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1932 citation(s) found for "Nuñez, Jessica".

BASECAMP-1: An approach to improve patient screening efficiency and to provide large datasets for precision medicine clinical trials.
Spencer, Kristen , Ulrickson, Matthew L. , Grierson, Patrick , Patel, Sandip Pravin , Specht, Jennifer M. , Morelli, Maria Pia , Maloney, David G. , Weng, Wen-Kai, Waschke, Brittany, Ng, Eric Wai-Choi , Tebbets, Jessica , Hecht, J. Randolph, Molina, Julian R. , Specht, Jennifer , Park, Jong Chul , Murthy, Hemant S. , Simeone, Diane M. , Maus, Marcela V , Weng, Wen‐Kai , Eng, Cathy , He, Kai , Kirtane, Kedar

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: No page available

10.1200/jco.2025.43.16_suppl.e23020 Get It

e23020 Background: Precision medicine studies must overcome the challenges of identifying patients with uncommon molecular targets and small cohort sizes for correlative studies. BASECAMP-1 (NCT04981119) is a prescreening study that uses a single assay to address both problems by: 1) efficiently screening potentially eligible patients for subsequent clinical trials of Tmod logic-gated chimeric antigen receptor T-cell (CAR T) therapies; and 2) providing a large dataset available for translational studies. Methods: Initially, patients were identified for BASECAMP-1 by consenting and genotyping all potentially eligible patients for human leukocyte antigen (HLA)-A*02 heterozygosity. To improve screening efficiency, we co-developed a patient-matching program with Tempus AI, Inc (Tempus), in which Tempus informs the principal investigators of next-generation sequenced (NGS) patients with HLA-A*02 heterozygosity within their health system who may be eligible for BASECAMP-1. Patients are screened using the Tempus xT platform, which uses genomic and RNA sequencing (RNAseq) analyses to determine gene mutations and expression. Enrolled patients with colorectal (CRC), non-small cell lung, ovarian (OVCA), and pancreatic cancers (PANC) were analyzed for tumor purity, mutation distribution, HLA-A*02 loss of heterozygosity (LOH) status, and RNAseq to validate the data available for future clinical analyses. Results: As of January 3, 2025, 80 participants have been enrolled in BASECAMP-1. Through individual screening methods, 1684 patients were screened at 13 study sites, of which 30 patients had tumor-specific HLA-A*02 LOH and were enrolled (~1 eligible patient identified per 56 screened). The patient-matching program identified 282 patients with HLA-A*02 LOH at 12 sites; of these, 50 consented to the BASECAMP-1 study (~1 patient enrolled per 6 identified), demonstrating higher efficiency than the individual patient screening method. Correlative data from xT were available for 360 patients with germline HLA-A*02 and provided insights into mutational distribution and target expression. LOH status was not associated with tumor-specific differences in missense mutational distribution. RNAseq data demonstrated higher CEACAM5 expression in CRC and higher MSLN in PANC and OVCA vs other tumor types, while EGFR was consistently expressed with reduced variance across tumor types, confirming these as potential target antigens for Tmod CAR T interventional trials EVEREST-1, EVEREST-2, and DENALI-1. Conclusions: Identification and enrollment of HLA-A*02 LOH patients into BASECAMP-1 for logic-gated CAR T therapeutic studies were accelerated by the patient matching program. Concurrent xT analysis provides opportunities to augment analyses from robust propensity-matched comparisons, response correlation studies, and other translational discovery. Clinical trial information: NCT04981119 .
Comparative effectiveness of delivering early palliative care via video versus in-person on end-of-life outcomes in patients with advanced lung cancer.
Bauman, Jessica R. , El‐Jawahri, Areej , Rinaldi, Simone , Jackson, Vicki A. , Gallagher-Medeiros, Emily R., Rabideau, Dustin J. , Strand, Jacob J. , Zachariah, Finly , Shoemaker, Laura, Spoozak, Lori , Grey, Carl, Curseen, Kimberly , Szmuilowicz, Eytan , Rhodes, Ramona L. , Cullinan, Amelia , Horick, Nora , Bakitas, Marie , Jewett, Brianna, Temel, Jennifer S. , Greer, Joseph Andrew

