CancerPhase 3NCT00109031
Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)
A Phase 3 study testing palifermin, Total Body Irradiation and Cyclophosphamide in people with Cancer and Lymphoma, run by Swedish Orphan Biovitrum.
Trial summary
To evaluate whether palifermin (rHuKGF) administered as a single dose is non-inferior to 3 consecutive doses of palifermin in reducing the incidence of severe oral mucositis (World Health Organization [WHO] grade 3 and 4). Read more about this study
- Who is it for?
- Ages 18 years and older
- What is being tested?
- palifermin, Total Body Irradiation and Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 47 participants
- Where
- No sites listed yet
- Study dates
- Jan 2005to Dec 2010
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent
- Subjects with: non-Hodgkin's lymphoma, Hodgkin's disease, acute myelogenous leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, chronic lymphocytic leukemia, or multiple myeloma
- Minimum of 1.5 x 10^6 CD34+ cells/kg cryopreserved and to be transplanted.
Who cannot join
- Cancer other than those specified in inclusion criteria above (except: adequately treated basal cell carcinoma of the skin)
- Prior bone marrow or peripheral blood stem cell transplantation - Negatively selected (purged) stem cell product - Current active infection or oral mucositis
- Congestive heart failure as defined by New York Heart Association class III or IV.
- History of or current diagnosis of pancreatitis
- Inadequate renal function (serum creatinine greater than 1.5x the upper limit of normal per the institutional guidelines)
- Inadequate liver function (direct bilirubin greater than 1.5x the upper limit of normal, aspartate aminotransferase (AST) greater than 3x upper limit of normal and/or alanine aminotransferase (ALT) greater than 3x upper limit of normal per the institutional guidelines)
- Inadequate pulmonary function as measured by a corrected diffusion capacity of carbon monoxide (DLCO) less than 50% of predicted.
- Subject is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug trial(s), or subject is receiving other investigational agent(s)
About this study
Official title: A Randomized, Blinded, Active-control Trial of Palifermin (rHuKGF) to Evaluate Oral Mucositis in Subjects With Hematologic Malignancies Undergoing Fractionated Total Body Irradiation (fTBI) and High Dose Chemotherapy With Autologous Peripheral Blood Progenitor Cell (PBPC) Transplantation
To evaluate whether palifermin (rHuKGF) administered as a single dose is non-inferior to 3 consecutive doses of palifermin in reducing the incidence of severe oral mucositis (World Health Organization [WHO] grade 3 and 4).
- Being tested
- palifermin, Total Body Irradiation, Cyclophosphamide, Etoposide, Placebo
- Keywords
- Cancer, Oncology
- Medical subject headings
- Neoplasms, Lymphoma, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
paliferminDrug
Also called Kepivance, Recombinant Human Keratinocyte Growth Factor (rHuKGF)
Administered as one daily intravenous bolus.
Total Body IrradiationRadiation
To be delivered before the administration of chemotherapy in 6, 8, or 10 fractions over 3 or 4 days.
CyclophosphamideDrug
Cyclophosphamide is administered at a total dose of 100 mg/kg given in 1 dose on Day -2
EtoposideDrug
Also called VP-16
Etoposide may be administered (optional) as a single intravenous infusion over 4 hours on the day after the last fTBI fraction.
PlaceboDrug
Administered as one daily intravenous bolus.
| Study group | Role | What they receive |
|---|---|---|
| Palifermin 60 µg/kg for 3 days | Active comparator | Palifermin 60 µg/kg plus placebo to match the total volume equivalent to a 180 µg/kg dose on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC). Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. Receives: Drug: palifermin; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Etoposide; Drug: Placebo |
| Palifermin 180 μg/kg on Day -1 | Experimental | Palifermin 180 μg/kg on Day -1 and matched placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. Receives: Drug: palifermin; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Etoposide; Drug: Placebo |
| Palifermin 180 μg/kg on Day -2 | Experimental | Palifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. Receives: Drug: palifermin; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Etoposide; Drug: Placebo |
| Palifermin 180 μg/kg on Day -3 | Experimental | Palifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. Receives: Drug: palifermin; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Etoposide; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)
Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until severe OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.
