Successful Neutrophil Engraftment
Primary
· 2 years
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 44 | |
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 1 |
Last reviewed · How we verify
NCT00987480
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Completed
Phase 2
Results posted
Last updated 10 July 2018
What this trial tests
Phase 2 trial testing Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine. in Aplastic Anemia in 45 participants. Completed in 10 July 2017.
Timeline
25 September 2009
Primary endpoint
10 July 2017
10 July 2017
10 July 2017
Quick facts
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 45 |
| Start date | 25 September 2009 |
| Primary completion | 10 July 2017 |
| Estimated completion | 10 July 2017 |
| Sites | 6 locations across United States |
Drugs / interventions tested
- Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine. — full drug profile →
- CliniMACS device
Conditions studied
- Aplastic Anemia — all drugs for Aplastic Anemia →
- Leukemia — all drugs for Leukemia →
- Myelodysplastic Syndrome — all drugs for Myelodysplastic Syndrome →
Sponsor
Memorial Sloan Kettering Cancer Center — full company profile →
Who can join
Eligibility, any sex, with Aplastic Anemia or Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
The Incidence of Early Transplant Related Mortality
Primary
· 2 years
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 0 |
The Incidence of Acute GvHD
Primary
· 100 days
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 6.7 |
The Incidence of Chronic GvHD
Primary
· 2 years
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 3 | |
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 42 |
Overall Survival at 3 Years
Secondary
· 3 years
Overall Survival is defined as time from date of transplant to event (death from any cause) or last follow-up.
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 80 |
Disease-free Survival at 3 Years
Secondary
· 3 years
Defined as time from date of transplant to relapse, graft rejection or graft failure, or death. Primary non-engraftment is diagnosed when the participants fails to achieve an ANC \>/= 500/ul at any time in the first 28 days post-transplant. For participants with MDS or AML, relapse will be analyzed as to type and genetic origin of the MDS/leukemic cells. These will be defined by an increasing number of blasts in the marrow over 5% by the presence of circulating peripheral blasts, or by the presence of blasts in any extramedullary site. Cytogenetic analysis of the marrow and/or peripheral blo
| Group | Value | 95% CI |
|---|---|---|
| Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected | 77.8 |
Adverse events — posted to ClinicalTrials.gov
Time frame: 1 year. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
Serious: 11/45 (24%)
Deaths: 9/45
Serious adverse events (24 terms)
| Reaction | System | Chemotherapy-based Cytored… |
|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — |
| Infection, other | Infections and infestations | — |
| Death NOS | General disorders | — |
| Pneumonitis/pulm infiltrates | Respiratory, thoracic and mediastinal disorders | — |
| Bilirubin (hyperbilirubinemia) | Investigations | — |
| Blood/Bone Marrow, Other | Blood and lymphatic system disorders | — |
| Cardiopulmonary arrest, cause unknkown | Cardiac disorders | — |
| Confusion | Psychiatric disorders | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — |
| Gastrointestinal, other | Gastrointestinal disorders | — |
| Hemorrhage, Oral cavity | Gastrointestinal disorders | — |
| Hemorrhage, Respiratory tract NOS | Respiratory, thoracic and mediastinal disorders | — |
| Hemorrhage, Stoma (GI) | Gastrointestinal disorders | — |
| Ileus, GI (func obstruction of bowel) | Gastrointestinal disorders | — |
| Inf norm ANC/gr1/2 neut-Blood | Infections and infestations | — |
| Inf norm ANC/gr1/2 neut-Meningitis(meninges) | Infections and infestations | — |
| Inf norm ANC/gr1/2 neut-Myositis infection(muscle) | Infections and infestations | — |
| Infection w/ Gr 3/4 neut, Blood | Infections and infestations | — |
| Mucositis (Clin exam)- Oral cavity | Gastrointestinal disorders | — |
| Neurology - Other (specify) | Nervous system disorders | — |
| Neuropathy: motor | Nervous system disorders | — |
| Pulm/upp respiratory - Other (spec) | Respiratory, thoracic and mediastinal disorders | — |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | — |
| Seizure | Nervous system disorders | — |
Other adverse events (33 terms — click to expand)
| Reaction | System | Chemotherapy-based Cytored… |
|---|---|---|
| Mucositis (Clin exam)- Oral cavity | Gastrointestinal disorders | — |
| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | — |
| ALT, SGPT | Investigations | — |
| Bilirubin (hyperbilirubinemia) | Investigations | — |
| AST, SGOT | Investigations | — |
| Potassium, low (hypokalemia) | Metabolism and nutrition disorders | — |
| Sodium, low (hyponatremia) | Metabolism and nutrition disorders | — |
| Albumin, low (hypoalbuminemia) | Metabolism and nutrition disorders | — |