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: 1649-1649

10.1200/jco.2025.43.16_suppl.1649 Get It

1649 Background: Findings from our prior large-scale comparative effectiveness trial showed the equivalent effect of delivering early palliative care via video versus in-person on quality of life among patients with advanced non-small cell lung cancer (NSCLC). We now report on whether the two care delivery modalities were equivalent with respect to patient-reported communication with clinicians about their end-of-life (EOL) care preferences and hospice utilization. Methods: Between 6/14/2018 and 5/4/2023, we enrolled 1250 patients with newly diagnosed advanced NSCLC in a randomized trial of early palliative care across 22 US cancer centers. Patients were randomly assigned to meet with a palliative care clinician every 4 weeks from enrollment through the course of the disease, either via video or in the outpatient clinic. Participants completed self-report surveys at baseline and weeks 12, 24, 36, and 48, including an item asking if they had discussed with their clinicians the care they would want to receive if dying (yes/no); patients’ final assessments prior to death or last follow up were analyzed. We reviewed patients’ health records to collect data on hospice referral and length of stay. To test the equivalence in these outcomes, we used a binomial generalized linear model with the identity link function (pre-specified equivalence margin of ±8% for patient-reported communication about EOL care) and linear regression (pre-specified equivalence margin of ±6 days for mean length of stay in hospice). P-values were adjusted for multiplicity using a Bonferroni correction. Results: Of the 1250 enrolled participants, 888 (71.0%) completed at least one survey post baseline regarding whether they communicated with clinicians about EOL care preferences. Among those, 29.1% of the video group and 26.0% of the in-person group reported “yes,” indicating that they recalled such EOL care discussions (difference = 3.1%, 95% CI: -1.8%, 8.1%; p = 0.26 for equivalence). During the course of the trial, 733/1250 (58.6%) patients died, of whom 537/733 (73.3%) were referred to hospice. Mean lengths of hospice stay were 25.3 days for the video group versus 25.1 days for the in-person group (difference = 0.2, 95% CI: -7.0, 7.4; p = 0.46 for equivalence). When excluding outlying patients receiving hospice services > 180 days (n = 13), the mean lengths of hospice stay were 19.1 (video group) versus 19.7 (in-person group) days (difference = -0.6, 95% CI: -4.6, 3.3; p = 0.06 for equivalence). Conclusions: Although thresholds were not met to confirm equivalence statistically, the two modalities for delivering early palliative care demonstrate very similar outcomes with respect to patient-clinician communication about EOL care and hospice utilization. These findings provide further evidence of the utility of video visits for providing high quality palliative and EOL care. Clinical trial information: ClinicalTrials.gov Identifier ( NCT03375489 ) .
Initial results of MC200710 investigating therapeutic vaccine (PDS0101) alone or with pembrolizumab prior to surgery or radiation therapy for locally advanced HPV associated oropharyngeal carcinoma, a phase 2 window of opportunity trial.
Price, Katharine A. , Asmus, Erik, Foster, Nathan R. , Moore, Eric J. , Tasche, Kendall K. , Erskine, Courtney L. , Jeng-Lin, Daniel, Neben-Wittich, M.A. , Wilson, Jessica , Block, Matthew S. , Routman, David M. , Price, Daniel L. , Yin, Linda X. , McGarrah, Patrick W. , Fuentes, Harry E , Bartemes, Kathleen R. , Stoff, Ronen , Lester, Scott C. , Abel, Kathryn M. Van , Chintakuntlawar, Ashish V.

