Latest news with #clinicaltrial
Bloomberg
13 hours ago
- Business
- Bloomberg
Intellia Plunges in Latest Example of Gene Therapy Troubles
Intellia Therapeutics Inc. fell Thursday after disclosing a patient experienced a severe side effect from its gene-editing treatment for a heart condition, the latest safety issue tied to DNA-targeting therapy. The patient, one of more than 200 who have received the Intellia treatment as part of a clinical trial, had elevated liver enzymes, 'which appear to be resolving without hospitalization or medical intervention,' the company said in a filing.
Medscape
14 hours ago
- Business
- Medscape
Telemonitoring Boosts Glycaemic Control in T2D
Compared with standard-of-care practices according to the European Association for the Study of Diabetes (EASD) and American Diabetes Association (ADA) consensus report, telemonitoring with continuous glucose monitoring (CGM) and connected insulin pens improved glycaemic control in patients with type 2 diabetes (T2D), showing a 13.6% increase in the time in range for CGM. METHODOLOGY: Researchers in Denmark conducted a clinical trial to assess the effectiveness and safety of telemonitoring among patients with T2D treated with insulin. They included 331 patients with a diagnosis of T2D for 12 months or more (mean age, 61.3 years; 61.6% men) and randomly assigned them to the telemonitoring or standard-of-care group over a 3-month period. The telemonitoring group used a continuous glucose monitor, a connected insulin pen, an activity tracker, and related smartphone applications; the clinical staff monitored their data and provided telephonic support at least three times during the trial and were capable to give advice on adjusting insulin doses after consulting a physician. The standard-of-care group received care according to the EASD-ADA consensus report; they used a continuous glucose monitor during the first and last 20 days of the trial and a connected insulin pen, with participants and the clinical staff having no access to data during the trial. The primary outcome measure was a change from baseline in the time in range for CGM (3.9-10.0 mmol/L), and secondary endpoints included changes from baseline in the A1c level, total daily insulin dose, and time both below and above range for CGM at 3 months. TAKEAWAY: Compared with the standard-of-care group, the telemonitoring group demonstrated significantly superior glycaemic control with an estimated treatment difference of 13.6% in the time in range for CGM ( P = .004). = .004). Compared with standard of care, telemonitoring significantly reduced the A1c level by 7.6 mmol/mol ( P = .001). = .001). The time above range for CGM decreased by 13.1% ( P = .004) in the telemonitoring vs standard-of-care groups, whereas no significant differences in the time below range for CGM and total daily insulin dose were found between groups. = .004) in the telemonitoring vs standard-of-care groups, whereas no significant differences in the time below range for CGM and total daily insulin dose were found between groups. Even though no episodes of severe hypoglycaemia were reported in either group, two serious adverse events related to the trial occurred in the telemonitoring group, where participants injected excessive amounts of fast-acting insulin. IN PRACTICE: "Telemonitoring was superior to standard of care in T2D as it effectively and safely improved glycemic control. Future studies should explore the potential of intelligent clinical decision support in telemonitoring of people with T2D to exploit the large amount of data that diabetes technology will generate," the authors wrote. SOURCE: This study was led by Stine Hangaard, Steno Diabetes Center North Denmark, Aalborg University Hospital, Aalborg, Denmark. It was published online on May 24, 2025, in European Journal of Internal Medicine . LIMITATIONS: The telemonitoring group's access to CGM data with the standard-of-care group remaining blinded to them may have underestimated the treatment effect due to immediate behaviour changes. As this was a telemedicine trial, it was not possible to identify specific components of the intervention that contributed to the observed effects. Moreover, the study faced selection bias, as patients who were more comfortable using technology were more likely to participate in the trial. DISCLOSURES: This study received support from the ADAPT-T2D consortium, The Innovation Fund Denmark, and Novo Nordisk A/S. Six authors reported being employees of and having shares in Novo Nordisk A/S. Some others reported receiving consultant fees, unrestricted grants, and travel grants from various pharmaceutical companies.
