Clinical Research Directory

Nirogacestat in Premenopausal Females With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)

This study is being conducted to study how nirogacestat may affect the ovarian function of adult premenopausal women with progressing desmoid tumors/aggressive fibromatosis.

A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF)

This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.

Familial Adenomatous Poliposys Italian Network (Rete Italiana Poliposi Adenomatosa Familiare)

RIPAF (Rete Italiana Poliposi Adenomatosa Familiare) is a national, multicenter observational registry designed to establish a coordinated Italian network for the management of Familial Adenomatous Polyposis (FAP) and related adenomatous polyposis syndromes. The registry includes patients with APC-related FAP (classic and attenuated forms), MUTYH-associated polyposis (MAP), and adenomatous polyposis not associated with APC or MUTYH mutations (NAMP), including cases linked to other susceptibility genes or without identified pathogenic variants. The study combines retrospective and prospective data collection across 28 Italian centers. Its primary purpose is to generate standardized, large-scale clinical data to better characterize disease presentation and evolution, evaluate current surveillance and surgical strategies, and assess oncological outcomes and quality-of-care indicators in real-world practice. The registry will collect detailed information on genotype-phenotype correlations, colorectal and upper gastrointestinal cancer incidence, desmoid tumor development, timing and type of prophylactic surgery, postoperative outcomes, and long-term survival. Additional objectives include evaluating adherence to surveillance guidelines, timing of genetic diagnosis, and preventive surgical uptake among at-risk relatives. By harmonizing data collection and promoting collaboration among referral centers, RIPAF aims to reduce variability in clinical management across Italy, improve risk stratification and decision-making, and create a national platform to support future multicenter research initiatives and international collaborations in hereditary colorectal cancer syndromes.

A Study of AL102 in Patients With Progressing Desmoid Tumors

The current study is designed to evaluate the efficacy and safety of AL102 in patients with progressive desmoid tumors.

Cryoablation Versus Medical Therapy in Desmoid Tumors Progressing After Watchful Waiting

"Wait \& see" is currently the standard of care of recently diagnosed desmoid tumors (DT). In case of progression or symptomatic disease, medical therapy is nowadays widely used including chemotherapy. Cryoablation has proven to be beneficial for the treatment of large, progressive and symptomatic DT. This randomized phase II trial aims to compare cryoablation versus medical therapy in DT patients progressing after the "wait \& see" period. Moreover, a cross-over design has been anticipated to allow all patients to undergo cryoablation if necessary.

Study of Cryoablation and Nirogacestat for Desmoid Tumor

The primary purpose of this protocol is Systemic therapy with oral study agent, nirogacestat, followed by a single cryoablation procedure.

A Prospective Clinical Study on the Safety and Efficacy of Radiofrequency Ablation for the Treatment of Patients With Desmoid Tumors

This is a prospective study on the safety and effectiveness of radiofrequency ablation in patients with desmoid tumors. In the study group, all patients after radiofrequency ablation of the tumor after 1 month will be evaluated using MRI and CT studies and, if solid components of the tumor are detected, repeated surgical treatment is performed followed by active monitoring after 1 month. In the absence of a solid component, the effect is estimated by the volume of the necrotic process and monitored in dynamics every 3 months.