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: 6061-6061

10.1200/jco.2025.43.16_suppl.6061 Get It

6061 Background: PDS0101 is a T cell stimulating immunotherapy (therapeutic vaccine) targeting HPV16. The combination of PDS0101 + pembrolizumab (5 cycles) has shown durable clinical responses in HPV-positive recurrent/metastatic HNSCC. MC200710 is a window of opportunity study in patients with HPV-associated oropharyngeal squamous cell carcinoma (HPV+ OPSCC). This prospective phase II trial utilized 2 cycles of neoadjuvant PDS0101 aloneor in combination with pembrolizumab prior to surgical resection or chemoradiotherapy (CRT). Herein, we report the initial results, including primary endpoint of ctDNA response. Methods: Between June 2022 and September 2024, 20 patients (10 per Arm) with locally advanced HPV+ OPSCC were enrolled in a sequential alternating design. Arm A received 2 cycles of PDS0101 and Arm B received 2 cycles of PDS0101 and pembrolizumab with all patients undergoing subsequent surgery or CRT. Assessments were done at baseline, post cycle 1, and post cycle 2 (prior to surgery or CRT). The coprimary endpoint of ctDNA response was defined as a ≥50% decline in ctDNA post cycle 2 compared to baseline as quantified using NavDx (TTMV fragments/mL). Radiologic objective response rate (ORR) was assessed as per RECIST 1.1. Toxicity through the neoadjuvant period was assessed using CTCAE criteria and recurrence rates following surgery or CRT are reported. Results: Patients were similar between arms: male 90%, median age 61 years, cT1/T2 70%, cN1 65%, and no smoking history 65%. All patients completed both cycles of therapy with 13 (65%) undergoing primary operative management and 7 CRT (35%). The most common toxicity was injection site reaction 85% grade 1, 15% grade 2, 0% grade 3, consistent with prior studies. One patient experienced grade 2 pneumonitis during the neoadjuvant window (Arm B). There was one grade 3 toxicity (5%) possibly attributable to study intervention with one patient experiencing transient hepatitis requiring hospitalization (Arm A). Zero of 10 patients in Arm A had a ≥50% decline in ctDNA from baseline while 5 of 10 patients (50%) met this primary endpoint in ARM B (p=0.03). After Cycle 2, based on RECIST 1.1, no Arm A patients had a partial response (PR), with 7 having stable disease (SD); Arm B had 2 patients with PR and 8 with SD. With median follow up of 6 months, 2 patients in Arm A recurred and 0 patients in Arm B. Conclusions: The combination of PDS0101 and pembrolizumab met the trial’s primary endpoint of ctDNA response and shows promise for further evaluation. ctDNA can be used to assess early response and future studies could use ctDNA to adapt neoadjuvant therapy. Based on these findings, a neoadjuvant dose optimization study in HPV16+ oropharyngeal carcinoma is warranted and evaluation of PDS0101 and pembrolizumab in comparison to pembrolizumab alone. Clinical trial information: NCT05232851 .
Ziftomenib in relapsed/refractory (R/R) NPM1 -mutant acute myeloid leukemia (AML): Phase 1b/2 clinical activity and safety results from the pivotal KOMET-001 study.
Foran, James M. , Rodríguez‐Arbolí, Eduardo , Fedorov, Kateryna , Altman, Jessica K. , Baer, Maria R. , Pettit, Kristen , Zhang, Zijing , Riches, Marcie , Corum, Daniel, Leoni, Mollie , Fathi, Amir T. , Pettit, Kristen , Wang, Eunice S. , Montesinos, Pau , Issa, Ghayas , Erba, Harry Paul , Heiblig, Maël , Heidel, Florian H. , Adès, Lionel , Peterlin, Pierre , Papayannidis, Cristina

