Impact of Budesonide on Incidence of ≥ Gr2 Diarrhea in Multiple Myeloma (MM) Patients Undergoing Autologous Stem Cell Transplant

NCT05405387 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: HCI152269
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

A randomized placebo controlled, phase 2 study of budesonide in subjects with multiple myeloma undergoing autologous stem cell transplant (ACST). The study includes a run-in period with 20 patients.

Conditions

Multiple Myeloma; Plasma Cell Leukemia

Outcome Measures

Primary Outcomes (2)

• Proportion of patients with delayed engraftment (engraftment after day 18) and severe infection related complications (≥ Grade 4 NCI CTCAE v5).

  impact of budesonide prophylaxis on transplant related outcomes in patients with multiple myeloma undergoing ASCT

  18 days

• incidence of Grade 2 or higher diarrhea from the time of ASCT until day 14 post ASCT as measured by NCI Common-Terminology Criteria (CTCAE).

  impact of budesonide prophylaxis plus standard of care (SOC) versus standard of care (SOC) alone on the incidence of ≥ Grade 2 diarrhea (NCI CTCAE v5) in multiple myeloma patients receiving high-dose melphalan in preparation for ASCT

  14 days

Secondary Outcomes (67)

• proportion of patients reporting "frequent" or "almost constant" diarrhea on Day -1 of transplant on the PRO-CTCAE score

  Day -1

• proportion of patients reporting "frequent" or "almost constant" diarrhea on Day 7 of transplant on the PRO-CTCAE score

  Day 7

• proportion of patients reporting "frequent" or "almost constant" diarrhea on Day 14 of transplant on the PRO-CTCAE score

  Day 14

• proportion of patients reporting "frequent" or "almost constant" diarrhea on Day 30 of transplant on the PRO-CTCAE score

  Day 30

• proportion of patients reporting "frequent" or "almost constant" diarrhea 3 months post ACST on the PRO-CTCAE score

  3 Months

Study Arms (3)

Run-In Phase

EXPERIMENTAL

Budesonide EC 3 mg three times a day, oral

Drug: Budesonide EC

Arm 1: Treatment

EXPERIMENTAL

Budesonide EC 3 mg three times a day, oral

Drug: Budesonide EC

Arm 2: Placebo

PLACEBO COMPARATOR

Placebo 3 mg three times a day, oral

Drug: Placebo

Interventions

Budesonide EC DRUG

Subjects will take 3mg of Budesonide or placebo. Dosing will begin on day prior to cell infusion and continue until Day 14 post transplant. Budesonide or placebo will be in capsule formulation. Budesonide or Placebo will be administered orally three times daily (every 8 hours ± 1 hours) with or without food.

Also known as: Entocort EC

Arm 1: Treatment; Run-In Phase

Placebo DRUG

Subjects will take 3mg of Budesonide or placebo. Dosing will begin on day prior to cell infusion and continue until Day 14 post transplant. Budesonide or placebo will be in capsule formulation. Budesonide or Placebo will be administered orally three times daily (every 8 hours ± 1 hours) with or without food.

Arm 2: Placebo

Eligibility Criteria

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

You may qualify if:

• Subject aged ≥ 18 years.
• History of histologically confirmed multiple myeloma and/or Plasma Cell Leukemia diagnosis undergoing ASCT who are determined to be fit by the investigator to undergo ASCT with melphalan 200 mg/m2 or melphalan 140 mg/m2 as conditioning.
• Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines.
• Adequate organ function as defined as:
• Hepatic:
• Total Bilirubin ≤ 2 x institutional upper limit of normal (ULN).
• AST(SGOT)/ALT(SGPT) ≤ 5 × institutional ULN
• For female subjects who have not undergone surgical sterilization: Negative pregnancy test or evidence of post-menopausal status. The post-menopausal status will be defined as having been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:
• Women \< 50 years of age:
• Amenorrheic for ≥ 12 months following cessation of exogenous hormonal treatments; and
• Luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution; or
• Underwent surgical sterilization (bilateral oophorectomy or hysterectomy).
• Women ≥ 50 years of age:
• Amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments; or
• Had radiation-induced menopause with last menses \>1 year ago; or

You may not qualify if:

• Ongoing or current use of oral budesonide at the time of enrollment.
• Receiving other investigational agents, unless deemed acceptable after consultation with the PI
• Subjects with moderate or severe pre-existing hepatic impairment as classified according to the Child-Pugh system.
• Prior history or current diagnosis of inflammatory bowel disease, microscopic colitis at baseline.
• Prior history of receiving an allogenic stem cell transplant
• Medical, psychiatric, cognitive, or other conditions that may compromise the subject's ability to understand the subject information, give informed consent, comply with the study protocol or complete the study.
• Known prior severe hypersensitivity to melphalan or budesonide or any component in their formulations or compounds of similar composition (NCI CTCAE v5.0 Grade ≥ 3).
• Subjects taking prohibited medications as described in Section 6.6.1. A washout period of prohibited medications for a period of at least five half-lives or 14 days (whichever is shorter) should occur before the start of treatment.

Sponsors & Collaborators

• University of Utah: lead

Study Sites (1)

Huntsman Cancer Institute at the University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

MeSH Terms

Conditions

Multiple Myeloma; Leukemia, Plasma Cell

Interventions

Budesonide

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Leukemia

Intervention Hierarchy (Ancestors)

Pregnenediones; Pregnenes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Ghulam Rehman Mohy-ud-din, MBBS

  Huntsman Cancer Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rachel Kingsford

CONTACT

801-585-0115; rachel.kingsford@hci.utah.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: To preserve the blind, only the Investigational Drug Services Pharmacy personnel and Data Safety Monitoring Committee (DSMC) at Huntsman Cancer Institute will know treatment assignments.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Stage one is open-label safety run-in. Stage 2 is randomized, placebo-controlled.

Study Record Dates

• First Submitted: May 31, 2022
• First Posted: June 6, 2022
• Study Start: May 3, 2023
• Primary Completion (Estimated): May 19, 2026
• Study Completion (Estimated): September 1, 2027
• Last Updated: March 20, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)