Medscape
2 days ago
- Business
- Medscape
Deupirfenidone Shows Promise Against IPF-Linked Lung Decline
SAN FRANCISCO — A molecular variant of an older, hard-to-tolerate antifibrotic agent showed promise for reducing decline in forced vital capacity (FVC) over 26 weeks among patients with idiopathic pulmonary fibrosis (IPF). These findings come from the phase 2B ELEVATE IPF trial, results of which were reported at the American Thoracic Society (ATS) 2025 International Conference here. The trial compared the investigational agent deupirfenidone to placebo for protection against FVC decline in 257 patients with IPF. The deupirfenidone molecule is nearly identical in conformation to pirfenidone, except for the substitution of hydrogen in pirfenidone for deuterium, or 'heavy hydrogen,' in deupirfenidone at the site of metabolism. This process, known as 'deuteration,' is designed to make the modified compound less toxic than pirfenidone, said lead investigator Toby Maher, MD, PhD, from the Keck School of Medicine at the University of Southern California, Los Angeles. Although the primary endpoint of the study was decline in FVC over 26 weeks, 'when we've looked at the open-label extension of patients continuing on the high dose, we've seen a sustained stabilization in FVC over 52 weeks, again suggesting a durable effect of treatment,' he said in an oral abstract session. Multinational Trial In the ELEVATE IPF trial, 257 patients with IPF from 87 sites in 14 countries were enrolled and randomly assigned to one of four treatment arms: 550 mg deupirfenidone thrice per day, 825 mg deupirfenidone thrice per day, 801 mg pirfenidone thrice per day, or placebo thrice per day. The primary Bayesian analysis showed that the mean adjusted change in FVC from baseline to 26 weeks was −48.4 ml for all patients randomized to deupirfenidone compared with −110.7 ml for patients assigned to placebo (posterior probability vs placebo, 98.5%). The respective adjusted mean changes from baseline in FVC% predicted were −48.4% and −1.1% (posterior probability, 99.6%). In a Frequentist inference analysis, compared with placebo the higher dose of deupirfenidone but not the lower dose was associated with significantly less decline in FVC and FVC% predicted. The adjusted mean changes over baseline were −21.5 ml for the 825 mg deupirfenidone group vs −112.5 ml for the placebo group ( P = .02), and the respective changes in FVC% predicted were −21.5% and −3.43% ( P = .01). Both pirfenidone and deupirfenidone 825 mg (but not 550 mg) significantly delayed time to progression compared with placebo. The respective hazard ratios were 0.50 ( P = .0076) and 0.439 ( P = .0033). In the placebo, pirfenidone, deupirfenidone 550 mg and 825 mg arms, respectively, treatment-emergent adverse events commonly associated with antifibrotics were nausea (7.7%, 27.0%, 16.9%, 20.3%), dyspepsia (3.1%, 22.2%, 12.3%, 14.1%), diarrhea (9.2%, 11.1%, 10.8%, 7.8%), abdominal pain (4.6%, 7.9%, 6.2%, 14.1%), photosensitivity reaction (0%, 7.9%, 6.2%, 7.8%), decreased appetite (7.7%, 14.3%, 18.5%, 20.3%), fatigue (1.5%, 11.1%, 7.7%, 9.4%), dizziness (3.1%, 7.9%, 9.2%, 12.5%), and headache (4.6%, 12.7%, 7.7%, 3.1%). GI Signal In the question and answer, session co-moderator Rachel Knipe, MD, from Massachusetts General Hospital in Boston, commented that there appeared to be a small signal for increased abdominal pain with deupirfenidone but less so for diarrhea and other gastrointestinal symptoms. She asked Maher whether investigators had also looked at liver function tests. 'The liver function tests didn't flag up as an adverse event that differed across groups or reached the threshold for inclusion in the table,' Maher said. Maher acknowledged that there are gastrointestinal events associated with the drug, but added that it's difficult to distinguish specific events from one another. 'The challenge with clinical trials in interpreting adverse events is that we are beholden on the individual investigators to label the side effects,' he said, noting that one center's dyspepsia, may be another center's loss of appetite or abdominal pain. In an interview, Medscape Medical News asked Knipe, whose laboratory at Mass General has a special focus on PF, for her impression of deupirfenidone. 'I think the data looks pretty good; it's exciting, and it looks like it has good effect on lung function,' she said, adding that the adverse event profile appears to be more favorable than that of pirfenidone. The study was funded by PureTech Health. Maher reported receiving grants and funding and serving as a consultant for the company and others. Knipe had no disclosures relevant to the study.