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: 6506-6506

10.1200/jco.2025.43.16_suppl.6506 Get It

6506 Background: NPM1 -m drives leukemogenesis in ~30% of AML. Despite current risk stratification, nearly half will have R/R disease within a year, after which outcomes are poor with <10% complete response following chemotherapy. Ziftomenib – a potent, highly selective, oral, investigational menin inhibitor – has shown clinical activity as monotherapy and in combination for adults with R/R NPM1 -m and KMT2A -r AML, with 600 mg once-daily (QD) as the recommended phase 2 monotherapy dose for NPM1 -m. Here we present the primary analysis for NPM1 -m patients (pts) treated with ziftomenib 600 mg QD in the pivotal KOMET-001 study. Methods: KOMET-001 (NCT04067336) is a multicenter, open-label phase 1/2 study of ziftomenib in adults with R/R AML. In phase 2, pts with NPM1 -m R/R AML received ziftomenib 600 mg QD. Phase 2 primary endpoint: complete remission with full/partial hematologic recovery (CR/CRh); key secondary endpoints: composite complete remission (CRc), durations of CR/CRh and CRc, and safety. The analyses below include NPM1 -m pts pooled from phase 1b/2. Results: The phase 2 primary endpoint was met ( p =0.0058). As of 20 Dec 2024, 112 pts were enrolled (51% US/Canada, 49% Europe/UK) in phase 1b/2 and treated with ziftomenib 600 mg QD, with a median follow-up of 4.2 months. Median age was 69 yrs (range 22–86), 56% female, 83% ECOG PS 0–1, median of 2 prior therapies (range 1–7), including 60% prior venetoclax (VEN) and 23% prior transplant. CR/CRh rate in all phase 1b/2 pts was 25% (28/112; 95% CI 17–34) and overall response rate was 35% (39/112; 95% CI 26–44). In phase 2 pts, 23% (21/92; 95% CI 15–33) achieved CR/CRh (Table), with 67% (10/15) MRD negativity among CR/CRh responders tested (local). Comparable CR/CRh rates were observed in both VEN-naïve and exposed pts (21% vs. 24%). Ziftomenib was well tolerated with 3% (3/112) discontinuing due to treatment-related adverse events (TRAEs). 40% (45/112) of pts had Grade (Gr) ≥3 TRAEs, including 13% differentiation syndrome (all Gr3), ≤5% each anemia, febrile neutropenia and thrombocytopenia, and 2% QTc prolongation (Gr3). Updated clinical activity and safety data will be presented. Conclusions: In the pivotal KOMET-001, the phase 2 primary endpoint was met: Ziftomenib achieved deep and durable responses in R/R NPM1 -m AML, regardless of prior VEN. Ziftomenib was well tolerated with limited myelosuppression and only 3% ziftomenib-related discontinuations. Taken together, these data support the potential use of ziftomenib monotherapy as a new treatment option for R/R NPM1 -m AML. Clinical trial information: NCT04067336 . Response, n (%) Phase 2600 mg QDN=92 Phase 1b/2600 mg QDN=112 CR 13 (14) 20 (18) CR/CRh 21 (23) 28 (25) CRc 24 (26) 32 (29) Median duration, months (95% CI) CR/CRh 3.7 (1.9–NE) 3.7 (1.9–7.7) CRc 4.6 (2.8–11.4) 5.1 (2.8–8.1) Restricted mean duration, months (95% CI) CR/CRh 4.3 (3.1–5.6) 5.2 (3.6–6.7) CRc 5.9 (4.0–7.7) 6.4 (4.6–8.1)
Comparing trends in telehealth utilization by medical oncology practices at Stanford Medicine and Mayo Clinic from 2019–2023.
Parameswaran, Vijaya , Miller, Marci, Grimm, Jessica, Gullerud, Rachel E., Fan, Winnie, Gibbons, Daniel J., Oyarzabal, Beatriz A., Lunde, Julianne J. , Rosengaus, Leah, Sharp, Christopher , Shah, Sumit , Haddad, Tufia C. , Grimm, Jessica, Coffey, Jordan , Harper, Sarah, Demaerschalk, Bart M. , Pritchett, Joshua C.

