A Multicentric, Open-label, Non-randomized Study to Evaluate the Pharmacokinetic, Safety and Tolerability of ITF2357 Given as an Oral Single 50 mg Dose in Participants With Chronic Hepatic Impairment Relative to Matched Participants With Normal Hepatic Function

This is a multicentric, open-label, non-randomized study to evaluate the pharmacokinetic, safety and tolerability of ITF2357 in participants with chronic hepatic impairment relative to matched participants with normal hepatic function.

Start Date01 Apr 2025

Sponsor / Collaborator

Italfarmaco SpA

[+1]

NCT06093672

/ RecruitingPhase 3

Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

Start Date26 Mar 2024

Sponsor / Collaborator

Italfarmaco SpA

NCT05933057

/ RecruitingPhase 3

Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to <18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months.
* Planned screening duration: approximately 4 weeks (±14 days)
* Planned treatment duration: 18 months (approximately 72 weeks)
* Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study)
* Total duration of study participation: up to 83 weeks (ie, 20-21 months)

Start Date19 Feb 2024

Sponsor / Collaborator

Italfarmaco SpA

[+1]

100 Clinical Results associated with Givinostat Hydrochloride

100 Translational Medicine associated with Givinostat Hydrochloride

100 Patents (Medical) associated with Givinostat Hydrochloride

140

Literatures (Medical) associated with Givinostat Hydrochloride

02 Jan 2025·Journal of biomolecular structure & dynamics

Exploring the potential of Andrographis paniculata for developing novel HDAC inhibitors: an in silico approach

Article

Author: Bandyopadhyay, Maumita ; Banerjee, Ritesh ; Nag, Anish ; Das, Debalina

Cancer is one of the dreaded diseases of the twentieth century, emerging the major global causes of human morbidity. Cancer research in the last 15 years has provided unprecedented information on the role of epigenetics in cancer initiation and progression. Histone deacetylases (HDACs) are recognized as important epigenetic markers in cancer, whose overexpression leads to increased metastasis and angiogenesis. In the current study, thirty-four (34) compounds from Andrographis paniculata were screened for the identification of potential candidate drugs, targeting three Class I HDACs (Histone deacetylases), namely HDAC1 (PDB id 5ICN), HDAC3 (PDB id 4A69) and HDAC8 (PDB id 5FCW) through computer-assisted drug discovery study. Results showed that some of the phytochemicals chosen for this study exhibited significant drug-like properties. In silico molecular docking study further revealed that out of 34 compounds, the flavonoid Andrographidine E had the highest binding affinities towards HDAC1 (-9.261 Kcal mol^-1) and 3 (-9.554 Kcal mol^-1) when compared with the control drug Givinostat (-8.789 and -9.448 Kcal mol^-1). The diterpenoid Andrographiside displayed the highest binding affinity (-9.588 Kcal mol^-1) to HDAC8 compared to Givinostat (-8.947 Kcal mol^-1). Statistical analysis using Principal Component Analysis tool revealed that all 34 phytocompounds could be clustered in four statistical groups. Most of them showed high or comparable inhibitory potentials towards HDAC target protein. Finally, the stability of top-ranked complexes (Andrographidine E-HDAC1 and HDAC3; Andrographiside-HDAC8) at the physiological condition was validated by Molecular Dynamic Simulation and MM-PBSA study.Communicated by Ramaswamy H. Sarma.

01 Jan 2025·JOURNAL OF PATHOLOGY

Macrophages producing chondroitin sulfate proteoglycan‐4 induce neuro‐cardiac junction impairment in Duchenne muscular dystrophy

Article

Author: Toccafondi, Mirco ; De Falco, Elena ; Bearzi, Claudia ; Rizzi, Roberto ; Ceraolo, Maria Grazia ; Fratini, Nicole ; De Paolis, Veronica ; Milan, Marika ; Zigiotto, Rebecca ; Landoni, Elisa ; Crosti, Maria Cristina ; Maiullari, Fabio ; Dotti, Gianpietro ; Chirivì, Maila ; Soluri, Andrea ; Brambilla, Francesca ; Cordiglieri, Chiara ; Bombaci, Mauro ; Seliktar, Dror ; Maiullari, Silvia ; Parisi, Chiara ; Lanzuolo, Chiara ; Calogero, Antonella ; Silvestre, Dario Di ; Mauri, Pierluigi ; Torrente, Yvan

Abstract:

Duchenne muscular dystrophy (DMD) is caused by the absence of the full form of the dystrophin protein, which is essential for maintaining the structural integrity of muscle cells, including those in the heart and respiratory system. Despite progress in understanding the molecular mechanisms associated with DMD, myocardial insufficiency persists as the primary cause of mortality, and existing therapeutic strategies remain limited. This study investigates the hypothesis that a dysregulation of the biological communication between infiltrating macrophages (MPs) and neurocardiac junctions exists in dystrophic cardiac tissue. In a mouse model of DMD (mdx), this phenomenon is influenced by the over‐release of chondroitin sulfate proteoglycan‐4 (CSPG4), a key inhibitor of nerve sprouting and a modulator of the neural function, by MPs infiltrating the cardiac tissue and associated with dilated cardiomyopathy, a hallmark of DMD. Givinostat, the histone deacetylase inhibitor under current development as a clinical treatment for DMD, is effective at both restoring a physiological microenvironment at the neuro‐cardiac junction and cardiac function in mdx mice in addition to a reduction in cardiac fibrosis, MP‐mediated inflammation, and tissue CSPG4 content. This study provides novel insight into the pathophysiology of DMD in the heart, identifying potential new biological targets. © 2024 The Author(s). The Journal of Pathology published by John Wiley & Sons Ltd on behalf of The Pathological Society of Great Britain and Ireland.

01 Dec 2024·Redox Biology

A promising drug repurposing approach for Alzheimer's treatment: Givinostat improves cognitive behavior and pathological features in APP/PS1 mice

Article

Author: Zhang, Sheng-Xiao ; Wang, Qi ; Wang, Zhao-Jun ; Gao, Qi-Chao ; Ji, Zhi-Lin ; Liu, Ge-Liang ; Yu, Qi ; Wu, Mei-Na ; He, Pei-Feng

Alzheimer's disease (AD) is the most common neurodegenerative disease, characterized by memory loss, speech and motor defects, personality changes, and psychological disorders. The exact cause of AD remains unclear. Current treatments focus on maintaining neurotransmitter levels or targeting β-amyloid (Aβ) protein, but these only alleviate symptoms and do not reverse the disease. Developing new drugs is time-consuming, costly, and has a high failure rate. Utilizing multi-omics for drug repositioning has emerged as a new strategy. Based on transcriptomic perturbation data of over 40,000 drugs in human cells from the LINCS-L1000 database, our study employed the Jaccard index and hypergeometric distribution test for reverse transcriptional feature matching analysis, identifying Givinostat as a potential treatment for AD. Our research found that Givinostat improved cognitive behavior and brain pathology in models and enhanced hippocampal synaptic plasticity. Transcriptome sequencing revealed increased expression of mitochondrial respiratory chain complex proteins in the brains of APP/PS1 mice after Givinostat treatment. Functionally, Givinostat restored mitochondrial membrane potential, reduced reactive oxygen species, and increased ATP content in Aβ-induced HT22 cells. Additionally, it improved mitochondrial morphology and quantity in the hippocampus of APP/PS1 mice and enhanced brain glucose metabolic activity. These effects are linked to Givinostat promoting mitochondrial biogenesis and improving mitochondrial function. In summary, Givinostat offers a promising new strategy for AD treatment by targeting mitochondrial dysfunction.

News (Medical) associated with Givinostat Hydrochloride

25 Jul 2024

·www.prnewswire.com

PANTHERx®Rare Partnering with ITF Therapeutics LLC for U.S. Distribution of DUVYZAT™

PITTSBURGH, Pa., July 25, 2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce it is partnering with ITF Therapeutics LLC for the U.S. distribution of DUVYZAT™ (givinostat), the first nonsteroidal drug approved to treat patients with all genetic variants of Duchenne muscular dystrophy (DMD) aged six years and older. DUVYZAT, a twice daily oral suspension, is a histone deacetylase (HDAC) inhibitor that reduces inflammation and loss of muscle observed in DMD. DMD is a rare and serious muscle disorder affecting approximately 1 in 5,000 male births worldwide and is caused by a variant in the DMD gene located on the X chromosome. This gene is responsible for regulating the production of dystrophin, which is a protein located in skeletal and cardiac muscle cells. Disease onset in DMD is typically observed between three and five years of age and progression is characterized by muscle wasting and weakness in the voluntary muscles of the body. The disease typically affects muscles closest to the trunk (e.g., upper legs, upper arms, shoulders, and pelvic muscles) first and then progresses outward toward the extremities. By late teenage years, many patients develop additional potentially life-threatening complications including cardiomyopathy and respiratory failure. "We are looking forward to serving the needs of people living with Duchenne muscular dystrophy and their families and caregivers by streamlining the process associated with getting rare medications from the people who create them to the people who need them most. We appreciate ITF Therapeutics' commitment to making a difference in the lives of patients with this disease," said Rob Snyder, CEO of PANTHERx® Rare Pharmacy. About PANTHERx Rare PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. About DUVYZAT™ (givinostat) DUVYZAT is a U.S. FDA approved therapy indicated for the treatment of patients six years of age and older with Duchenne muscular dystrophy (DMD) that was discovered through the research and development efforts of Italfarmaco in collaboration with Telethon and Duchenne Parent Project (Italy). DUVYZAT is a histone deacetylase (HDAC) inhibitor that modulates the deregulated activity of HDACs in the dystrophic muscle, which is a major consequence of the lack of dystrophin associated with DMD. Though the exact mechanism of action of DUVYZAT is unknown, it is believed to inhibit HDAC pathological overactivity to address the cascade of events leading to muscle damage, thereby counteracting the disease pathology and slowing down muscle deterioration. References: Duvyzat. Package Insert. ITF Therapeutics, LLC; 2024. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA; March 21, 2024; Duchenne Muscular Dystrophy. NORD; (accessed April 2, 2024) For more information, please contact [email protected] SOURCE PANTHERx Rare Pharmacy

