Trial reportLeukemia & lymphoma2015
Phase II evaluation of IPI-926, an oral Hedgehog inhibitor, in patients with myelofibrosis.
Trial report in Leukemia & lymphoma, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 57 citations in OpenAlex.
- Phase 1/2 trial of glasdegib in patients with primary or secondary myelofibrosis previously treated with ruxolitinib.Leukemia research · 2019Trial
- TGF-β1/SMAD3-mediated Non-canonical Hedgehog Signaling Promotes Pancreatic Stellate Cell Activation and Fibrosis in Chronic Pancreatitis.International journal of biological sciences · 2025Article
- Hedgehog Signaling Pathway in Fibrosis and Targeted Therapies.Biomolecules · 2024Review
- The Role of Mutated Calreticulin in the Pathogenesis ofInternational journal of molecular sciences · 2024Article
- Non-canonical Hedgehog signaling mediates profibrotic hematopoiesis-stroma crosstalk in myeloproliferative neoplasms.Cell reports · 2024Article
- Systematic dissection of coordinated stromal remodeling identifies Sox10Cell stem cell · 2023Article
- The Hedgehog Pathway as a Therapeutic Target in Chronic Myeloid Leukemia.Pharmaceutics · 2023Review
- GLI1 activates pro-fibrotic pathways in myelofibrosis fibrocytes.Cell death & disease · 2022Article
- The hedgehog pathway in hematopoiesis and hematological malignancy.Frontiers in oncology · 2022Review
- Animal and Human Models of Tissue Repair and Fibrosis: An Introduction.Methods in molecular biology (Clifton, N.J.) · 2021Review
- Mechano-therapeutics: Targeting Mechanical Signaling in Fibrosis and Tumor Stroma.Pharmacology & therapeutics · 2020Review
- Neutral effect of Glioma-associated oncogene-1 expression on survival in myelofibrosis.Wiener klinische Wochenschrift · 2020Article
- Management of myelofibrosis after ruxolitinib failure.Leukemia & lymphoma · 2020Article
- Statistical analysis of longitudinal data on tumour growth in mice experiments.Scientific reports · 2020Article
- Bone marrow niche dysregulation in myeloproliferative neoplasms.Haematologica · 2020Review
- Article
- Review
- Review
- Novel-smoothened inhibitors for therapeutic targeting of naïve and drug-resistant hedgehog pathway-driven cancers.Acta pharmacologica Sinica · 2019Article
- Safety and Tolerability of Sonic Hedgehog Pathway Inhibitors in Cancer.Drug safety · 2019Review
Corrections and comments
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Authors and funding
10 authors at 5 institutions in 1 country.
Funding
Abstract
The clinical safety and efficacy of IPI-926 was evaluated in 14 patients with myelofibrosis in a phase II study. Patients received 160 mg IPI-926 orally in continuous 28-day cycles. The median treatment duration was 5.1 months, and all patients had discontinued treatment by 7.5 months. Nine patients discontinued due to lack of response as determined by the treating physician, two after developing acute leukemia and one due to disease progression/loss of response. Twelve patients had slight reductions in spleen size (less than 50% from baseline), but symptoms did not improve consistently. One patient achieved transfusion independence lasting 5 months. Reductions in GLI1 mRNA and protein levels, JAK2V617F allele burden, degree of fibrosis or cytokine levels were observed in some patients, but were not significant when evaluated for the cohort. Low-grade gastrointestinal/liver abnormalities were the most common toxicities. The results did not support continued evaluation of IPI-926 as a monotherapy in myelofibrosis.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.