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Phase I Dose Escalation Trial of High Dose Melphalan Conditioning Regimen With Palifermin for Cytoprotection Followed by Autologous Peripheral Blood Stem Cell Transplantation for Multiple Myeloma

Phase 1
18 Years
Open (Enrolling)
Mucositis, Multiple Myeloma

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Trial Information

Phase I Dose Escalation Trial of High Dose Melphalan Conditioning Regimen With Palifermin for Cytoprotection Followed by Autologous Peripheral Blood Stem Cell Transplantation for Multiple Myeloma



- Determine the maximum tolerated dose of high-dose melphalan when administered with
palifermin in patients undergoing autologous peripheral blood stem cell transplantation
for stage II or III multiple myeloma.


- Assess overall response (complete and partial response and stable disease) in these
patients at 28 and 100 days post-transplantation.

- Assess the efficacy of palifermin as a cytoprotective agent in reducing incidence and
duration of mucositis in patients treated with this regimen.

- Assess patient-reported outcomes and impact of palifermin on quality of life of these

- Assess the qualitative and quantitative toxicities of this regimen in these patients.

OUTLINE: This is a dose-escalation study of melphalan. Patients are stratified according to
creatinine clearance (normal vs < 60 mL/min).

Patients receive high-dose melphalan IV on day -2 and palifermin IV on days -5 to -3 and
1-3. Patients undergo autologous peripheral blood stem cell transplantation on day 0.

In each stratum, cohorts of 3-6 patients receive escalating doses of melphalan until the
maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at
which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients complete questionnaires about overall health, mouth and throat soreness (MTS), and
activity limitations due to MTS once daily on days -5 to 28.

After completion of study treatment, patients are followed at days 28 and 100 and then
periodically thereafter.

Inclusion Criteria


- Diagnosis of multiple myeloma

- Stage II or III disease

- Must have undergone successful stem cell mobilization (≥ 2.0 x 10^6 CD34+ cells/kg)

- No oral lesions from any other etiology

- No unhealed mucositis from induction treatment


- ECOG performance status (PS) 0-2 OR Karnofsky PS 60-100%

- Amylase and lipase normal

- Bilirubin ≤ 1.5 times upper limit of normal (ULN)

- AST and ALT ≤ 3 times ULN

- Creatinine normal (stratum 1 only)

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No HIV positivity

- No history of allergic reaction attributed to melphalan

- No uncontrolled illness, including, but not limited to, any of the following:

- Ongoing or active infection

- Symptomatic congestive heart failure

- Unstable angina pectoris

- Cardiac arrhythmia

- No psychiatric illness or social situation that would preclude study compliance

- No hepatitis B or C positivity

- No prior or concurrent pancreatitis

- No known sensitivity to any of the study drugs, including E. coli-derived products


- Prior bone marrow or stem cell transplantation allowed

- No prior palifermin

- More than 30 days since prior investigational agents

- No concurrent dialysis

- No concurrent amifostine

- No concurrent prophylactic oral cryotherapy during melphalan administration

- No concurrent mouthwash solutions containing any of the following:

- Chlorhexidine

- Hydrogen peroxide

- Diphenhydramine hydrochloride

- No concurrent recombinant interleukin-11 or sargramostim (GM-CSF)

- No concurrent sucralfate in suspension form

- Sucralfate tablets allowed

- No concurrent povidone-iodine rinses

- No concurrent glutamine as a prophylactic agent for mucositis

- No other concurrent investigational agents

- No concurrent antithymocyte globulin suppression or alemtuzumab

- No concurrent rituximab

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Maximum tolerated dose of melphalan when treated with palifermin to prevent mucositis

Outcome Time Frame:

Days -5, -4, -3, 2, +1, +2 and +3

Safety Issue:


Principal Investigator

Muneer H. Abidi, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Barbara Ann Karmanos Cancer Institute


United States: Federal Government

Study ID:




Start Date:

June 2007

Completion Date:

October 2012

Related Keywords:

  • Mucositis
  • Multiple Myeloma
  • mucositis
  • drug/agent toxicity by tissue/organ
  • stage II multiple myeloma
  • stage III multiple myeloma
  • refractory multiple myeloma
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Mucositis



Barbara Ann Karmanos Cancer Institute Detroit, Michigan  48201