A Phase II Controlled Trial of Velafermin for Prevention of Oral Mucositis

Study Overview
=================
Brief Summary
-----------------
CG53135-05 (velafermin), a recombinant human fibroblast growth factor-20 (rhFGF-20) protein, is under investigation for the prevention of oral mucositis. Oral mucositis is a commonly occurring side effect of high-dose chemotherapy in patients undergoing autologous hematopoietic stem cell transplant. The objective of this Phase II trial is to confirm the safety and efficacy of CG53135-05 (velafermin) when administered as a single dose to patients at risk for developing oral mucositis.

Official Title
-----------------
A Phase II Randomized Double Blind Placebo Controlled Trial to Assess Safety and Efficacy of Velafermin for Prevention of Oral Mucositis in Hematologic Cancer Patients Receiving Autologous Stem Cell Transplant

Conditions
-----------------
Oral Mucositis, Stomatitis

Intervention / Treatment
-----------------
* Drug: velafermin
* Drug: placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Age 18 years or older Patients with multiple myeloma or lymphoma receiving myeloablative CT with or without TBI that require autologous stem cell support. The CT regimens are limited to high dose melphalan (200 mg/m2) as a single agent, BEAM, or TBI with cyclophosphamide or VP-16. Adequate organ function that meets institutional requirements for autologous stem cell transplant. A minimum CD34+ cell dose of 2X106 /kg based on ideal body weight (IBW) has been or will be infused. ECOG Performance Score of 2 or less Signed Informed Consent Form (ICF) Exclusion Criteria: Premenopausal female patients who are pregnant, lactating or are likely to become pregnant Patients diagnosed with active sero-positive acquired immunodeficiency syndrome (AIDS) or Hepatitis B/ C Patients with known hypersensitivity to recombinant protein therapeutics Patients who have taken velafermin (CG53135-05) previously Patients who have taken palifermin in the past 90 days Patients who have taken other investigational drugs in the past 30 days Patients who have untreated symptomatic dental infection Patients with a history of sensitivity or allergy to E. coli-derived products Patients with WHO Grade 3 or 4 OM at the time of randomization Patients who are sensitive or allergic to G-CSF, Fluconazole or acyclovir or equivalent Patients with altered mental status precluding understanding of the informed consent process and/or completion of the necessary assessments Patients with baseline creatinine level greater than or equal to 3 or any patient with renal insufficiency requiring dialysis.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: 1<br>placebo | Drug: placebo<br>* administered intravenously as 100mL single dose over 15 minutes on day 1<br>|
| Experimental: 2<br>30 mcg/kg velafermin | Drug: velafermin<br>* administered intravenously as 100mL single dose over 15 minutes on day 1<br>* Other names: CG53135-05;|
| Experimental: 3<br>10 mcg/kg velafermin | Drug: velafermin<br>* administered intravenously as 100mL single dose over 15 minutes on day 1<br>* Other names: CG53135-05;|
| Experimental: 4<br>60 mcg/kg velafermin | Drug: velafermin<br>* administered intravenously as 100mL single dose over 15 minutes on day 1<br>* Other names: CG53135-05;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| the incidence of grade 3/4 oral mucositis using WHO grading system |  | evaluated throughout the study | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
oral mucositis, hematologic, autologous stem cell transplant, velafermin, stomatitis, oncology - supportive care, mouth diseases