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MucositisPhase 1/2NCT00626639

A Study of Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy and Concurrent Chemotherapy

A Phase 1/2 study testing palifermin, Radiotherapy and Cisplatin in people with Mucositis and Head and Neck Cancer, run by Swedish Orphan Biovitrum.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Oral Mucositis associated with adjuvant radiation and concurrent chemotherapy in postoperative Head and Neck setting Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Mucositis, Head and Neck Cancer
What is being tested?
palifermin, Radiotherapy and Cisplatin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
5 participants
Where
No sites listed yet
Study dates
Jul 2005to Jul 2015
Phase
Phase 1/2
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

  • History of newly diagnosed histologically confirmed squamous cell carcinoma (American Joint Committee on Cancer [AJCC] Stage II, III, IVA, or IVB) involving either the oral cavity, oropharynx, nasopharynx, hypopharynx, or larynx, post surgical resection (R0, R1)
  • Scheduled to receive adjuvant concurrent chemoradiation treatment within 12 weeks of surgery
  • High-risk subject defined by presence of at least one of the following: R1 resection margins; T3 or T4 tumor stage; 3 or more positive lymph node metastases; <3 lymph node metastases with extracapsular extension of the disease
  • Radiation treatment field to receive planned dose of at least 50Gy to areas of the oral cavity/oropharynx mucosa that can be visualized

Who cannot join

  • Tumors of the lips, paranasal sinuses, salivary glands, or of unknown primary tumors
  • Metastatic disease (M1) / Stage IV C
  • Presence or history of any other primary malignancy
  • History of pancreatitis
  • Prior radiotherapy to the site of disease
  • Prior chemotherapy
  • Other investigational procedures
  • Thirty days or less since receiving an investigational product or device in another clinical trial. Current enrollment in another clinical trial is not permitted unless the sole purpose of the trial is for long-term follow-up/survival data

About this study

Official title: A Phase 1/2 Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Weekly Doses of Palifermin (rHuKGF) for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Cancer (HNC) Receiving Postoperative Radiotherapy With Concurrent Chemotherapy

Oral Mucositis associated with adjuvant radiation and concurrent chemotherapy in postoperative Head and Neck setting

Read the detailed description

This study consisted of 2 phases. The acute oral mucositis (OM) evaluation phase includes the time from randomization to the time of severe OM (WHO Grade 3 or 4) resolution (up to Week 12 or up to Week 15 for participants whose severe OM is not resolved at Week 12). In the acute OM evaluation phase, participants were randomized to receive either a single IV bolus dose of palifermin or placebo at 120 μg/kg, 3 days before the start of radiotherapy, plus 7 once-weekly palifermin or placebo doses at the same dose level during a 7-week radio/chemotherapy course. In the long-term follow up phase, participants are followed until death, withdrawal of consent, or loss to follow-up. The long-term follow up phase is still ongoing.

Being tested
Placebo, palifermin, Radiotherapy, Cisplatin
Keywords
palifermin, Clinical Trial, Oncology, Head & Neck
Medical subject headings
Mucositis, Head and Neck Neoplasms, Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PlaceboDrug

    Administered by intravenous (IV) bolus injection

  • paliferminDrug

    Administered by intravenous (IV) bolus injection

  • RadiotherapyRadiation

    Once daily irradiation of 20 centigray (cGy)/day x 33 fractions for a total target dose of 6600 cGy (conventional radiation therapy using standard fractionation [one fraction per day])

  • CisplatinDrug

    100 mg/m^2 intravenously (IV) on days 1, 22 and 43.

Study groups in NCT00626639
Study groupRoleWhat they receive
PlaceboPlacebo comparatorThree days before the start of radiotherapy (Day -3), participants received a single intravenous (IV) bolus injection of matching placebo. During radiotherapy (beginning on Day 1), participants received a weekly single IV bolus injection of matching placebo after the last radiation fraction of that week (usually on Fridays) until grade ≥3 oral mucositis occurred, or for a maximum 8 doses (completion of radiotherapy). Participants also received cisplatin 100 mg/m^2 on days 1, 22 and 43.
Receives: Drug: Placebo; Radiation: Radiotherapy; Drug: Cisplatin
PaliferminExperimentalThree days before the start of radiotherapy (Day -3), participants received a single intravenous (IV) bolus injection of palifermin at 120 μg/kg. During radiotherapy (beginning on Day 1), participants received a weekly single IV bolus injection of palifermin at 120 μg/kg after the last radiation fraction of that week (usually on Fridays) until grade ≥3 oral mucositis occurred, or for a maximum 8 doses (completion of radiotherapy). Participants also received cisplatin 100 mg/m^2 on days 1, 22 and 43.
Receives: Drug: palifermin; Radiation: Radiotherapy; Drug: Cisplatin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Adverse Events (AEs)

