Phase 2 N=46 Treatment

T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts

Acute Lymphoblastic Leukemia · Non Hodgkins Lymphoma · Myelodysplastic Syndrome · Acute Myeloid Leukemia · Chronic Myelogenous Leukemia

Bottom Line

View on ClinicalTrials.gov: NCT00368355 ↗

Enrolled (actual)

Serious AEs

63.0%

Results posted

Jan 2020

Primary outcome: Primary: Engraftment Rate After Transplant — 100; 100 percentage of participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Ara-C (Drug); Cyclophosphamide (Drug); Campath-1H (Biological); Total Body Irradiation (Radiation); Stem Cell Infusion (Procedure)
Age: Pediatric, Adult
Sex: All
Sponsor: Baylor College of Medicine
Primary completion: Nov 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Engraftment Rate After Transplant	100; 100	—
SECONDARY Early Post BMT Toxicities	14; 14	—
SECONDARY Severe GVHD Rate	0; 4.3	—
SECONDARY Patients With Acute GVHD	7; 16; 3; 6; 0; 0	—
SECONDARY Patients With Chronic GVHD	10; 20; 0; 2	—

Summary

Subjects are being asked to participate in this study because treatment of their disease requires them to receive a stem cell transplant. Stem cells or "mother" cells are the source of normal blood cells and lead to recovery of blood counts after bone marrow transplantation (BMT). Unfortunately, there is not a perfectly matched stem cell donor (like a sister or brother) and the subject's disease is considered rapidly progressive and does not permit enough time to identify another donor (like someone from a registry list that is not their relative). We have, however, identified a close relative of the subject's whose stem cells are not a perfect match, but can be used. However, with this type of donor, there is typically an increased risk of developing graft-versus-host disease (GVHD), a high rate of transplant failure, and a longer delay in the recovery of the immune system. GVHD is a serious and sometimes fatal side effect of stem cell transplant. GVHD occurs when the new donor cells (graft) recognizes that the body tissues of the patient (host) are different from those of the donor. When this happens, cells in the graft may attack the host organs, primarily the skin, liver, and intestines. The number of occurrences and harshness of severe GVHD depends on several factors, including the degree of genetic differences between the donor and recipient, the intensity of the pre-treatment conditioning regimen, the quantity of transplanted cells, and the recipient's age. In recipients of mismatched family member or matched unrelated donor stem cell transplants, there is a greater risk of GVHD so that 70-90% of recipients of unchanged marrow will develop severe GVHD which could include symptoms such as marked diarrhea, liver failure, or even death. In an effort to lower the occurrences and severity of graft-versus-host disease in patients and to lower the rate of transplant failure, we would like to specially treat the donor's blood cells to remove cells that are most likely to attack the patient's tissues. This will occur in combination with intense conditioning treatment that the patient will receive before the transplant.

Eligibility Criteria

INCLUSION CRITERIA

Lack of suitable conventional donor (i.e. 5/6 or 6/6 related or 5/6 or 6/6 unrelated donor) or presence of a rapidly progressive disease not permitting time to identify an unrelated donor
Age less than or equal to 55 years of age
Patients with high risk ALL in CR1 or ALL or high grade (stage III or IV) NHL after first relapse or with primary refractory disease or minimal residual diseases.
Myelodysplastic syndrome
Patients with high risk AML in CR1 or after first relapse or with primary refractory disease or minimal residual disease.
CML
Hemophagocytic lymphohistiocytosis (HLH), familial hemophagocytic lymphohistiocytosis (FLH), viral-associated hemophagocytic syndrome (VAHS), X-linked lymphoproliferative disease (XLP), Severe chronic active Epstein Barr virus infection (SCAEBV) with predilection for T- or NK-cell malignancy
Donor cells should be collected and frozen before conditioning starts

EXCLUSION CRITERIA

Patients with a life expectancy (< / = 6 weeks) limited by diseases other than leukemia
Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction < 25%)
Patients with severe renal disease (i.e., creatinine clearance less than 40 cc/1.73 m^2)
Patients with pre-existing severe restrictive pulmonary disease (FVC less than 40% of predicted)
Patients with severe hepatic disease (direct bilirubin greater than 3 ug/dl or SGPT (serum glutamic-pyruvic transaminase) greater than 500 ug/dl)
Patients with severe personality disorder or mental illness
Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation
Patients with documented HIV positivity

'High risk' ALL or AML refers to those acute leukemias identified by the presence of specific biologic features, which predict high likelihood of failure to conventional chemotherapy. As biologic features of high risk disease evolve with improvement of conventional chemotherapy, it is not practical to define this indication with any further specificity. Therefore, high risk AML/ALL will be determined by the primary physician.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00368355). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.