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: No page available

10.1200/jco.2025.43.16_suppl.e13890 Get It

e13890 Background: Many oncology practices have reported continued use of telemedicine since onset of the COVID-19 pandemic. However, the future sustainability of telemedicine within oncology practices remains unclear, particularly because federal policies that enabled adoption in response to the public health emergency (PHE) have not been made permanent. Understanding similarities and differences in telemedicine utilization at high-volume oncology centers is crucial for shaping evidence-based policy that enables equitable access to oncology care across varying geographies. Methods: This is a cross-sectional, year-over-year, retrospective cohort analysis of trends in telemedicine utilization across all medical oncology clinical appointments conducted among adult patients (age >18) from January 2019 to December 2023 at Stanford Medicine Cancer Center (SM) – comprising multiple campuses in the California Bay Area – and the Mayo Clinic Comprehensive Cancer Center (MC) – a multiregional cancer practice with tertiary referral campuses in Minnesota, Florida, and Arizona, as well as community-based clinics throughout the Upper Midwest. Results: Both institutions exhibited a similar trend in telemedicine adoption over time. This was characterized by an initial surge in utilization following the PHE declaration in March 2020, followed by sustained utilization rates from 2021 to 2023. However, telemedicine use as a proportion of the overall practice differed significantly between institutions. At SM, the rate of telemedicine visits as a proportion of overall clinical practice was 60.3% (49,136/ 81,502) in 2021, 59.9% (52,247/ 87,215) in 2022, and 57.2% (55,007/ 96,163) in 2023. At MC, the proportion of telemedicine utilization was 12.8% in 2021 (23,873/186,086), 14.7% in 2022 (28,985/196,777), and 14.0% in 2023 (30,208/215,941). Deployment in the setting of new versus return visits also differed between institutions. In 2023, telemedicine accounted for 53.7% (6,440/11,998) of new and 57.7% (48,567/84,165) of return visits at SM, compared to 15.5% (3,359/21,728) and 13.8% (26,849/194,213) at MC. Additionally, while video visits were the predominant modality used to conduct telemedicine visits at both institutions from 2021 onward, in 2023 phone visits still made up 17.9% (5,394/30,208) of all telemedicine visits at MC, compared to just 5.4% (2,990/55,007) at SM. Conclusions: Stanford Medicine and Mayo Clinic exhibited a pattern of initial growth of telemedicine adoption in 2020, followed by sustained utilization from 2021-2023. We observed unique differences in the overall proportion, visit type deployment, and modality of telemedicine utilization between institutions. However, this study confirms telemedicine visits have remained a fixture of medical oncology care for many patients at these institutions since the COVID-19 pandemic.
Clinical impact of integrating polygenic risk scores with breast cancer risk assessment models: Results from the prospective multisite GENRE-2 clinical trial.
Pruthi, Sandhya , Klassen, Christine L. , Fraker, Jessica , Chumsri, Saranya , Khatri, Lakshmi, Vachon, Celine M. , Schaid, Daniel J. , Carlson, Erin E. , Ongie, Laura J., Hasadsri, Linda , Yadav, Siddhartha , Stan, Daniela , Khan, Seema A. , Vachon, Celine M. , Sinnwell, Jason P. , Couch, Fergus J.

Journal of Clinical Oncology | May 28, 2025 | Volume: 43 | Issue: 16_suppl | Pages: 10544-10544

10.1200/jco.2025.43.16_suppl.10544 Get It

10544 Background: Incorporation of Polygenic Risk Scores (PRS) can refine traditional breast cancer risk assessment models to provide precise estimates of breast cancer risk. However, the impact of such an integrated model on clinical decision-making related to breast cancer surveillance and preventive strategies is not fully understood. Methods: The GENRE-2 is a prospective single-arm multisite clinical trial (NCT04474834) incorporating PRS into standard breast cancer risk assessment models to determine the impact of PRS on clinical decisions on breast cancer prevention and surveillance. Women at high risk of breast cancer due to NCI-BCRAT 5-year risk of ≥ 3%, or IBIS (Tyrer-Cuzik) 10-year breast cancer risk of ≥5%, biopsy-proven high-risk breast lesion, or a pathogenic variant (PV) in ATM , BRCA1, BRCA2, CHEK2 or PALB2 , were enrolled from five sites in the United States. All women were invited to complete surveys on their breast surveillance and cancer prevention decisions based on pre-PRS standard risk models and post-PRS risk estimation, and further annual surveys are planned for 10 years. Results: Among 902 women enrolled in the study, 605 (median age: 52 years) received PRS results and completed a survey to date. Of those who received PRS results,195 (32.2%) were PV carriers. Among non-carriers, the median 10-year and lifetime pre-PRS IBIS-based risk was 10.0% and 28.7%, respectively. Among PV carriers, the CanRisk-based 10-year and lifetime pre-PRS risk estimates were 6.3% and 25.2% for ATM , 22.3% and 77.6% for BRCA1 , 18.5% and 77.7% for BRCA2 , 7.1% and 25.7% for CHEK2 , and 16.8% and 38.0% for PALB2 PV carriers, respectively. After the incorporation of PRS, the lifetime risk of breast cancer increased by at least 10% in 31% of non-carriers and 7.7% of PV carriers and decreased by at least 10% in 10.7% of non-carriers and 7.7% of PV carriers. The proportion of non-carriers with lifetime risk < 20% or > 40% changed from 17.3% and 22.0%, respectively, in the pre-PRS evaluation to 24.4% and 32.9%, in the post-PRS evaluation. A higher lifetime post-PRS score was associated with intent to take preventive action (surgery or endocrine agents). In non-carriers, the proportion of women with a lifetime risk < 20% with intent to take preventive action was 11%, compared to 36.8% of those with a lifetime risk > 40% (p < 0.001). Similarly, in PV carriers, the proportion of women with lifetime risk < 20% with intent to take preventive action was 20.8% compared to 41.1% of those with lifetime risk > 40% (p = 0.015). Conclusions: The GENRE-2 trial demonstrates that the incorporation of PRS into breast cancer risk assessment models in high-risk women is feasible and leads to clinically meaningful changes in breast cancer risk estimates and decision-making regarding preventive strategies. Evaluation of the implementation of breast cancer risk management strategies in study participants is ongoing and will be reported. Clinical trial information: NCT04474834 .
Phase Determination and Demonstration of Parental Mosaicism of Intragenic PRKN Deletions Initially Identified by Chromosomal Microarray Analysis
Choate, Lauren A. , Hoffman, Francis, Newman, Jessica , Runke, Cassandra , Webley, Matthew, Hoppman, Nicole L., Thorland, Erik C., Newman, Jessica H, Hoffman, Francis, Newman, Jessica H.