Medscape
2 days ago
- General
- Medscape
Eptinezumab for Episodic Cluster Headache Prevention
Compared with placebo, eptinezumab failed to significantly reduce the number of episodic cluster headache (ECH) attacks but showed higher responder rates and improved quality-of-life measures. METHODOLOGY: Eptinezumab, an anti–calcitonin gene-related peptide monoclonal antibody, was evaluated for the treatment of ECH. This phase 3, double-blind, placebo-controlled trial (ALLEVIATE) was conducted across 64 sites in 18 countries from December 2020 to October 2023 and screened 628 adults with ECH. A total of 231 adults (aged 18-75 years; mean age, 44 years; 78% men) meeting the criteria (a history of ECH for 1 or more years and seven or more CH attacks during screening) were randomly assigned to receive either eptinezumab 400 mg (n = 113) or placebo (n = 118) via intravenous infusion. After the 4-week placebo-controlled phase, participants who received placebo transitioned to delayed-start active treatment for an additional 4 weeks, followed by 12 weeks of observation and an 8-week safety follow-up. The primary outcome was the change from baseline in the number of weekly attacks (weeks 1-2), assessed via a daily electronic diary; secondary outcomes included responder rates of 50% or greater/75% or greater; pain severity; change in disease status, assessed using the Patient Global Impression of Change (PGIC); quality of life, assessed using the Sleep Impact Scale; participant well-being, assessed using the EQ-5D-5L; and self-rated productivity, assessed using the Work Productivity and Activity Impairment. TAKEAWAY: No significant reduction in the number of weekly attacks were observed with eptinezumab vs placebo during weeks 1 and 2 (least-square mean difference, 0.7; P = .50). = .50). A higher proportion of eptinezumab-treated participants achieved 50% or greater response than placebo-treated participants over week 2 (50.9% vs 37.3%; odds ratio [OR], 1.77; P = .04), week 3 (62.5% vs 43.8%; OR, 2.26; P = .004), and week 4 (66.7% vs 50.5%; OR, 2.14; P = .009). = .04), week 3 (62.5% vs 43.8%; OR, 2.26; = .004), and week 4 (66.7% vs 50.5%; OR, 2.14; = .009). Compared with placebo, eptinezumab demonstrated improvements for 75% or greater responder rates by week 4 (35.5% vs 52.0%; OR, 1.98; P = .02). = .02). Numerically greater improvements were observed with eptinezumab than with placebo in terms of PGIC scores, EQ-5D-5L visual analog scale scores (mean difference, 7.8 points; P = .02), and sleep/activity metrics. = .02), and sleep/activity metrics. Treatment-emergent adverse events were similar between eptinezumab and placebo (25.0% and 26.5%, respectively), confirming tolerability. IN PRACTICE: "Among adults with episodic cluster headache, eptinezumab did not significantly reduce the number of attacks vs placebo, although it was associated with numerically higher responder rates and improvements in average daily pain and patient-reported outcomes. Eptinezumab was generally well tolerated," the authors of the study wrote. SOURCE: This study was led by Rigmor H. Jensen, Danish Headache Center, Department of Neurology, Rigshospitalet-Glostrup, University of Copenhagen, Copenhagen, Denmark. It was published online on May 19 in JAMA Neurology . LIMITATIONS: T he study's generalisability may be limited due to the predominantly male (78%) and European population. Early termination due to futility reduced the sample size, although the cohort remained sufficient for the primary analysis. The 4-week placebo-controlled period was too brief to assess the long-term efficacy, unlike the 12-week regimens used for migraine prevention. DISCLOSURES: This trial was sponsored and funded by H. Lundbeck A/S, including medical writing support. Jensen reported receiving grants from Københavns Universitet, Lundbeck Pharma, Novo Nordisk, and Lundbeck Foundation paid to the institution during the conduct of the study. Additional disclosures are noted in the original article.
