P2, N=70, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Suspended --> Recruiting

Targeting these pathways, including BMPR2 restoration, TGF-β inhibition, and tyrosine kinase blockade, has shown encouraging results beyond the vasodilator-focused standard of care. These innovations may reshape PAH management by improving outcomes, and potentially altering disease progression.

P2, N=25, Not yet recruiting, Philipps University Marburg

P3, N=815, Recruiting, Merck Sharp & Dohme LLC | Not yet recruiting --> Recruiting

P2, N=130, Active, not recruiting, Merck Sharp & Dohme LLC | Recruiting --> Active, not recruiting | Trial completion date: Sep 2027 --> Jun 2028 | Trial primary completion date: Sep 2027 --> Jun 2028

P2, N=164, Completed, Merck Sharp & Dohme LLC | Active, not recruiting --> Completed

New therapeutic options to reduce the red blood cell (RBC) transfusion burden have emerged since 2020 and include luspatercept and imetelstat. Erythropoiesis-stimulating agents and lenalidomide also address anemia and are generally recommended to start at the time of transfusion dependency, although emerging data suggest that an earlier start of these interventions might offer clinical benefits...Targeted therapy directed to the presence of an IDH1 mutation is U.S. Food and Drug Administration (FDA) approved for the rare IDH1 mutated MDS (<10% of the time) and consideration to use an IDH2 inhibitor for IDH2 mutated MDS (<5% of the time) is reasonable. Interestingly, IDH mutations seem to appear with increased frequency in older patients and in patients with underlying autoimmune/rheumatological disorders.1.

Current therapeutic strategies remain largely experimental, focusing on antifibrotic agents such as pirfenidone, TGF-β inhibitors and modulation of oxidative stress, alongside emerging interventions such as stem cell therapies, which are offer potential avenues for intervention in the ovary. This review synthesizes current insights into the cellular and molecular mechanisms driving ovarian fibrosis, its association with reproductive disorders, and emerging therapeutic strategies. It underscores key knowledge gaps and emphasizes the need for future research focused on fibroblast activation, inflammatory signaling and immune-ECM interactions to facilitate the development of targeted, long-term interventions aimed at preventing or reversing ovarian fibrosis and preserving female fertility.

P=N/A, N=232, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Nov 2025 --> Nov 2027 | Trial primary completion date: Nov 2025 --> Nov 2027

These findings demonstrate that serum tumor markers and pulmonary function parameters reflect disease heterogeneity between PF and PF+LC, with Cyfra21-1, FVC, DLCO, age, and fibrosis type serving as important survival determinants. Additionally, pirfenidone therapy may reduce lung cancer-related mortality, underscoring its potential therapeutic benefit in managing PF+LC.

P3, N=106, Active, not recruiting, Bristol-Myers Squibb | Trial primary completion date: Jan 2026 --> Oct 2025

P2, N=160, Recruiting, Takeda | Trial completion date: Oct 2030 --> Oct 2031