Experimental drug P1101 tested for rare bone marrow disease
NCT ID NCT02370329
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tested a drug called P1101 (a type of interferon) in 11 people with early myelofibrosis, a rare bone marrow disorder. The goal was to see if the drug could shrink the spleen, improve symptoms, or boost blood counts. The study was terminated early, so the full results are not available.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Ropeginterferon alfa-2b (a type of interferon that boosts the immune system)
- What this could lead to
- If successful, this could point toward a treatment that slows or controls myelofibrosis progression.
- What could go wrong
- This was a very small, early-phase trial that was terminated early, so results are limited. Interferons can cause flu-like symptoms and other side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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11 people
The number who actually took part.
- Started
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Aug 2015
- Finished
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Nov 2023
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Evaluable myelofibrosis by IWG-MRT criteria including one or more of the following: * Spleen \>= 5 cm below the left costal margin * MPN-SAF total symptom score (TSS) \> 10 at baseline * Hemoglobin \< 10 g/dL * Confirmed diagnosis of myelofibrosis (primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia vera) by World Health Organization (WHO) diagnostic criteria (3 major and 2 minor criteria: major criteria: megakaryocyte proliferation and atypia with either reticulin and/or collagen fibrosis, not meeting criteria for chronic myelogenous leukemia \[CML\], polycythemia vera \[PV\], myelodysplastic syndrome \[MDS\], or other myeloid neoplasm, JAK2V617F or other clonal marker or no evidence of reactive marrow fibrosis; minor criteria: leukoerythroblastosis, increased lactate dehydrogenase \[LDH\], anemia, palpable splenomegaly) * For cohort 1: early stage MF (low or intermediate 1 stage as defined by Dynamic International Prognostic Scoring System \[DIPSS\]) without currently available treatment options * For cohort 2: intermediate-2 or high risk MF patients as defined by DIPSS either not eligible for ruxolitinib or having failed under ruxolitinib * No prior treatment for myelofibrosis (for cohort 1 only) * Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2 * Platelet count \>= 100,000/mm\^3 (obtained =\< 14 days prior to registration) * Absolute neutrophil count (ANC) \>= 1000/mm\^3 (obtained =\< 14 days prior to registration) * Aspartate transaminase (AST) =\< 2.5 x upper limit of normal (ULN) (obtained =\< 14 days prior to registration) * Alanine aminotransferase (ALT) =\< 2.5 x ULN (obtained =\< 14 days prior to registration) * Calculated creatinine clearance must be \>= 50 ml/min using the Cockcroft-Gault formula (obtained =\< 14 days prior to registration) * Negative pregnancy test done =\< 7 days prior to registration, for women of childbearing potential only * Ability to complete questionnaire(s) by themselves or with assistance * Provide informed written consent * Willing to return to enrolling institution for follow-up * Willing to provide blood samples for correlative research purposes Exclusion Criteria: * Patients who have had chemotherapy or radiation =\< 2 weeks of registration * For cohort 1 only: patients with evidence of intermediate 2 or high risk disease (according to DIPSS) * For cohort 1 only: patients with a bone marrow biopsy with \< 15% cellularity, evidence of collagen fibrosis, osteosclerosis, or blasts \> 10% in peripheral blood or marrow (demonstrating advanced disease) * Patients who have received a prior stem cell transplant * Patients who have received radiation to the spleen within 3 months prior to registration * Patients with intolerance to compounds similar to pegylated interferon alpha-2b * Patients with evidence of \>= grade 2 peripheral sensory neuropathy * Any of the following: * Pregnant women * Nursing women * Men or women of childbearing potential who are unwilling to employ adequate contraception * Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens * Immunocompromised patients or patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy * Uncontrolled simultaneous illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, history of depression, or psychiatric illness/social situations that would limit compliance with study requirements * Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm * History of myocardial infarction =\< 6 months prior to registration, or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias * History of significant or major funduscopic findings including, but not limited to, retinal exudates, hemorrhage, detachment, neovascularization, papilledema, optic atrophy, micro-aneurysm or macular changes * Other active malignancy at time of registration; EXCEPTIONS: non-melanotic skin cancer or carcinoma-in-situ of the cervix
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Mayo Clinic in Arizona
Scottsdale, Arizona, 85259, United States
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