Associated Press
2 days ago
- Business
- Associated Press
SeaStar Medical Announces CMS Coverage for Medicare and Medicaid Eligible Patients with Cardiorenal Syndrome Awaiting LVAD in Investigational Trial of SCD Therapy
Coverage marks SeaStar Medical's second award by CMS for reimbursement of medical expenses for Medicare and Medicaid patients in a clinical trial Stands out as rare award with less than 100 clinical trials covered annually DENVER, May 28, 2025 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a commercial-stage healthcare company focused on transforming treatments for critically ill patients facing organ failure and potential loss of life, announced today that the U.S. Centers for Medicare & Medicaid Services (CMS) has agreed to pay for certain expenses incurred by medical centers treating patients covered by Medicare or Medicaid who are enrolled in the NEUTRALIZE-CRS investigational clinical trial. This follows the company's first award for reimbursement by the CMS that was granted in July 2024 for qualified patients treated in the ongoing NEUTRALIZE-AKI pivotal clinical trial. 'To receive CMS coverage of certain expenses for patients in a clinical trial is rare, with less than 100 per year, and we believe reflects the life-saving potential of our technology,' said Eric Schlorff, SeaStar Medical CEO. 'We fulfilled multiple criteria set forth by the CMS for this award, including ten study criteria elements that included assessment of how we could improve health outcomes, how generalizable the Selective Cytopheretic Device (SCD) therapy would be to the Medicare population, and how the study results would not duplicate existing knowledge.' Mr. Schlorff continued, 'As we initiate our pre-commercialization efforts for our SCD therapy in patients with Acute Kidney Injury (AKI), we recognize that CMS coverage in the commercial setting will be a key element to bringing our potential organ-sparing and life-saving therapies to more patients. We have already engaged a third-party reimbursement policy expert to analyze the feasibility of obtaining reimbursement coverage upon a potential FDA approval for our SCD therapy in adult patients with AKI. Based on the results of the analysis, the high unmet need, and Healthcare Economics and Outcomes Research (HEOR) data supporting reduced healthcare costs, we are building a compelling case that should enable CMS and private payers to understand the value of the SCD therapy in adult patients with AKI.' The NEUTRALIZE-AKI pivotal clinical trial and NEUTRALIZE-CRS investigational clinical trial are evaluating the ability of SeaStar Medical's SCD therapy to neutralize destructive hyperinflammation to improve health outcomes. The SCD therapy is designed as a disease-modifying device that neutralizes over-active immune cells and stops the cytokine storm that yields destructive hyperinflammation and creates a cascade of events that wreak havoc in the patient's body. The NEUTRALIZE-AKI pivotal trial was granted CMS coverage in July 2024 for qualified patients. The trial is evaluating the safety and efficacy of the SCD therapy in 200 adults with AKI in the ICU receiving CRRT. It is currently 50% enrolled, with full enrollment anticipated near the end of 2025. The trial's primary endpoint is a composite of 90-day mortality or dialysis dependency of patients treated with the SCD therapy in addition to CRRT as the standard of care, compared with the control group receiving only CRRT standard of care. The FDA has granted Breakthrough Device Designation for the SCD therapy in adult patients with AKI and CRRT. The NEUTRALIZE-CRS trial is designed to evaluate the safety and initial efficacy of the SCD therapy in reducing destructive hyperinflammation in adult patients with acute heart failure with worsening renal function due to cardiorenal syndrome or severe right ventricular failure awaiting a left ventricular assist device (LVAD) implantation. The trial is expected to enroll 20 patients at up to five clinical sites and will be funded by a previously announced $ 3.6 million National Institutes of Health (NIH) grant awarded to