Genes | May 25, 2025 | Volume: 16 | Issue: 6 | Pages: 630

10.3390/genes16060630 | PMID 40565523 Get It

Background: Autosomal recessive juvenile Parkinson disease (ARJP) is an early-onset neurodegenerative disorder characterized by Parkinsonian motor symptoms with slow progression and preserved cognition. Biallelic pathogenic variants within the PRKN gene are associated with ARJP. Among PRKN pathogenic variants, deletions are a frequent occurrence and may be identified through chromosomal microarray testing. Methods: Here we present a case with two intragenic PRKN deletions initially identified as a secondary finding using chromosomal microarray. One deletion was paternally inherited and the second initially appeared to be de novo. In addition to microarray which initially identified the two deletions, long-range GAP-PCR and Sanger sequencing were used to further characterize the de novo deletion and phase of the deletions. Results: Molecular characterization of the apparently de novo deletion demonstrated low-level maternal mosaicism of this deletion, thus proving that these deletions are in trans in the proband, yielding a diagnosis of autosomal recessive juvenile Parkinson disease. Conclusions: This case highlights the utility of a diagnostic approach combining microarray, long-range PCR, and Sanger sequencing to establish the phase and confirm biallelic PRKN deletions in a patient with ARJP. Furthermore, these findings highlight the importance of investigating the possibility of parental mosaicism to determine the phase of autosomal recessive variants and establish accurate recurrence risks.
0831 Effectiveness and Safety of Low-Sodium Oxybate in Participants with Narcolepsy: Results from the DUET Study
Schneider, Logan , Ruoff, Chad , Nichols, Deborah A. , Steininger, Teresa L. , Fuller, Douglas S. , Akerman, Sarah , Alexander, Jessica, Whalen, Marisa, Cairns, Alyssa , Ruoff, Chad M , Plante, David T. , Kirby, M

