PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation

Study Overview
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Brief Summary
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This is an open-label, single-arm, multicentre study conducted in Spain to estimate the effectiveness of palifermin administered at a dose of 60 mg/kg/day IV for 3 consecutive days before the start of the conditioning regimen and for 3 consecutive days after autologous PBSCT for treating oral mucositis in patients with NHL and MM who have received high-dose conditioning chemotherapy.

Official Title
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An Open-label, Single-arm Study of Palifermin for Reduction of Mucositis in Subjects With Non-Hodgkin's Lymphoma or Multiple Myeloma Undergoing High-Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Transplantation

Conditions
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Non-Hodgkin's Lymphoma, Multiple Myeloma

Intervention / Treatment
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* Drug: Kepivance (Palifermin)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Non-Hodgkin's lymphoma (NHL) subjects scheduled to receive BEAM conditioning chemotherapy followed by autologous PBSCT, or multiple myeloma (MM) subjects scheduled to receive high-dose Melphalan (200 mg/m2) conditioning chemotherapy, in a one or two-day schedule, followed by autologous PBSCT ≥Age 18 years ECOG performance status <= 2. In the MM group, ECOG status >2 will be accepted provided that it is exclusively due to MM (e.g. pathological fracture) Adequate pulmonary function as measured by a corrected carbon monoxide (CO) diffusing capacity (DLCO) ≥ 60% of predicted Left ventricular ejection fraction (LVEF) ≥ 50% Minimum of 1.5 x 10^6 CD34+ cells/kg for autologous transplantation Adequate haematological function (ANC ≥ 1.5 x 10^9/L and platelet count ≥ 100 x 10^9/L) Serum creatinine <= 2.0 mg/dL Total bilirubin <= 2 mg/dL Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 4.0 x IULN Negative serum or urine pregnancy test for women of child bearing potential within 14 days prior to enrolment Each subject must give informed consent directly or through a legally acceptable representative before participating in any study specific procedure, or receiving any study medication. Exclusion Criteria: History of or concurrent cancer other than NHL or MM Prior treatment with palifermin, or other keratinocyte growth factors (eg, KGF-2)- Prior autologous or allogeneic transplants Oral abnormalities defined as baseline oral assessment of WHO grade >0 Other investigational procedures are excluded Subject currently is enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s), or subject is receiving other investigational agent(s) Subject of child-bearing potential is evidently pregnant (eg, positive HCG test) or is breast feeding Subject is not using adequate contraceptive precautions Known to be sero-positive for human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) Subject has known sensitivity to any of the products to be administered during dosing, including E coli-derived products Subject has previously been treated on this study or with other keratinocyte growth factors Unwilling or unable to complete the patient-reported outcome questionnaires Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with study procedures.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Kepivance (palifermin) 60 μg/kg/day IV<br>60 μg/kg/day IV for 3 consecutive days before the conditioning regimen and 3 consecutive days after the peripheral blood stem cell transplantation. | Drug: Kepivance (Palifermin)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The primary efficacy endpoints are the incidence (%) and duration of severe oral mucositis (WHO grades 3 or 4). | The study consisted of a screening period of up to 42 days to determine subject eligibility, followed by a treatment period of a maximum of 40 days. | Up to 40 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Oral mucositis, Non-Hodgkin's lymphoma (NHL), conditioning chemotherapy, autologous PBSCT, multiple myeloma (MM), Melphalan