Measured: Up to Day 28
Secondary outcomes (6)
Duration of Severe Oral Mucositis (WHO Grade 3 and 4)
The duration of severe oral mucositis (OM) was calculated as the number of days from the onset of severe OM (first time a WHO grade 3 or 4 was observed) to the day when severe OM was resolved (first time WHO grade 2 or less was observed after last WHO grade 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 3 or 4 during the study.
Measured: Up to Day 28
Area Under the Curve (AUC) of Mouth and Throat Soreness Score
The Oral Mucositis Daily Questionnaire (OMDQ) is a self-reported tool that evaluates overall health, mouth and throat soreness (MTS) and activity limitations due to MTS. The OMDQ was completed once daily beginning with the first day of study drug administration through Day 28. The area under the curve of mouth and throat soreness score was assessed from the question "How much mouth and throat soreness did you experience in the past 24 hours?" Participants answered on a scale from 0 (no soreness) to 4 (extreme soreness). A higher value in MTS AUC indicates worse self-assessed MTS.
Measured: From the first day of study drug administration through Day 28
Number of Participants With Parenteral or Transdermal Opioid Analgesic Use
Includes nonprophylactic intravenous opioid analgesics (fentanyl, morphine, morphine sulphate, hydromorphone, meperidine) and transdermal opioid analgesics (fentanyl patch) for the indication of oral mucositis and dysphagia.
Measured: Up to Day 28
Number of Participants With WHO Grades 2, 3 or 4 Oral Mucositis
Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.
Measured: Up to Day 28
Duration of WHO Grade 2, 3 or 4 Oral Mucositis
The duration of grade 2, 3 or 4 oral mucositis (OM) was calculated as the number of days from the onset of grade 2, 3 or 4 OM (first time a WHO grade 2, 3 or 4 was observed) to the day when WHO grade 2 - 4 OM was resolved (first time WHO grade less than 2 was observed after last WHO grade 2, 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 2, 3 or 4 during the study.
OM was evaluated using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.
Measured: Up to Day 28
Number of Participants With WHO Grade 4 Oral Mucositis
Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.
Measured: Up to Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2005 (estimated)
- Study startJan 2005
- Primary completionFeb 2006
- Study completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for CancerStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00109031
- Sponsor's protocol ID
- 20040212
- Status last verified
- Sep 2014
- First submitted
- 22 Apr 2005
- Last updated
- 15 Sep 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Blinded, Active-control Trial of Palifermin (rHuKGF) to Evaluate Oral Mucositis in Subjects With Hematologic Malignancies Undergoing Fractionated Total Body Irradiation (fTBI) and High Dose Chemotherapy With Autologous Peripheral Blood Progenitor Cell (PBPC) Transplantation
- ClinicalTrials.gov ID
- NCT00109031
- Acronym / sponsor ID
- 20040212
- Keywords
- Cancer, Oncology
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- Amgen
- Study start
- Jan 2005
- Primary completion
- Feb 2006 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 25 Apr 2005 (estimated)
- Results
- Posted 15 Sep 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate whether palifermin (rHuKGF) administered as a single dose is non-inferior to 3 consecutive doses of palifermin in reducing the incidence of severe oral mucositis (World Health Organization [WHO] grade 3 and 4).
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Palifermin 60 µg/kg for 3 days | Active comparator | Drug: palifermin, Radiation: Total Body Irradiation, Drug: Cyclophosphamide, Drug: Etoposide, Drug: PlaceboPalifermin 60 µg/kg plus placebo to match the total volume equivalent to a 180 µg/kg dose on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC). Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. |
| Palifermin 180 μg/kg on Day -1 | Experimental | Drug: palifermin, Radiation: Total Body Irradiation, Drug: Cyclophosphamide, Drug: Etoposide, Drug: PlaceboPalifermin 180 μg/kg on Day -1 and matched placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. |
| Palifermin 180 μg/kg on Day -2 | Experimental | Drug: palifermin, Radiation: Total Body Irradiation, Drug: Cyclophosphamide, Drug: Etoposide, Drug: PlaceboPalifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. |
| Palifermin 180 μg/kg on Day -3 | Experimental | Drug: palifermin, Radiation: Total Body Irradiation, Drug: Cyclophosphamide, Drug: Etoposide, Drug: PlaceboPalifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Written informed consent
- Subjects with: non-Hodgkin's lymphoma, Hodgkin's disease, acute myelogenous leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, chronic lymphocytic leukemia, or multiple myeloma
- Minimum of 1.5 x 10^6 CD34+ cells/kg cryopreserved and to be transplanted.