Drug ApprovalOrphan Drug

27 Jun 2024

·www.biospace.com

KAZIA ANNOUNCES UPCOMING DIPG DATA PRESENTATIONS AT ISPNO AND PUBLICATION IN EUROPEAN JOURNAL OF CANCER

SYDNEY, June 27, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specialising in oncology, is pleased to announce the presentation of new data from its lead program, paxalisib, at the upcoming 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) June 29 – July 2, 2024, in Philadelphia, PA. Kazia concurrently announces publication of an article in European Journal of Cancer highlighting the need for evaluating mutation-specific, CNS penetrant, inhibitors to treat pediatric patients with Diffuse Midline Glioma (DMG). There will be three paxalisib-related presentations in total at ISPNO, including data from the Phase 2 PNOC DMG-ACT (DMG-Adaptive Combination Trial, PNOC022) study evaluating the efficacy and safety of paxalisib in combination with ONC201. As a follow up to data presented last year at the Society for NeuroOncology, 28th Annual Meeting, lead researchers will discuss survival, pharmacokinetics, and tumor biomarkers from 132 diffuse midline glioma (DMG) patients enrolled in the Phase 2 study. Highlights of the abstract include median overall survival of 13.2 months in Cohort 1 (newly diagnosed, enrolled pre-radiation n=33), 15.8 months in Cohort 2 (newly diagnosed, enrolled post-radiation n=69) and 8.8 months in Cohort 3 (relapsed patients, enrolled after progression n=30). The second presentation is based on novel preclinical data utilizing the addition of a novel HDAC inhibitor to the backbone therapy of paxalisib in DMG models. The third presentation will highlight preclinical data results of the combination therapy of paxalisib and gemcitabine for patients with relapsed/recurrent atypical teratoid/rhabdoid tumors AT/RT by Johns Hopkins University researchers. Based on these findings, the Pacific Pediatric Neuro-Oncology Consortium is planning to include this combination therapy in its next AT/RT international clinical trial. Summary of Abstracts ( ) Clinical Trials; July 2, 2024; 8:15am TRLS-14: PNOC022 report: a combination therapy trial using an adaptive platform design for patients with diffuse midline glioma at initial diagnosis, post-radiation therapy, or progression Cassie Kline, Andrea Franson, Anuradha Banerjee, Alyssa T Reddy, et al Poster Session I; June 30, 2024; 5pm ATRT-15 Combining the PI3K inhibitor paxalisib with nucleoside analog gemcitabine to improve survival of atypical teratoid/rhabdoid tumors Tyler Findlay, Kristen Malebranche, Anupa Geethadevi, Charles Eberhart, Jeffrey Rubens, Eric Raabe DIPG-21 Preclinical assessment of a multimodal treatment approach with Givinostat, Paxalisib, and radiotherapy for Diffuse Midline Glioma (DMG) Aimée du Chatinier, Michaël H Meel, Piotr Waranecki, Dennis S Metselaar, Esther The European Journal of Cancer publication titled Paediatric Strategy Forum for Medicinal Product Development of PI3-K, mTOR, AKT and GSK3β Inhibitors in Children and Adolescents with Cancer is the output from a two-day forum in April 2023 at Dana Farber Cancer Institute. Consisting of patient advocates, regulators, researchers and pediatric clinicians, the publication concludes "Evaluation of mutation-specific, CNS-penetrant PI3-K inhibitors in children with DMG should be prioritised and innovative regulatory approaches are needed in view of the rarity of the population." The paper can be accessed at the following website: (24)00801-3/fulltext About Kazia Therapeutics Limited Kazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2 study in glioblastoma reported early signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, has completed enrollment, with final data expected imminently. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data. Paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours in June 2022 and July 2022, respectively. For more information, please visit or follow us on Twitter @KaziaTx. Forward-Looking Statements This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the United States Securities and Exchange Commission (SEC), and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. This announcement was authorized for release by Dr John Friend, CEO. Company Codes: NASDAQ-SMALL:KZIA

Phase 2Orphan DrugRadiation TherapyASCO

24 Jun 2024

·www.prnewswire.com

Parent Project Muscular Dystrophy Hosts 30th Annual Conference in Orlando, Florida

Milestone event brings together record-breaking 1,300 plus families, physicians, researchers, caregivers, industry partners, and those living with Duchenne and Becker in person and virtually to connect, share information, and learn the latest progress in the fight for every future. ORLANDO, Fla., June 24, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its 30th Annual Conference in Orlando, Florida, June 27–29. