Clinical trial NCT04151082: High Dose Steroid Therapy (Prednisone or Methylprednisolone) for the Improvement of Symptoms of Late Radiation-Associated Lower Cranial Neuropathy in Oropharyngeal Cancer Survivors
Official title: Stop LCNP: High Dose Steroid Therapy for Late Radiation-Associated Lower Cranial Neuropathy: A Phase I/II Dose Finding Trial and Data Registry
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Cranial Nerve Disorder
Head and Neck Carcinoma
Oropharyngeal Carcinoma
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2019-11-05
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: This phase I/II trial studies the side effect and best dose of steroid therapy (prednisone or methylprednisolone) in improving symptoms of late radiation-associated lower cranial neuropathy in oropharyngeal cancer survivors. Steroid therapy with prednisone or methylprednisolone may help to improve symptoms associated with late radiation-associated lower cranial neuropathy.

Detailed description: PRIMARY OBJECTIVE:

I. To establish feasibility and maximum tolerated dose (MTD) of steroid therapy that provides symptomatic improvement in oropharyngeal cancer (OPC) survivors with late lower cranial neuropathy (LCNP).

SECONDARY OBJECTIVE:

I. To examine endpoints that may be sensitive to functional or symptomatic gains with steroid therapy for late LCNP in OPC survivors by characterizing changes in symptoms, functional status, quality of life (QOL), neurophysiology, and imaging studies after steroid therapy.

TERTIARY OBJECTIVE:

I. To establish a hypothesis-generating database of physiologic, functional, and patient-reported outcomes (PROs) among head and neck cancer (HNC) survivors with late lower cranial neuropathy treated with steroid therapy.

OUTLINE: This is a phase I, dose-escalation study of steroid therapy followed by a phase II study.

Patients receive prednisone orally (PO) (or by feeding tube) once daily (QD) on days 1-5 and then taper off over 2 weeks or methylprednisolone intravenously (IV) over 1 hour on days 1-5 in the absence of disease progression or unacceptable toxicity.

Eligibility criteria: Inclusion Criteria:

* INCLUSION CRITERIA FOR CLINICAL TRIAL: Disease free adult survivors of oropharyngeal cancer
* INCLUSION CRITERIA FOR CLINICAL TRIAL: Treated with radiotherapy \>= 2 years post treatment (disease status per surveillance imaging and clinical surveillance)
* INCLUSION CRITERIA FOR CLINICAL TRIAL: Late radiation-associated lower cranial neuropathy of XII with or without X nerve (LCNP cases will be considered therapy-related when imaging, physical examination, and/or biopsy fail to demonstrate structural or malignant source)
* INCLUSION CRITERIA FOR CLINICAL TRIAL: Willing and able to return for assessment post-steroid therapy
* INCLUSION CRITERIA FOR CLINICAL TRIAL: Able to complete symptom survey (MD Anderson Symptom Inventory - Head and Neck \[MDASI-HN\]) in validated languages: English, simplified Chinese, Filipino, Greek, Japanese, Korean, Russian, Spanish, Taiwanese
* INCLUSION CRITERIA FOR REGISTRY: Disease-free adult survivors of head and neck cancer
* INCLUSION CRITERIA FOR REGISTRY: \>= 2 years post treatment (disease status per surveillance imaging and clinical surveillance)
* INCLUSION CRITERIA FOR REGISTRY: Late lower cranial neuropathy of XII with or without X nerve (LCNP cases will be considered therapy-related when imaging, physical examination, and/or biopsy fail to demonstrate structural or malignant source)
* INCLUSION CRITERIA FOR REGISTRY: Able to complete symptom survey (MDASI-HN) in validated languages: English, simplified Chinese, Filipino, Greek, Japanese, Korean, Russian, Spanish, Taiwanese

Exclusion Criteria:

* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Uncontrolled diabetes
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Uncontrolled hypertension (systolic \> 160; diastolic \> 90)
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Known gastrointestinal ulcer
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: History of psychosis
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Pregnant women
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Untreated or treatment refractory obstructive pharyngoesophageal stricture
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: Known history or diagnosis of bipolar disorder
* EXCLUSION CRITERIA FOR CLINICAL TRIAL: History of surgery near hypoglossal nerve path
* EXCLUSION CRITERIA FOR REGISTRY: Uncontrolled diabetes
* EXCLUSION CRITERIA FOR REGISTRY: Uncontrolled hypertension (systolic \> 160; diastolic \> 90)
* EXCLUSION CRITERIA FOR REGISTRY: Known gastrointestinal ulcer
* EXCLUSION CRITERIA FOR REGISTRY: History of psychosis
* EXCLUSION CRITERIA FOR REGISTRY: Pregnant women
* EXCLUSION CRITERIA FOR REGISTRY: Untreated or treatment refractory obstructive pharyngoesophageal stricture
* EXCLUSION CRITERIA FOR REGISTRY: Known history or diagnosis of bipolar disorder

Primary outcomes:
measure: Maximum tolerated dose
timeFrame: Up to 3 weeks
measure: Change in MD Anderson Symptom Inventory - Head and Neck (MDASI-HN) top 5 mean
timeFrame: Baseline up to 3 weeks
Secondary outcomes:
measure: Improvement in tongue innervation on electromyography (EMG) findings
timeFrame: Up to 3 years
measure: Improvement in dynamic imaging grade of swallowing toxicity (DIGEST) scores
timeFrame: Up to 3 years
measure: Changes in maximum isometric lingual strength (MILS) and lingual range of motion (LROM)
timeFrame: Baseline up to 3 years
measure: Changes in patient reported outcomes (PRO) on MD Anderson Dysphagia Inventory (MDADI), Performance Status Scale for Head and Neck (PSS-HN), or EuroQOL (EQ-5D)
timeFrame: Baseline up to 3 years
Other outcomes:
Locations:
city: Houston
country: United States
facility: M D Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Katherine A Hutcheson, BA,MS,PHD
role: PRINCIPAL_INVESTIGATOR
References: