PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation

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

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.

Study Overview

• Status: Completed
• Conditions: Multiple Myeloma; Non-Hodgkin's Lymphoma
• Intervention / Treatment: Drug: Kepivance (Palifermin)

• Study Type: Interventional
• Enrollment (Actual): 145
• Phase: Phase 4

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Kepivance (palifermin) 60 μg/kg/day IV; 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)

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Investigators: Study Director: MD, Biovitrum AB

Study record dates

Study Major Dates

• Study Start: April 1, 2006
• Primary Completion (Actual): April 1, 2007
• Study Completion (Actual): May 1, 2007

Study Registration Dates

• First Submitted: July 13, 2006
• First Submitted That Met QC Criteria: July 14, 2006
• First Posted (Estimate): July 17, 2006

Study Record Updates

• Last Update Posted (Estimate): November 2, 2014
• Last Update Submitted That Met QC Criteria: October 31, 2014
• Last Verified: October 1, 2014

More Information

Terms related to this study

• Keywords: Non-Hodgkin's lymphoma (NHL); Oral mucositis; Melphalan; multiple myeloma (MM); conditioning chemotherapy; autologous PBSCT
• Additional Relevant MeSH Terms: Digestive System Diseases; Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Hematologic Diseases; Gastrointestinal Diseases; Hemorrhagic Disorders; Gastroenteritis; Stomatognathic Diseases; Mouth Diseases; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Lymphoma; Multiple Myeloma; Neoplasms, Plasma Cell; Lymphoma, Non-Hodgkin; Mucositis
• Other Study ID Numbers: 20050100