SLEEP | May 19, 2025 | Volume: 48 | Issue: Supplement_1 | Pages: A360-A360

10.1093/sleep/zsaf090.0831 Get It

Abstract Introduction Jazz DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) is a phase 4, prospective, multicenter, single-arm, multiple-cohort, open-label study (NCT05875974) assessing the effectiveness of low-sodium oxybate (LXB; Xywav®) treatment on daytime and nighttime symptoms in participants with narcolepsy (type 1 [NT1] or 2 [NT2]) or idiopathic hypersomnia. Data presented are from the narcolepsy cohort. Methods DUET included a screening period (with 2-week washout for current oxybate users), 8-day baseline (BL) period, 2- to 8-week LXB titration period, 2-week stable-dose period, 8-day end-of treatment (EOT) period, and 2-week safety follow-up. The primary endpoint was change in Epworth Sleepiness Scale (ESS) score from BL to EOT. Key secondary endpoints for the narcolepsy cohort included change in polysomnography parameters: total shifts from deeper to lighter sleep stages, stage N3 sleep duration (minutes), and number of awakenings. Additional endpoints included Patient Global Impression of Change (PGIc)–overall narcolepsy disease. Primary and key secondary endpoints were controlled for multiplicity with sequential testing; other P-values are considered nominal. Results Fifty-five narcolepsy participants enrolled and were dosed with LXB (NT1, n=26; NT2, n=29); 13 transferred to another cohort and 34 were completers (NT1, n=16; NT2, n=18). Enrolled participants were mostly female (72.7%) and White (80.0%); mean (SD) age was 33.4 (12.9) years (NT1=34.6 [12.6]; NT2=32.4 [13.2]). For completers, least-squares mean (LSM) (SE) changes in ESS score were −7.7 (0.9), P&lt;.0001, overall; NT1=−6.5 (1.4), P&lt;.0001; NT2=−8.8 (1.3), P&lt;.0001. Compared with BL, sleep architecture at EOT showed fewer deeper to lighter sleep stage shifts (LSM [SE]: −13.1 [2.9], P&lt;.0001, overall; NT1=−13.9 [4.3], P=0.0029; NT2=−12.3 [4.1], P=0.0050); increased stage N3 sleep (minutes) (45.0 [8.8], P&lt;.0001, overall; NT1=49.8 [13.0], P=0.0006; NT2=40.8 [12.3], P=0.0023); and fewer awakenings (−3.2 [0.9], P=0.0015, overall; NT1=−4.1 [1.4], P=0.0056; NT2=−2.3 [1.3], P=0.0771). Most participants (93.3%, n=30; NT1, 86.7%, n=15; NT2, 100%, n=15) reported improvement (very much, much, minimally) in overall narcolepsy disease on PGIc. Treatment-emergent adverse events were consistent with the known safety profile of LXB. Conclusion Participants with NT1 and NT2 taking open-label LXB showed improvements in daytime sleepiness (decreased ESS scores) and reduced symptom burden (improved PGIc), which paralleled improvements in sleep architecture and disruption. Support (if any) Jazz Pharmaceuticals
0851 Effectiveness and Safety of Low-Sodium Oxybate in Idiopathic Hypersomnia Participants: Results from the DUET Study
Cairns, Alyssa , Schneider, Logan , Nichols, Deborah A. , Steininger, Teresa L. , Kirby, Melanie, Akerman, Sarah , Alexander, Jessica, Whalen, Marisa, Ruoff, Chad , Ruoff, Chad M , Plante, David T. , Fuller, Douglas, Kirby, M

