A Phase 2 interventional study of CliniMACs device in Bone Marrow Failure Syndromes, Immunodeficiencies and Immune Dysregulation Syndromes, sponsored by Children's Hospital of Philadelphia. Completed at 1 site in United States. Open to participants aged Up to 22 Years. Per ClinicalTrials.gov, last updated 2018-04-11.
Sponsored by Children's Hospital of Philadelphia · 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 51st largest by enrollment of the 55 interventional ones that give a count, is one of 3 that started in 2011, is one of 25 in phase 2, and was the 28th 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 22 Years · all sexes · no healthy volunteers.
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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. 3 started in 2011.
This study's enrollment of 2 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 of Philadelphia is the lead sponsor of 489 studies on the registry; 90 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
Children's Hospital of Philadelphia has 5 studies 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:
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This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors. There are two conditioning regimens depending upon patient diagnosis and age.
This study will allow transplantation using a reduced intensity conditioning regimen for children with non-malignant diseases who lack a matched related or unrelated donor. Donors will be unrelated or partially matched related, depending upon urgency and availability. If each parent is haploidentical, the mother will be preferred, as there is evidence of reduced transplant related mortality and superior survival with a maternal donor. The risks of severe graft vs host disease (GVHD) and Epstein-Barr lymphoproliferative disorder will be reduced or eliminated by T and B cell depletion using the Miltenyi Clinimacs device. Patients with bone marrow failure syndromes, who are at high risk for rejection, will undergo pre-conditioning immune suppression with Thymoglobulin. It is recommended that patients with immunedysregulation syndromes receive pre-RIC alemtuzumab as this may reduce the risk on non-engraftment and hyperinflammatory states.
Post-transplant immune suppression will be used to prevent GVHD, as CD3 depletion does not deplete as completely as CD34+ selection. It will be rapidly weaned if no GVHD by day 100 to allow immune reconstitution.
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.
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
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.
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.
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
Device: CliniMACs device
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
Device: CliniMACs device
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
Number of Participants With Engraftment
The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.
Time frame: One Year
| Milestone | Bone Marrow Failure Syndrome | Immunodeficiency / Dysregulation |
|---|---|---|
| Started | 0 | 2 |
| Completed | 0 | 2 |
| Not completed | 0 | 0 |
The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.
| Participants | Bone Marrow Failure Syndrome | Immunodeficiency / Dysregulation |
|---|---|---|
| Number of Participants With Engraftment | — | 2 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Bone Marrow Failure Syndrome | — | — | — |
| Immunodeficiency / Dysregulation | 0/2 (0%) | 0/2 (0%) | 0/2 (0%) |
| Age, Continuous(years) | Bone Marrow Failure Syndrome | Immunodeficiency / Dysregulation | Total |
|---|---|---|---|
| Median | — | 9.5 (4 to 15) | 9.5 (4 to 15) |
| Sex: Female, Male(Participants) | Bone Marrow Failure Syndrome | Immunodeficiency / Dysregulation | Total |
|---|---|---|---|
| Female | — | 1 | 1 |
| Male | — | 1 | 1 |
The record gives a start of Nov 2011, a primary completion of Mar 2016 and a completion of Mar 2016. It does not say whether these dates are actual or estimated.
Results were first posted in Apr 2018, 2 years 1 month after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 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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