Key exclusion criteria (8 listed)
- Cancer other than those specified in inclusion criteria above (except: adequately treated basal cell carcinoma of the skin)
- Prior bone marrow or peripheral blood stem cell transplantation - Negatively selected (purged) stem cell product - Current active infection or oral mucositis
- Congestive heart failure as defined by New York Heart Association class III or IV.
- History of or current diagnosis of pancreatitis
- Inadequate renal function (serum creatinine greater than 1.5x the upper limit of normal per the institutional guidelines)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| paliferminAlso: Kepivance, Recombinant Human Keratinocyte Growth Factor (rHuKGF)Administered as one daily intravenous bolus. | Drug | Palifermin 180 μg/kg on Day -1, Palifermin 180 μg/kg on Day -2, Palifermin 180 μg/kg on Day -3, Palifermin 60 µg/kg for 3 days |
| Total Body IrradiationTo be delivered before the administration of chemotherapy in 6, 8, or 10 fractions over 3 or 4 days. | Radiation | Palifermin 180 μg/kg on Day -1, Palifermin 180 μg/kg on Day -2, Palifermin 180 μg/kg on Day -3, Palifermin 60 µg/kg for 3 days |
| CyclophosphamideCyclophosphamide is administered at a total dose of 100 mg/kg given in 1 dose on Day -2 | Drug | Palifermin 180 μg/kg on Day -1, Palifermin 180 μg/kg on Day -2, Palifermin 180 μg/kg on Day -3, Palifermin 60 µg/kg for 3 days |
| EtoposideAlso: VP-16Etoposide may be administered (optional) as a single intravenous infusion over 4 hours on the day after the last fTBI fraction. | Drug | Palifermin 180 μg/kg on Day -1, Palifermin 180 μg/kg on Day -2, Palifermin 180 μg/kg on Day -3, Palifermin 60 µg/kg for 3 days |
| PlaceboAdministered as one daily intravenous bolus. | Drug | Palifermin 180 μg/kg on Day -1, Palifermin 180 μg/kg on Day -2, Palifermin 180 μg/kg on Day -3, Palifermin 60 µg/kg for 3 days |
Primary outcome
Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)
Time frame: Up to Day 28
Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until severe OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.
6 secondary outcomes
Duration of Severe Oral Mucositis (WHO Grade 3 and 4)
Time frame: Up to Day 28
Area Under the Curve (AUC) of Mouth and Throat Soreness Score
Time frame: From the first day of study drug administration through Day 28
Number of Participants With Parenteral or Transdermal Opioid Analgesic Use
Time frame: Up to Day 28
Number of Participants With WHO Grades 2, 3 or 4 Oral Mucositis
Time frame: Up to Day 28
Duration of WHO Grade 2, 3 or 4 Oral Mucositis
Time frame: Up to Day 28
Number of Participants With WHO Grade 4 Oral Mucositis
Time frame: Up to Day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Cancer
Not yet recruitingPhase 1
Hepatic Artery Infusion of Carfilzomib in Participants With Liver Metastatic Disease Previously Treated With Hepatic Artery Infusion Pump Therapy
Colorectal Neoplasms, Neoplasms
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Enrolling by invitationPhase 2
A Single-Arm Study of Nimotuzumab Combined With NALIRIFOX as First-Line Treatment for Pancreatic Cancer
Pancreatic Cancer Metastatic
Recruiting
Methylation Profiling in Self-Samples for Identification of (Epi)Genetic Biomarkers for Gynecological Cancers
Gynaecological Cancers, Ovarian Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing palifermin, Total Body Irradiation and Cyclophosphamide in people with Cancer and Lymphoma, run by Swedish Orphan Biovitrum.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Sep 2014. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Sep 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4) (time frame: Up to Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2014.