Pilocarpine in Preventing Mucositis and Dry Mouth in Patients Receiving Radiation Therapy for Head and Neck Cancer

This study has been completed.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Radiation Therapy Oncology Group
ClinicalTrials.gov Identifier:
First received: November 1, 1999
Last updated: November 14, 2015
Last verified: November 2015

RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs such as pilocarpine may protect normal cells from the side effects of radiation therapy. It is not yet known if pilocarpine may be effective in preventing mucositis and dry mouth in patients receiving radiation therapy for head and neck cancer.

PURPOSE: Randomized, double-blinded, phase III trial to study the effectiveness of pilocarpine in preventing mucositis and dry mouth in patients receiving radiation therapy for head and neck cancer.

Condition Intervention Phase
Head and Neck Cancer
Oral Complications
Drug: pilocarpine hydrochloride
Other: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Supportive Care
Official Title: A Phase III Study to Test the Efficacy of the Prophylactic Use of Oral Pilocarpine to Reduce Hyposalivation and Mucositis Associated With Curative Radiation Therapy in Head and Neck Cancer Patients

Resource links provided by NLM:

Further study details as provided by Radiation Therapy Oncology Group:

Primary Outcome Measures:
  • Acute salivary gland toxicity [ Time Frame: From the start of treatment to 13 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Acute mucositis in the pharynx, palate, tongue, or buccal [ Time Frame: From the start of treatment to 13 weeks ] [ Designated as safety issue: Yes ]
  • Quality of life as measured by the University of Washington Head and Neck Symptom questionnaire [ Time Frame: Pretreatment to 26 weeks from the start of treatment ] [ Designated as safety issue: No ]
  • Effects of continuing pilocarpine out to 6 months from the start of treatment [ Time Frame: From the start of treatment to 26 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 249
Study Start Date: March 1998
Study Completion Date: October 2002
Primary Completion Date: January 2001 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pilocarpine hydrocloride
5mg pilocarpine hydrochloride tablets commencing 3 days prior to irradiation
Drug: pilocarpine hydrochloride
5mg pilocarpine hydrochloride tablets
Placebo Comparator: Placebo
Placebo tablets commencing 3 days prior to irradiation
Other: Placebo

Detailed Description:

OBJECTIVES: I. Determine whether prophylactic use of pilocarpine can shelter unstimulated and stimulated whole salivary flow in patients with head and neck cancer. II. Determine whether prophylactic use of pilocarpine can moderate xerostomia in these patients. III. Determine whether prophylactic use of pilocarpine can reduce the grade and duration of radiation induced mucositis in these patients. IV. Evaluate quality of life outcomes between patients receiving pilocarpine versus placebo. V. Evaluate the impact of xerostomia on patients receiving irradiation to the head and neck.

OUTLINE: This is a randomized, double blind study. Patients receive a central axis midplane dose of radiotherapy five days per week over 6 to 7 weeks. Oral pilocarpine or placebo is administered beginning 3 days prior to radiotherapy, one tablet four times per day for three months. A tablet will be taken 45-60 minutes before radiotherapy. After three months, and after a 3-4 day rest period, all patients receive non blinded pilocarpine for an additional three month period. Patients are followed at weeks 4, 13, and 26 after the start of radiotherapy.

PROJECTED ACCRUAL: A total of 244 patients will be accrued (122 per treatment arm).


Ages Eligible for Study:   18 Years to 120 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

DISEASE CHARACTERISTICS: Histologically proven oral cavity and/or oropharyngeal squamous cell carcinoma Radiation volume to encompass at least 50% of parotid glands and have at least 50 Gy delivered to that volume via external beam No salivary gland malignancies or diseases

PATIENT CHARACTERISTICS: Age: 18 and over Performance Status: Karnofsky 60-100% Life Expectancy: Not specified Hematopoietic: Not specified Hepatic: Not specified Renal: Not specified Other: Not pregnant or nursing Effective contraceptive method must be used during study No pilocarpine allergy

PRIOR CONCURRENT THERAPY: Biologic therapy: Not specified Chemotherapy: Not specified Endocrine therapy: Not specified Radiotherapy: No prior radiotherapy to head or neck Surgery: Not specified Other: No concurrent adrenergic antagonists, cholinergic drugs, anti-cholinergics, or tricyclics

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Please refer to this study by its ClinicalTrials.gov identifier: NCT00003139

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Sponsors and Collaborators
Radiation Therapy Oncology Group
National Cancer Institute (NCI)
Study Chair: Francis G. LeVeque, DDS Barbara Ann Karmanos Cancer Institute
  More Information

Fisher JJ, Scott CB, Scarantino CW, et al.: Phase III quality of life (Qol) study: reduction in hyposalivation does not improve Qol for head and neck (H&N) cancer patients post radiation therapy (P-RT). RTOG 9709. [Abstract] Proceedings of the American Society of Clinical Oncology 21: A-1423, 2002.
Scarantino CW, Leveque FG, Scott CB, et al.: A phase III study of concomitant oral pilocarpine to reduce hypo-salivation and mucositis associated with curative radiation therapy (RT) in the head and neck (H&N) cancer patients. RTOG 9709. [Abstract] Proceedings of the American Society of Clinical Oncology 20: A-897, 225a, 2001.
Scarantino CW, Leveque F, Scott C, et al.: A phase III study on the concurrent use of oral pilocarpine to reduce hyposalivation and mucositis associated with radiation therapy in head and neck cancer patients: final results of RTOG 97-09. [Abstract] Int J Radiat Oncol Biol Phys 51(3 suppl 1): A-157, 85-86, 2001.

Responsible Party: Radiation Therapy Oncology Group
ClinicalTrials.gov Identifier: NCT00003139     History of Changes
Other Study ID Numbers: RTOG-9709  CDR0000065912  NCI-P97-0121 
Study First Received: November 1, 1999
Last Updated: November 14, 2015
Health Authority: United States: Federal Government

Keywords provided by Radiation Therapy Oncology Group:
oral complications
lip and oral cavity squamous cell carcinoma
oropharyngeal squamous cell carcinoma

Additional relevant MeSH terms:
Head and Neck Neoplasms
Neoplasms by Site
Autonomic Agents
Cholinergic Agents
Cholinergic Agonists
Molecular Mechanisms of Pharmacological Action
Muscarinic Agonists
Neurotransmitter Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 23, 2016