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Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Phase 2 trial testing anti-thymocyte globulin in Fanconi Anemia in 14 participants. Completed in 10 October 2020.
| Lead sponsor | Masonic Cancer Center, University of Minnesota |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 14 |
| Start date | 26 March 2002 |
| Primary completion | 10 October 2020 |
| Estimated completion | 10 October 2020 |
| Sites | 1 location across United States |
Masonic Cancer Center, University of Minnesota
Under 44, any sex, with Fanconi Anemia. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Graft failure is defined as absolute neutrophil count( ANC ) \<5 x 10\^8/L by day 30.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 1 | |
| USB Arm | 0 | |
| Marrow Clinimacs | 0 | |
| Sibling withoutCliniMACS | 0 |
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 0 | |
| USB Arm | 0 | |
| Marrow Clinimacs | 0 | |
| Sibling withoutCliniMACS | 0 |
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 0 | |
| USB Arm | 0 | |
| Marrow Clinimacs | 0 | |
| Sibling withoutCliniMACS | 0 |
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 0 | |
| USB Arm | 1 | |
| Marrow Clinimacs | 0 | |
| Sibling withoutCliniMACS | 0 |
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 0 | |
| USB Arm | 0 | |
| Marrow Clinimacs | 0 | |
| Sibling withoutCliniMACS | 0 |
Patients with leukemia will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate.
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 0 | |
| USB Arm | 2 | |
| Marrow Clinimacs | 1 | |
| Sibling withoutCliniMACS | 0 |
Overall Survival - Number of patients alive at 1 year post transplant
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 1 | |
| USB Arm | 4 | |
| Marrow Clinimacs | 1 | |
| Sibling withoutCliniMACS | 1 |
Number of participants experiencing Major Infections by the end of treatment
| Group | Value | 95% CI |
|---|---|---|
| Marrow Isolex | 3 | |
| USB Arm | 8 | |
| Marrow Clinimacs | 2 | |
| Sibling withoutCliniMACS | 1 |
Time frame: 1 year. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Marrow Isolex | USB Arm | Marrow Clinimacs | Sibling withoutCliniMACS |
|---|---|---|---|---|---|
| Death | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — | — | — | — |
| diffuse alveolar hemorrhage | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Primary Graft Failure | General disorders | — | — | — | — |
| Relapse | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — | — | — | — |
| Reaction | System | Marrow Isolex | USB Arm | Marrow Clinimacs | Sibling withoutCliniMACS |
|---|---|---|---|---|---|
| Infection | Infections and infestations | — | — | — | — |
| Pneumonia | Infections and infestations | — | — | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — | — | — |
| Engraftment syndrome | General disorders | — | — | — | — |
| Hypertension | Vascular disorders | — | — | — | — |
| Intubation | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Veno occlusive disease | Hepatobiliary disorders | — | — | — | — |
| Acute hypoxic respiratory failure | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Chest Tube Placement | Surgical and medical procedures | — | — | — | — |
| Chest Xray | Surgical and medical procedures | — | — | — | — |
| Coagulopathy | Blood and lymphatic system disorders | — | — | — | — |
| Dialysis | Renal and urinary disorders | — | — | — | — |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | — | — | — | — |
| Drug Rash with Eosinophilia | General disorders | — | — | — | — |
| Eosinophilic gut | Gastrointestinal disorders | — | — | — | — |
| GI bleeding | Gastrointestinal disorders | — | — | — | — |
| hearing loss | Ear and labyrinth disorders | — | — | — | — |
| pericardial effusion | Cardiac disorders | — | — | — | — |
| Pleuracentesis | Surgical and medical procedures | — | — | — | — |
| Pneumoperitoneum | Gastrointestinal disorders | — | — | — | — |
| Primary Graft Failure | General disorders | — | — | — | — |
| pulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Renal failure | Renal and urinary disorders | — | — | — | — |
| Respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | — | — | — | — |
| Thrush | Infections and infestations | — | — | — | — |
| Unresponsive | Nervous system disorders | — | — | — | — |
Most-reported serious reactions: Death, diffuse alveolar hemorrhage, Primary Graft Failure, Relapse.
Data from ClinicalTrials.gov NCT00258427 adverse events section.
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia. PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
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