CompletedPhase 2/3

Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

United States38 participantsStarted 2002-09

Plain-language summary

This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in a population of patients with non-malignant diseases (non-cancer). Eligible patients will have a non-malignant disease in one of the following four strata: bone marrow failure syndromes, immunodeﬁciencies, inborn errors of metabolism, or histiocytoses. Patients will be assigned to therapy according to diagnosis. Patients will be stratiﬁed by disease into one of four strata and treatment regimens will be based on speciﬁc disease criteria and conditions. Although these diseases are non-malignant in name, they are often malignant by nature of the disease progression, treatment and associated complications.

Who can participate

Age range30 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria Patients must meet the eligibility criteria for organ function regardless of diagnosis: * Age \< 30 or = 30 years of age * Adequate renal function defined as serum creatinine \< or = 1.5 x normal, or creatinine clearance or radioisotope glomerular filtration rate (GFR) \> or =40 ml/min/m2 or \>60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range * Adequate liver function defined as serum glutamic oxaloacetic transaminase (SGOT)(Aspartate transaminase (AST)) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase (ALT)) \< 5.0 x normal * Adequate cardiac function defined as shortening fraction of \> or = 28% by echocardiogram, or ejection fraction of \> or = 48% by radionuclide angiogram or echocardiogram * Adequate pulmonary function defined as asymptomatic or, if symptomatic, carbon monoxide diffusing capacity test (DLCO) \>45% of predicted (corrected for hemoglobin level). If unable to obtain pulmonary function test, O2 saturation \>85% in room air. Bone Marrow Failure Syndromes Patients with the following diagnoses are eligible: Severe Aplastic Anemia: * Hypocellular bone marrow biopsy (\<25% cellularity) and 2/3 of the following (at diagnosis or nadir): * Absolute Neutrophil Count (ANC) \<200/mm3, * Platelets \<20,000/mm3 * Reticulocyte count \<60,000/mm3 Fanconi Anemia: * Abnormal clastogenic studies (all patients) Severe Congenital Neutropenia (Kostmann's Syndrome) Amegakaryocytic Thrombocytope…

What they're measuring

Number of participants with Adverse Events

Timeframe: Up to 2 years

Trial details

NCT IDNCT01019876

SponsorColumbia University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-09-07