SLEEP | May 19, 2025 | Volume: 48 | Issue: Supplement_1 | Pages: A369-A369

10.1093/sleep/zsaf090.0851 Get It

Abstract Introduction Introduction: Jazz DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) is a phase 4, prospective, multicenter, single-arm, multiple-cohort, open-label study (NCT05875974) evaluating the effectiveness of low-sodium oxybate (LXB, Xywav®) treatment on daytime and nighttime symptoms, including excessive daytime sleepiness (EDS) and polysomnography in participants with idiopathic hypersomnia (IH) or narcolepsy. Data presented are from the IH cohort. Methods DUET included screening (with 2-week washout for oxybate users), 8-day baseline (BL), 2- to 8-week LXB titration, 2-week stable-dose, 8-day end-of-treatment (EOT), and 2-week safety follow-up periods. The primary endpoint was change in Epworth Sleepiness Scale (ESS) score from BL to EOT. Secondary endpoints for the IH cohort included change in Idiopathic Hypersomnia Severity Scale (IHSS) total score, Patient Global Impression of Change (PGIc)−overall IH disease, PGIc−sleep inertia, and Patient Global Impression of Severity (PGIs)−sleep inertia. Exploratory endpoints included IHSS component scores (daytime functioning, sleep inertia). Primary and key secondary (IHSS total score) endpoints were controlled for multiplicity with sequential testing; all other P-values are considered nominal. Results Forty-six people with IH enrolled and were dosed with LXB; 40 were completers. Enrolled participants were mostly female (80.4%) and White (84.8%), with a mean (SD) age of 38.1 (11.8) years. For completers, BL mean (SD) ESS score and IHSS total score were 16.5 (2.7) and 32.9 (7.1), respectively; least-squares mean (LSM) (SE) changes from BL to EOT were −8.4 (0.7), P&lt;.0001 and −15.5 (1.5), P&lt;.0001. BL mean (SD) IHSS component scores for daytime functioning and sleep inertia were 17.7 (4.2) and 10.7 (3.1), respectively; LSM (SE) changes from BL to EOT were −8.8 (1.0), P&lt;.0001 and −4.8 (0.4), P&lt;.0001. At EOT, most participants reported improvement (very much, much, minimally) on PGIc−overall IH disease (94.6%) and PGIc−sleep inertia (81.1%). At BL, 25.0% of participants reported sleep inertia severity as very mild/mild/not present on PGIs–sleep inertia vs 59.0% at EOT. Treatment-emergent adverse events were consistent with the known safety profile of LXB. Conclusion People with IH taking open-label LXB showed improvements in EDS and sleep inertia (decreased ESS and IHSS component scores) and reported reduced multi-symptom burden (decreased IHSS total scores and improved PGIc/PGIs ratings). Support (if any) Jazz Pharmaceuticals
0785 Real-World Evaluation of Agent Longevity and Satisfaction in the Treatment of Residual Sleepiness in Patients with Obstructive Sleep Apnea
Seo, Heather, Auger, R. Robert , Roberts, Jessica, Seo, Heather, Morgenthaler, Timothy I.

SLEEP | May 19, 2025 | Volume: 48 | Issue: Supplement_1 | Pages: A340-A340

10.1093/sleep/zsaf090.0785 Get It

Abstract Introduction Residual excessive daytime sleepiness (RES) despite effective positive airway pressure (PAP) therapy for obstructive sleep apnea (OSA) is a clinical problem impacting quality of life and daily function. Wake-promoting agents (WPAs) such as modafinil, armodafinil, and solriamfetol have demonstrated efficacy in reducing RES in randomized controlled trials (RCTs); however, their effectiveness in typical sleep clinic populations remains underexplored. This study aimed to evaluate the efficacy, safety, and tolerability of these WPAs in real-world clinical settings. Methods This retrospective study analyzed electronic health records of 69 randomly selected, consenting patients with OSA who are on PAP therapy and a WPA at Mayo Clinic from 9/19/2019 to 9/19/2024. Researchers reviewed charts for treatment indication, duration, adverse effects, and response (categorized as “complete,” “partial,” or “poor”). Therapy longevity was assessed using initiation, refill, and discontinuation data. Outcomes were analyzed with paired t-tests, chi-square tests, and regression models. Results Of 69 patients, 45 were excluded because: WPA use before observation period (24), off-label WPA use (13), WPA use before CPAP (3), or other (5). Off-label reasons included neurologic-condition-related fatigue (6), depression augmentation (5), and autoimmune-condition-related fatigue (2). 24 patients were on WPAs for RES with PAP-treated OSA, including 3 with mixed indications, and were included in analyses. The median age was 65 (IQR 11). BMI at OSA diagnosis was 33.6 (IQR 6.4). Most subjects were male (75%). Initial Epworth Sleepiness Scale scores (ESS) were 12.3±1.1. ESS at the first WPA prescription were 15.4±0.9 (p=0.04). Most patients (83.3%) started on modafinil. Of the 4 patients who started on armodafinil or solriamfetol, chart review showed a previous trial of modafinil without benefit. After optimizing the initial WPA dosage, ESS improved by 3.3±1.4 points (p=0.04) to 12.1 ±1.2. Side effects limited WPA use in 20.8% of patients. Median first WPA duration was 141 days (IQR 53-280) with no significant difference between these WPAs (p=0.81). Conclusion Although WPAs have narrowly defined approved indications, they are often prescribed off-label for comorbidities. Unlike straightforward use in RCTs, real-life use is more complicated. Patients prescribed WPAs for the targeted approved indication show statistically significant benefits, although adverse effects limited their use for some. Support (if any)