Allogeneic Hematopoietic Cell Transplantation With Pegylated Interferon Alfa-2a for Primary and Secondary Myelofibrosis

NCT05535764 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: HCI149616
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single site, open-label, dose de-escalation, Phase 1 study of pegylated interferon alfa-2a administered after alloHCT in subjects with primary or secondary myelofibrosis. Part 1 of the study will assess the rate of dose-limiting toxicities (DLTs) during the DLT evaluation period and identify the Recommended Phase 2 Dose (RP2D). Once the RP2D is identified, 6 additional patients will be enrolled in the expansion cohort.

Conditions

Myelofibrosis

Outcome Measures

Primary Outcomes (1)

• Rate of dose-limiting toxicities (DLTs) during the DLT evaluation period

  To identify the Recommended Phase 2 Dose (RP2D) of pegylated interferon alfa-2a in subjects undergoing allogeneic hematopoietic cell transplantation for primary or secondary myelofibrosis

  Start of treatment to 86 days after treatment initiation

Secondary Outcomes (6)

• Assess the safety of pegylated interferon alfa-2a in the study population.

  3 years

• Assess the tolerability of pegylated interferon alfa-2a in the study population.

  3 years

• Assess the rate of treatment-related mortality in the study population.

  3 years

• Assess leukemia-free survival (LFS) in the study population.

  3 years

• Assess the incidence of acute and/or chronic graft versus host disease (GVHD) in the study population

  3 years

Study Arms (1)

Treatment: All Patients

EXPERIMENTAL

A 3+3 dose de-escalation design will be used to determine the recommended phase 2 dose,while ensuring the safety and tolerability of the treatment. In this trial, the dose determined to be the maximum tolerated dose will be the recommended phase 2 dose and will be utilized in the cohort expansion.

Drug: Pegylated interferon alpha2a

Interventions

Pegylated interferon alpha2a DRUG

PegINFa will be given once every 2 weeks by subcutaneous administration in the abdomen or thigh.

Treatment: All Patients

Eligibility Criteria

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

You may qualify if:

• Male or female subject aged ≥ 18 years.
• Diagnosis of primary or secondary myelofibrosis.
• Eligible to undergo a myeloablative or reduced intensity conditioning regimen (MAC or RIC)
• Eligible to undergo a standard of care bone marrow biopsy with aspirate as part of his or her routine pre-transplant work-up.
• Peripheral blood stem cell (PBSC) graft
• /10 HLA matched related or matched unrelated donor
• ECOG performance status ≤ 2.
• For female subjects: 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:

• Receiving other investigational agents concurrently
• Prior systemic anti-cancer therapy or any investigational therapy within five half-lives prior to starting study treatment.
• Prior radiotherapy within 6 weeks prior to the first dose of study treatment.
• Major surgery within 6 weeks prior to starting study drug or patients who have not fully recovered from major surgery.
• The diagnosis of another malignancy within ≤ 2 years before study enrollment, except for those considered to be adequately treated with no evidence of disease or symptoms and/or will not require therapy during the study duration (i.e., basal cell or squamous cell skin cancer, carcinoma in situ of the breast, bladder or of the cervix, or low-grade prostate cancer with Gleason Score ≤ 6).
• Current evidence of uncontrolled, significant intercurrent illness including, but not limited to, the following conditions:
• Graft-versus-host disease:
• Acute or chronic
• Cardiovascular disorders:
• Congestive heart failure New York Heart Association Class III or IV, unstable angina pectoris, serious cardiac arrhythmias.
• Stroke (including transient ischemic attack \[TIA\]), myocardial infarction (MI), or other ischemic events, or thromboembolic event (eg, deep venous thrombosis, pulmonary embolism) within 3 months before the first dose.
• QTc prolongation defined as a QTcF \> 500 ms.
• Known congenital long QT.
• Left ventricular ejection fraction \< 55%.
• Uncontrolled hypertension defined as ≥ 140/90 as assessed from the mean of three consecutive blood pressure measurements taken over 10 minutes.

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

Primary Myelofibrosis

Interventions

peginterferon alfa-2a

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Sagar Patel, MD

  Huntsman Cancer Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Catherine Cromar

CONTACT

801-213-5652; catherine.cromar@hci.utah.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2022
• First Posted: September 10, 2022
• Study Start: August 2, 2023
• Primary Completion: April 1, 2026
• Study Completion (Estimated): October 30, 2027
• Last Updated: May 7, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)