Dose-Escalation Trial of Mesenchymal Stromal Cells in Patients With Medical Xerostomia

NCT06392711 | Recruiting Phase 1 DMC FDA Drug

• 5 other identifiers: 2024-0386A534290SMPH/MEDICINE/RHEUMATOLUW23129Protocol Version 11/29/25
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the safety and tolerability of injecting certain cells produced in bone marrow called mesenchymal stromal cells (MSCs) into salivary glands. The main question it aims to answer is whether injection of MSCs into salivary glands results in any improvement in dry mouth. Participants will:

• have bone marrow collected using a needle
• undergo a salivary gland ultrasound
• complete questionnaires
• receive an injection of the bone marrow cells into a salivary gland

Conditions

Graft-versus-host-disease; Sjogren's Disease; Xerostomia

Outcome Measures

Primary Outcomes (3)

• Proportion of participants experiencing DLT of submandibular pain

  Recommended phase II dose (RP2D) will be determined by the proportion of subjects experiencing the dose-limiting toxicity (DLT) of submandibular pain \> 5 on a standard 10-point pain scale of 0-10 at 1-month after MSC injection

  1 month post-injection

• Proportion of participants experiencing DLT as serious adverse events (AEs)

  RP2D will be determined by the proportion of subjects experiencing the dose-limiting toxicity (DLT) of any serious AE within one-month post-injection

  1 month post-injection

• Proportion of participants experiencing DLT as pre-specified toxicities

  RP2D will be determined by the proportion of subjects experiencing the dose-limiting toxicity (DLT) of any selected toxicity that is specified in the protocol within one-month post-injection

  1 month post-injection

Secondary Outcomes (6)

• Change in xerostomia scores

  Baseline to 24 months

• Change in salivary function-rate of salivary production

  Baseline, 1 month, 3 months, 6 months, 12 months, and 24 months

• Change in salivary function-saliva composition

  Baseline, 1 month, 3 months, 6 months, 12 months, and 24 months

• Change in salivary function-saliva composition

  Baseline, 1 month, 3 months, 6 months, 12 months, and 24 months

• Change in salivary function-saliva composition

  Baseline, 1 month, 3 months, 6 months, 12 months, and 24 months

Study Arms (3)

MSCs Dose Level 0 into one submandibular gland

ACTIVE COMPARATOR

6 subjects will receive Mesenchymal Stromal Cells (MSC) at Dose Level 0, which is 10 (8-12) x10\^6 MSCs in a single submandibular gland. If this dose is deemed tolerable when injected into a single submandibular gland, this dose will be administered to both submandibular glands in the initial subjects in the dose escalation arm.

Biological: Mesenchymal Stromal Cells (MSC) Dose Level 0

MSCs into both submandibular glands - Dose Escalation Cohort

ACTIVE COMPARATOR

8-18 subjects in the Dose Escalation phase of study will receive MSCs into both submandibular glands. The initial subjects in this cohort will receive Dose Level 0: 10 (8-12) x10\^6 MSCs/gland. If this dose is tolerated, subsequent subjects will receive Dose Level 1: 20 (16-24) x10\^6 MSCs/gland. The highest tolerated dose (recommended phase II dose or RP2D) will be administered to the subjects in the Expansion Cohort.

Biological: Mesenchymal Stromal Cells (MSC) Dose Level 0; Biological: Mesenchymal Stromal Cells (MSC) Dose Level 1

MSCs into both submandibular glands - Expansion Cohort

ACTIVE COMPARATOR

12 subjects in Expansion Cohort will receive MSCs into both submandibular glands at the RP2D.

Biological: Mesenchymal Stromal Cells (MSC) Dose Level 0; Biological: Mesenchymal Stromal Cells (MSC) Dose Level 1

Interventions

Mesenchymal Stromal Cells (MSC) Dose Level 0 BIOLOGICAL

10 (8-12) x 10\^6 MSCs

MSCs Dose Level 0 into one submandibular gland; MSCs into both submandibular glands - Dose Escalation Cohort; MSCs into both submandibular glands - Expansion Cohort

Mesenchymal Stromal Cells (MSC) Dose Level 1 BIOLOGICAL

20 (16-24) x 10\^6

MSCs into both submandibular glands - Dose Escalation Cohort; MSCs into both submandibular glands - Expansion Cohort

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Xerostomia, defined as an unstimulated salivary flow \<1.2 mL in 5 minutes documented at any time following xerostomia diagnosis and prior to enrollment
• Xerostomia not resulting from radiotherapy (medical xerostomia)
• ≥ 18 years of age, ≤ 90 years of age
• Karnofsky performance status ≥ 70, patient eligible for bone marrow aspirate with wakeful anesthesia
• Willing and able to give informed consent
• Radiographically confirmed bilateral submandibular glands
• If female of childbearing potential, negative pregnancy test
• Males and females of childbearing potential willing to use acceptable contraception
• Laboratory Values (within 28 calendar days of enrollment):
• Hgb ≥ 9 g/dL (5.58 mmol/L)
• Platelets ≥ 100,000/µL
• ANC ≥ 1000/µL
• Lymphocytes ≥ 800/µL
• PT/INR and PTT within normal limits based on age/sex

You may not qualify if:

• Patients with one submandibular gland
• Sialolithiasis
• Poorly-controlled diabetes mellitus (HbA1c ≥ 7%)
• Patients who initiated any diuretic therapy before developing dry mouth symptoms and are still on diuretic therapy and the referring provider believes the dryness symptoms are driven by diuretic use
• Untreated oral candidiasis based on physical exam at enrollment
• Malignancy within the last 2 years (except adequately treated stage I lung cancer, low risk prostate cancer that has been treated or is undergoing active surveillance, adequately treated non-melanoma skin cancer, adequately treated DCIS, or adequately treated stage I cervical cancer)
• For patients on immunosuppressive therapy, must be on stable dose of immunosuppressive therapy for at least 2 months, allowing for dose adjustments for blood levels of drugs
• Transfusion dependency
• Life expectancy ≤ 6 months as determined by the investigator
• Use of investigational drugs, biologics, or devices within 30 calendar days prior to enrollment
• Pregnant or lactating women or those who plan to become pregnant during the study
• Not suitable for study participation due to other reasons at discretion of investigators.
• Enrollment in another clinical study possibly interfering with the endpoints of this study

Sponsors & Collaborators

• University of Wisconsin, Madison: lead

Study Sites (1)

University of Wisconsin

Madison, Wisconsin, 53705, United States

RECRUITING

MeSH Terms

Conditions

Xerostomia; Graft vs Host Disease; Sjogren's Syndrome

Condition Hierarchy (Ancestors)

Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Immune System Diseases; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases

Study Officials

• Sara McCoy, MD, PhD

  University of Wisconsin, Madison

  PRINCIPAL INVESTIGATOR

• Jacques Galipeau, MD

  University of Wisconsin, Madison

  STUDY DIRECTOR

Central Study Contacts

Cancer Connect

CONTACT

800-622-8922; clinicaltrials@cancer.wisc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2024
• First Posted: April 30, 2024
• Study Start: October 4, 2024
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2028
• Last Updated: February 10, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)