Trial Outcomes & Findings for MSC in Patients With Xerostomia Post XRT in Head and Neck Cancer (NCT NCT04489732)

Results Overview

Dose limiting toxicity is defined as: submandibular pain \> 5 on a pain scale of 0-10 at 1-month after MSC injection OR any serious AE OR any of the selected toxicities listed per protocol within one-month post-injection.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE1

Target enrollment

6 participants

Primary outcome timeframe

up to 1 month post injection (up to 3 months from consent)

Results posted on

2025-04-18

Participant Flow

Participants were enrolled at the UW Hospital and Clinics from February 2022 to September 2022.

Participant milestones

Participant milestones
Measure	Treatment With MSCs A single dose of MSCs injected into the submandibular glands of patients with radiation-induced xerostomia Autologous bone-marrow derived, interferon gamma stimulated mesenchymal stromal cells: Single dose, starting at * Dose Level 0: 10 x 10\^6 injected into one submandibular gland on Day 1
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

MSC in Patients With Xerostomia Post XRT in Head and Neck Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment With MSCs n=6 Participants A single dose of MSCs injected into the submandibular glands of patients with radiation-induced xerostomia Autologous bone-marrow derived, interferon gamma stimulated mesenchymal stromal cells: Single dose, starting at * Dose Level 0: 10 x 10\^6 injected into one submandibular gland on Day 1
Age, Customized 60-69 years	2 Participants n=5 Participants
Age, Customized 70-79 years	4 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: up to 1 month post injection (up to 3 months from consent)

Dose limiting toxicity is defined as: submandibular pain \> 5 on a pain scale of 0-10 at 1-month after MSC injection OR any serious AE OR any of the selected toxicities listed per protocol within one-month post-injection.

Outcome measures

Outcome measures
Measure	Treatment With MSCs n=6 Participants A single dose of MSCs injected into the submandibular glands of patients with radiation-induced xerostomia Autologous bone-marrow derived, interferon gamma stimulated mesenchymal stromal cells: Single dose, starting at * Dose Level 0: 10 x 10\^6 injected into one submandibular gland on Day 1
Percentage of Subjects Experiencing Dose Limiting Toxicity (DLT)	0 Participants

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

Whole saliva production rates will be measured under unstimulated and stimulated saliva collection conditions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

Salivary pH will be measured using a pH meter. The normal range of saliva pH is 6.2-7.6 .

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

ELISA will be used to quantify total protein concentration in saliva. The normal range of total protein in saliva is 2-5 mg/mL.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

The enzyme-linked immunosorbent assay (ELISA) will be used to quantify amylase concentration in saliva. The normal range of amylase concentration in saliva is 10-150 U/mL.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

ELISA will be used to quantify mucin concentration in saliva.The normal range of mucin concentration in saliva is 1,000-3,000 ug/mL.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

The University of Michigan Xerostomia Related Quality of Life (XeQOL) scale is a validated patient-reported assessment 15 item scale with 4 domains: physical functioning, pain/discomfort, personal/psychologic functioning, and social functioning. Participants will answer the questions on a scale of 1-5 (not a all, a little, somewhat, quite a bit, very much) for every item. Higher scores represent greater degree of symptoms.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline(up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

The MDADI is a 20-item questionnaire designed for evaluating the impact of dysphagia on the quality of life of patients with head and neck cancer. The MDADI score ranges from 20-100 with a lower scale representing worse dysphagia.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 1, 3, 6, 12, and 24 months post-injection

A Visual Analogue Scale questionnaire for subjective assessment of salivary dysfunction. The VAS xerostomia questionnaire is an 8-item questionnaire that provides a validated measure of the perception of dry mouth. Participants will be asked to mark their responses to each item by placing a vertical line on the 100-mm horizontal scale. The VAS ranges from 8-80 with a lower scale representing less dysphagia/symptoms

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 3, 6, and 12 months post-injection

Salivary gland size measured by ultrasound imaging

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline (up to 8 weeks before injection), 3, 6, and 12 months post-injection

Salivary gland stiffness (fibrosis) will be measured by acoustic radiation force impulse imaging. Evaluators will be blinded to the time point of evaluation (pre- or post- MSC injection)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months post-injection (up to 26 months from consent)

Study feasibility will in part be measured by participant drop out rate.

Outcome measures

Outcome data not reported

Adverse Events

Treatment With MSCs

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment With MSCs n=6 participants at risk A single dose of MSCs injected into the submandibular glands of patients with radiation-induced xerostomia Autologous bone-marrow derived, interferon gamma stimulated mesenchymal stromal cells: Single dose, starting at * Dose Level 0: 10 x 10\^6 injected into one submandibular gland on Day 1
General disorders Injection Site reaction (pain)	50.0% 3/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
General disorders Vaccine site lymphadenopathy	33.3% 2/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Investigations Lymphocyte decrease	33.3% 2/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Musculoskeletal and connective tissue disorders Bone pain (bone marrow aspiration)	33.3% 2/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Musculoskeletal and connective tissue disorders Neck Pain	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Nervous system disorders Dizziness	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Vascular disorders Lymphedema	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Investigations Platelet Count Decrease	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Investigations Cardiac Troponin 1 increase	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Blood and lymphatic system disorders Anemia	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Blood and lymphatic system disorders Eosinophilia	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).
Skin and subcutaneous tissue disorders Telangiectasia	16.7% 1/6 • Toxicities regardless of attribution were recorded through the 3 month post injection visit per protocol (up to 5 months from participant consent) for primary outcomes. Follow up for serious adverse events continues through 24 months post injection (up to 26 months from participant consent).

Additional Information

Randy Kimple, MD, PhD, FASTRO

University of Wisconsin Carbone Cancer Center

Phone: 608-263-5361

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place