Innovative BioTherapies (IBT), which is led by SCD inventor H. David Humes, MD, Professor, Division of Nephrology, Internal Medicine, University of Michigan and SeaStar Medical Scientific Advisor. Dr. Humes will serve as lead investigator for the study and SeaStar Medical will act as clinical research organization (CRO). The FDA has granted Breakthrough Device Designation for the SCD in cardiorenal syndrome awaiting LVAD implantation. About SeaStar Medical SeaStar Medical is a commercial-stage healthcare company focused on transforming treatments for critically ill patients facing organ failure and potential loss of life. SeaStar's first commercial product, QUELIMMUNE (SCD-PED), was approved in 2024 by the U.S. Food and Drug Administration (FDA). It is the only FDA approved product for the ultra-rare condition of life-threatening acute kidney injury (AKI) due to sepsis or a septic condition in critically ill pediatric patients. SeaStar's Selective Cytopheretic Device (SCD) therapy has been awarded Breakthrough Device Designation for six therapeutic indications by the FDA, enabling the potential for a speedier pathway to approval and preferable reimbursement dynamics at commercial launch. The company is currently conducting a pivotal trial of its SCD therapy in adult patients with AKI requiring continuous renal replacement therapy (CRRT), a life-threatening condition with no effective treatment options that impacts over 200,000 adults in the U.S. annually. Forward-Looking Statements This press release contains certain forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1955. These forward-looking statements include, without limitation, SeaStar Medical's expectations with respect to anticipated patient benefits and cost savings from our products; the expected regulatory approval process and timeline for our products; and the ability of SeaStar Medical to meet the expected timeline. Words such as 'believe,' 'project,' 'expect,' 'anticipate,' 'estimate,' 'intend,' 'strategy,' 'future,' 'opportunity,' 'plan,' 'may,' 'should,' 'will,' 'would,' 'will be,' 'will continue,' 'will likely result,' and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to significant risks and uncertainties that could cause the actual results to differ materially from the expected results. Most of these factors are outside SeaStar Medical's control and are difficult to predict. Factors that may cause actual future events to differ materially from the expected results include, but are not limited to: (i) the risk that SeaStar Medical may not be able to obtain regulatory approval of its SCD product candidates; (ii) the risk that SeaStar Medical may not be able to raise sufficient capital to fund its operations, including current or future clinical trials; (iii) the risk that SeaStar Medical and its current and future collaborators are unable to successfully develop and commercialize its products or services, or experience significant delays in doing so, including failure to achieve approval of its products by applicable federal and state regulators, (iv) the risk that SeaStar Medical may never achieve or sustain profitability; (v) the risk that SeaStar Medical may not be able to secure additional financing on acceptable terms; (vi) the risk that third-party suppliers and manufacturers are not able to fully and timely meet their obligations, (vii) the risk of product liability or regulatory lawsuits or proceedings relating to SeaStar Medical's products and services, (viii) the risk that SeaStar Medical is unable to secure or protect its intellectual property, and (ix) other risks and uncertainties indicated from time to time in SeaStar Medical's Annual Report on Form 10-K, including those under the 'Risk Factors' section therein and in SeaStar Medical's other filings with the SEC. The foregoing list of factors is not exhaustive. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and SeaStar Medical assumes no obligation and do not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. For more information visit or visit us on LinkedIn or X. Contact: SeaStar Investor Relations: [email protected]
