Disorders; 29 are open to participants now.
Browse Bone Marrow Failure Disorders studies →21 of the 67 studies in Bone Marrow Failure Disorders with a site in United States are open now. Recruiting first, the newest first:
St. Jude Children's Research Hospital · Sites in United States
Dren Bio · Sites in United States
Medical College of Wisconsin · Sites in United States
Nationwide Children's Hospital · Sites in United States
Suneet Agarwal · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 23 Bone Marrow Failure Disorders studies recruiting now, 9 (39%) set no upper age limit and 12 (52%) stop at 75 or younger; 19 take children under 18; 2 take healthy volunteers.
This study: Up to 35 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
67 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 20 · Phase 2 37 · Phase 3 3
First in 1986; this study’s year is marked. 1 started in 2010.
This study's enrollment of 23 is below the median of 26 across 69 interventional studies indexed under Bone Marrow Failure Disorders.
Median enrollment 23 across the 55 interventional studies, 325 across the 10 observational. Of the 32 that completed or were terminated, 15 (47%) have results posted.
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5 of 67 have an industry lead sponsor. The 10 most frequent:
Children's Hospital Medical Center, Cincinnati is the lead sponsor of 673 studies on the registry; 130 are open to participants now.
Of its 57 completed or terminated interventional studies of FDA-regulated products, 31 (54%) have results posted.
Children's Hospital Medical Center, Cincinnati has 1 study filed under Bone Marrow Failure Disorders — fewer than ten, so there is no comparison to draw.
6 of the studies in Bone Marrow Failure Disorders with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01659606Boston Children's HospitalRadiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita | 2026-12 |
| NCT04644016Memorial Sloan Kettering Cancer CenterCord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders | 2026-12-20 |
| NCT02356653Children's Hospital of PhiladelphiaExpanded Access Protocol Using CD3+/CD19+ Depleted PBSC | 2027-01 |
| NCT03579875Masonic Cancer Center, University of MinnesotaAlpha/Beta TCD HCT in Patients With Inherited BMF Disorders | 2027-01-01 |
| NCT04356469Johns Hopkins All Children's HospitalTCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children | 2027-06-30 |
Among the 67 studies in Bone Marrow Failure Disorders with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to determine if infusing additional special donor cells will help to improve graft or immune function in previously transplanted children with immune deficiencies and bone marrow failures.
The purpose of this study is to investigate the usefulness of infusing purified CD34+ cells of donor origin in order to augment graft function in response to declining chimerism after initially performing an allogeneic hematopoietic stem cell transplant (HSCT) for children with primary immunodeficiency diseases. This protocol will be utilized for patients with waning mixed donor chimerism that is inadequate for correction of clinical condition or disease for which stem cell transplant was performed, or for augmentation of immune function. An infusion of selected CD34+ stem cells will be given without any preparative regimen. As the children eligible for this protocol have reduced immune function and pre-existing donor chimerism, we hypothesize that stem cells will be able to engraft and the infusion will augment graft function. This therapy serves as an alternative to a second stem cell transplant that is known to be associated with significant morbidity and mortality. CD34+ stem cells will be collected from the donor used for initial stem cell transplant. Cells will be T-cell depleted (TCD) by performing a CD34 selection using the CliniMACS device (Miltenyi Biotec) in order to prevent development of new or exacerbation of existing graft versus host disease (GVHD), as avoidance of GVHD in nonmalignant diseases is desirable. There is sufficient data showing that mixed donor chimerism is adequate for reverting disease phenotype in certain primary immunodeficiencies. Observations from Europe and CCHMC show that donor chimerism might be boosted by CD34+ stem cell infusion alone without any specific preparative regimen. This therapy is likely to be associated with low toxicity due to the absence of a preparative regimen and lack of exposure to fresh donor cells capable of initiating GVHD, and offers potential significant benefit.
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.
To be eligible for this protocol, patients must have the following:
One of the following must be true:
-OR-
-OR-
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
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.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
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.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
Biological: CD34+
CD34+ cells are selected using the CliniMACS System; without preparative regimen
Number of Participants Who Showed Successful Augmentation of Graft Function
Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.
Time frame: 12 months
| Milestone | CD34+ Selected Stem Cell Infusion |
|---|---|
| Started | 23 |
| Completed | 22 |
| Not completed | 1 |
| Withdrew: Died prior to boost infusion | 1 |
Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.
| Participants | CD34+ Selected Stem Cell Infusion |
|---|---|
| Number of Participants Who Showed Successful Augmentation of Graft Function | 3 |
Collected over All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CD34+ Selected Stem Cell Infusion | 3/23 (13%) | 8/23 (34.8%) | 2/23 (8.7%) |
| Event | CD34+ Selected Stem Cell Infusion |
|---|---|
| InfectionInfections and infestations | 4/23 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 2/23 |
| HypotensionCardiac disorders | 2/23 |
| Multiorgan FailureGeneral disorders | 2/23 |
| Chronic SinusitisRespiratory, thoracic and mediastinal disorders | 1/23 |
| Respiratory FailureRespiratory, thoracic and mediastinal disorders | 1/23 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 1/23 |
| Renal FailureRenal and urinary disorders | 1/23 |
| EncephalopathyNervous system disorders | 1/23 |
| AscitesGastrointestinal disorders | 1/23 |
| Event | CD34+ Selected Stem Cell Infusion |
|---|---|
| HemolysisCardiac disorders | 1/23 |
| HemorrhageInjury, poisoning and procedural complications | 1/23 |
| Febrile NeutropeniaInfections and infestations | 1/23 |
| Age, Categorical(Participants) | CD34+ Selected Stem Cell Infusion |
|---|---|
| <=18 years | 16 |
| Between 18 and 65 years | 7 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | CD34+ Selected Stem Cell Infusion |
|---|---|
| Female | 5 |
| Male | 18 |
| Race (NIH/OMB)(Participants) | CD34+ Selected Stem Cell Infusion |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 18 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
| Region of Enrollment(participants) | CD34+ Selected Stem Cell Infusion |
|---|---|
| United States | 23 |
PDFs hosted by the registry, ClinicalTrials.gov.
The record gives a start of Oct 2010, a primary completion of Aug 2018 and a completion of Aug 2018. It does not say whether these dates are actual or estimated.
Results were first posted in Oct 2019, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Dec 2018. You cannot join it, but the record below documents what was studied.
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Bone Marrow Failure Disorders →
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