    An adverse event is an undesirable medical occurrence (sign, symptom, or diagnosis) or worsening of a pre-existing medical condition occurring after start of study drug up to the end of acute oral mucositis (OM) evaluation phase, whether or not considered to be study drug related. If severe OM was not resolved by Week 12, AEs were documented until resolution of severe OM or Week 15, whichever occurred first. A serious AE is any event that is fatal, life threatening, requires or prolongs hospitalization, is a persistent or significant disability/incapacity or is a congenital anomaly/birth defect. The intensity of AEs was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v3 based on the following: Grade 1 = Mild AE, Grade 2 = Moderate AE, Grade 3 = Severe AE, Grade 4 = Life-threatening or disabling AE, Grade 5 = Death related to AE. A Protocol-specific Limiting Toxicity (PSLT) is any non-hematologic Grade 3 or 4 AE considered related to study drug.

    Measured: Up to Week 12 (or Week 15 for participants with severe OM was not resolved by Week 12)

  2. Ratio of Ki67-positive Cells Before and After Palifermin Treatment

    The effect of palifermin on cell proliferation was to be assayed by staining for the cell cycle proliferation marker Ki67 in buccal mucosal biopsy samples taken prior to the first dose and either 24 or 48 hours after the first dose. Due to the small sample size, this analysis was not performed.

    Measured: Day -3 predose and 24 or 48 hours post-dose

  3. Pharmacokinetics of Palifermin

    Due to the small sample size this analysis was not performed.

    Measured: Day -3, predose and at 2, 5, 15, 30, 60, and 90 minutes and 2, 4, 6, 8, 10, 12, 24 and 48 hours after the first dose

Secondary outcomes (4)

  1. Number of Participants With Severe Oral Mucositis (OM) (Adapted RTOG/EORTC Grade ≥3)

    The adapted RTOG/EORTC mucositis assessment scale as follows: Grade 0 = no change; Grade 1 = mild enanthema, mild pain; Grade 2 = patchy mucositis, moderate edema, moderate pain; Grade 3 = confluent fibrinous mucositis, massive edema, massive pain; Grade 4 = extensive ulceration, confluent necrosis, massive hemorrhage.

    Due to the small sample size this analysis was not performed.

    Measured: Assessed daily up to Week 12 (or Week 15 if severe oral mucositis not resolved ≤ adapted RTOG/EORTC Grade 2 by Week 12).

  2. Patient-Reported Mouth and Throat Soreness Score

    The average patient-reported mouth and throat soreness (MTS) score as reported on question 3 of the Oral Mucositis Questionnaire for Head and Neck Cancer [OMQ-HN]): "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).

    Due to the small sample size this analysis was not performed.

    Measured: Assessed daily up to Week 12 (or Week 15 if severe oral mucositis not resolved ≤ adapted RTOG/EORTC Grade 2 by Week 12).

  3. Number of Participants With Disease Progression by Week 12

    Disease progression was determined by clinical examination and histopathologic examination by the Investigator.

    Measured: Up to Week 12

  4. Overall Survival

    Deaths during long-term follow up of subject participating in the acute phase of the study receiving placebo or Palifermin.

    Measured: During long-term follow-up phase, until December 2015

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Feb 2008 (estimated)
  2. Study startJul 2005
  3. Primary completionMay 2007
  4. Study completionJul 2015

Resources

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 1/2
Primary purpose
Supportive care
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
5 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00626639
Sponsor's protocol ID
20040124
Status last verified
May 2017
First submitted
21 Feb 2008
Last updated
21 Jun 2017
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing palifermin, Radiotherapy and Cisplatin in people with Mucositis and Head and Neck Cancer, 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 4 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 21 Jun 2017. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 6 Mar 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 Adverse Events (AEs) (time frame: Up to Week 12 (or Week 15 for participants with severe OM was not resolved by Week 12)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00626639.

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 21 Jun 2017.

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