| Creatinine | Investigations | — |
| Hemoglobin | Investigations | — |
| Hypertension | Vascular disorders | — |
| Leukocytes (total WBC) | Investigations | — |
| Lymphopenia | Blood and lymphatic system disorders | — |
| Neutrophils/granulocytes (ANC/AGC) | Investigations | — |
| Platelets | Investigations | — |
| Alkaline phosphatase | Investigations | — |
| Potassium, high (hyperkalemia) | Metabolism and nutrition disorders | — |
| PTT | Investigations | — |
| Hyperglycemia | Metabolism and nutrition disorders | — |
| INR | Investigations | — |
| Magnesium, high (hypermagnesemia) | Metabolism and nutrition disorders | — |
| Magnesium, low (hypomagnesemia) | Metabolism and nutrition disorders | — |
| Trglycrde, high (hypertriglyceridemia) | Metabolism and nutrition disorders | — |
| Alanine aminotransferase increased | Investigations | — |
| Infection, other | Infections and infestations | — |
| Glucose, low (hypoglycemia) | Metabolism and nutrition disorders | — |
| Hypermagnesemia | Metabolism and nutrition disorders | — |
| Mucositis (Clin exam)- Pharynx | Gastrointestinal disorders | — |
| Phosphate, low (hypophosphatemia) | Metabolism and nutrition disorders | — |
| Aspartate aminotransferase increased | Investigations | — |
| Bicarbonate, serum-low | Investigations | — |
| Blood bilirubin increased | Investigations | — |
| Sodium, high (hypernatremia) | Metabolism and nutrition disorders | — |
Most-reported serious reactions: Hypoxia, Infection, other, Death NOS, Pneumonitis/pulm infiltrates, Bilirubin (hyperbilirubinemia), Blood/Bone Marrow, Other, Cardiopulmonary arrest, cause unknkown, Confusion.
Data from ClinicalTrials.gov NCT00987480 adverse events section.
Sponsor's own description
This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic syndrome (MDS) or leukemia (acute myelogenous leukemia or AML). Without treatment these complications of Fanconia anemia (FA) are fatal. The only treatment that can cure these complications is an allogeneic transplant of stem cells, meaning, giving the patient bone marrow cells from a healthy donor that can produce normal blood cells that will replace the bone marrow that is sick. What has been given for the treatment of FA in the past is to use a combination of low doses of radiation to the whole body (total body irradiation) and low doses of the chemotherapy drugs (cyclophosphamide and fludarabine) before the transplant. However, the use of radiation can, later on, increase the chances of getting a second cancer of the skin, head or the neck. These chances of a second cancer are higher than normal in patients with FA. The purpose of this study is to find out if the doctors can do the same thing with the same chemotherapy drugs used in the past. However physicians will use another chemotherapy drug called busulfan instead of the radiation. The goal of this study is to get rid of the short term and long term risks of the radiation. The first new part of this treatment will be to replace drugs for radiation with chemotherapy drugs.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Cyclophosphamide-based in vivo T-cell depletion for HLA-haploidentical transplantation in Fanconi anemia.
Thakar MS, Bonfim C, Sandmaier BM, O'Donnell P, et al · · 2012 · cited 18× · PMID 22839094 · DOI 10.3109/08880018.2012.708708 -
Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Agarwal S. · · 2023 · cited 2× · PMID 38066900 · DOI 10.1182/hematology.2023000470
Verify or expand the search:
- PubMed search for NCT00987480
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Aplastic Anemia
Currently open trials in the same condition.
- NCT07101770 — A Prediction Model in Pregnant Women With Aplastic Anemia · recruiting
- NCT06752694 — Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult P · Phase 2 · recruiting
- NCT06936930 — Reproductive and Mental Health of Patients With Aplastic Anemia · recruiting
- NCT06689800 — Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy · NA · recruiting
- NCT06613880 — Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia · Phase 2 · recruiting
Other Memorial Sloan Kettering Cancer Center trials
Trials by the same sponsor.
- NCT07566377 — Cord Blood Transplantation in Children and Young Adults With Blood Cancer · Phase 2 · recruiting
- NCT07507643 — A Study of Methylprednisolone in People Having Liver Surgery · Phase 3 · recruiting
- NCT07483307 — A Study of the Impact of Endocrine Therapy on Surgical Outcomes in People With Breast Cancer · Phase 2 · recruiting
- NCT07459673 — HERizon-Breast: A ctDNA-Guided Adaptive Study of Sequential Anti-HER2 Therapies and CNS Prophylaxis to Induce Long-Term · Phase 2 · recruiting
- NCT07449195 — A Study Comparing Two Optical Coherence Tomography Devices in People With Ocular Tumors · NA · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT00987480 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Memorial Sloan Kettering Cancer Center
- Last refreshed: 10 July 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00987480.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing