A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCT
Completed · Phase 1
Conditions studied: Leukemia
In brief
20010133 is an open-label, dose escalation study in pediatric patients with acute leukemias receiving myelotoxic therapy (high dose etoposide, cyclophosphamide and total body irradiation \[TBI\]) followed by hematopoietic stem cell transplant (HSCT). The study will evaluate the safety and pharmacokinetics of palifermin in pediatric patients. Three doses (40 μg/kg/day, 60 μg/kg/day, and 80 μg/kg/day) are to be evaluated in each age group (1 to 2, 3 to 11, and 12 to 16 years, respectively) using a conventional dose escalation design. Palifermin is administered for 3 consecutive days (Day -10 to Day -8, respectively) before the start of the conditioning regimen and for 3 consecutive days (Day 0 to Day +2) following HSCT. Patients will be enrolled simultaneously to each age group to identify a safe, well tolerated, efficacious dose in each age group. Patients will also be followed for secondary malignancies, progression-free survival (PFS) and overall survival (OS)
Key facts
- Study ID
- NCT00460421
- Run by
- Swedish Orphan Biovitrum
- People needed
- 27
- Starts
- 2006-08-01
- Expected to finish
- 2011-05-01
- Last updated by the study team
- 2014-12-05
Who can join
Age: 1 and older, up to 16. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML) requiring HSCT
- Age ≥ 1 and ≤ 16 years at screening
- Lansky performance status > 60%
- Candidate for allogeneic HSCT protocol:
- Adequate kidney function: Serum creatinine: ≤ 1.5 mg/dL or creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 60 mL/min/1.73m2
- Adequate liver function: Serum total bilirubin: ≤ 2.0 mg/dl; aspartate transaminase (AST)/alanine aminotransferase (ALT) ≤ 4.0 x institutional upper limits of normal (IULN); Albumin ≥ 2 g/dL
- Adequate cardiac function: shortening fraction > 29% documented by echocardiogram, or ejection fraction ≥ 50% documented by multigated acquisition scan (MUGA).
- Adequate pulmonary function documented by corrected lung diffusion capacity test (DLCO) > 50% or oxygen saturation of ≥ 92% on room air if unable to perform pulmonary function tests
- Negative for human immunodeficiency virus (HIV), hepatitis C virus (HCV), human T cell lymphotropic virus (HTLV)
- Identification of an HLA-compatible donor per institutional standards
- Assent from a minor (if the child is capable of giving assent) per Department of Health and Human Services (DHHS) guidelines listed in 21CFR 50.55 and local Institutional Review Board (IRB) standards.
- Serum amylase and lipase: ≤ 1.2 x IULN
- Negative serum/urine pregnancy test for females with childbearing potential within 4 days before administration of the first palifermin dose
- Agreement by males and females of reproductive potential to use an effective means of contraception 30 days prior to enrollment through Day +30 (end of treatment)
You may not qualify if…
- Prior treatment with palifermin or other keratinocyte growth factors
- Received an investigational product or device, with the exception investigational stem cell separators, in another clinical trial within 30 days before enrollment.
- Known to have a life threatening infection not responding well to treatment
- Past history of veno-occlusive disease of the liver
- Known sensitivity to any Escherichia coli-derived products with grade 3 to 4 allergies to L-asparaginase [grade 1 to 2 allergies to L-asparaginase will be allowed].
- Receiving glutamine or any other medication to reduce the incidence of oral mucositis (OM) within 30 days before enrollment
- Previous or concurrent malignancy other than entry diagnostic criteria and/or solid organ transplantation and/or treatment of congenital immunodeficiency
- History of pancreatitis
- Breastfeeding (giving)
Where it is running
- Arizona Cancer Center — Tucson, Arizona, United States
- Loma Linda University — Loma Linda, California, United States
- Children´s Hospital — Los Angeles, California, United States
- Regents of University of California — Los Angeles, California, United States
- Children´s Hospital of Orange — Orange, California, United States
- Children´s Memorial — Chicago, Illinois, United States
- University of Texas — Dallas, Texas, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.