About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

Pegylated interferon alpha2a
Drug
PegINFa will be given once every 2 weeks by subcutaneous administration in the abdomen or thigh.

Treatment groups

18 Participants

are divided into 1 treatment group

Group A: Treatment: All PatientsExperimental treatment 1 intervention

Trial outcomes

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

Time frame

Start of treatment to 86 days after treatment initiation

Secondary outcomes

1

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

Frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by type, severity (as defined by the NIH CTCAE version 5.0), seriousness, duration, and relationship to study treatment.

Time frame

3 years

2

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

Frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by type, severity (as defined by the NIH CTCAE version 5.0), seriousness, duration, and relationship to study treatment.

Time frame

3 years

3

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

Rate of treatment-related mortality which is defined as proportion of deaths in patients who have received at least one dose of study therapy, which is attributed to complications from alloHCT or conditioning therapy (in the absence of myelofibrosis relapse) from 30 days after the end of conditioning therapy to one week after the last dose of study therapy.

Time frame

3 years

4

Assess Objective Response Rate (ORR) in the study population.

Response rates according to IWG-MRT including, CR, PR, CI, PD, SD, or Relapse.

Time frame

3 Years

AlloHCT With PegINFa for Myelofibrosis

About this trial

Eligibility criteria

Qualifiers

Disqualifiers

Trial design

Treatments tested in this trial

Treatment groups

Trial outcomes

Primary outcomes

Secondary outcomes

Other outcomes

Sponsors and contacts