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SCID Bu/Flu/ATG Study With T Cell Depletion
Phase 1, PHASE2 trial testing unrelated BM with T cell depletion in Severe Combined Immunodeficiency in 9 participants. Terminated before completion.
| Lead sponsor | Neena Kapoor, M.D. |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 9 |
| Start date | 2 January 2007 |
| Primary completion | 1 August 2016 |
| Estimated completion | 1 August 2016 |
| Sites | 1 location across United States |
Neena Kapoor, M.D. — full company profile →
Under 21, any sex, with Severe Combined Immunodeficiency. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
| Group | Value | 95% CI |
|---|---|---|
| Unrelated BM With T Cell Depletion | 7 | |
| Unrelated Cord Blood | 2 |
Absolute number of donor-derived CD3+ T lymphocytes \>/= 100/mm3 in participating subjects.
| Group | Value | 95% CI |
|---|---|---|
| Unrelated BM With T Cell Depletion | 7 | |
| Unrelated Cord Blood | 1 | |
| Haplo BM With T Cell Depletion | 0 | |
| Unrelated PBSC With T Cell Depletion | 0 |
Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia
| Group | Value | 95% CI |
|---|---|---|
| Unrelated BM With T Cell Depletion | 2 | |
| Unrelated Cord Blood | 1 | |
| Haplo BM With T Cell Depletion | 0 | |
| Unrelated PBSC With T Cell Depletion | 0 |
GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.
| Group | Value | 95% CI |
|---|---|---|
| Unrelated BM With T Cell Depletion | 1 | |
| Unrelated Cord Blood | 0 | |
| Haplo BM With T Cell Depletion | 0 | |
| Unrelated PBSC With T Cell Depletion | 0 |
Overalls survival of patient at 1 year post transplant
| Group | Value | 95% CI |
|---|---|---|
| Unrelated BM With T Cell Depletion | 6 | |
| Unrelated Cord Blood | 1 | |
| Haplo BM With T Cell Depletion | 0 | |
| Unrelated PBSC With T Cell Depletion | 0 |
Time frame: Adverse event data was collected over a period of 1 year post transplant.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Unrelated BM With T Cell D… | Unrelated Cord Blood | Haplo BM With T Cell Deple… | Unrelated PBSC With T Cell… |
|---|---|---|---|---|---|
| Sinusoidal obstruction syndrome | Hepatobiliary disorders | — | — | — | — |
| Reaction | System | Unrelated BM With T Cell D… | Unrelated Cord Blood | Haplo BM With T Cell Deple… | Unrelated PBSC With T Cell… |
|---|---|---|---|---|---|
| Graft Versus Host Disease | Skin and subcutaneous tissue disorders | — | — | — | — |
| Infection - Aspergillis | Infections and infestations | — | — | — | — |
| Infection - Fungal | Infections and infestations | — | — | — | — |
| Pulmonary Hemorrhage | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Seizure | Nervous system disorders | — | — | — | — |
Most-reported serious reactions: Sinusoidal obstruction syndrome.
Data from ClinicalTrials.gov NCT02127892 adverse events section.
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD), 2) unrelated cord blood donor, or 3) a haplo-identical (parental) donor (in descending order of preference).Patients will receive a novel conditioning regimen with Busulfan, Fludarabine and Anti-thymocyte globulin (ATG) followed by an unrelated donor hematopoietic stem cell transplant (HSCT) with T-cell depletion using the CliniMACS device.
No peer-reviewed publications indexed yet for this trial.
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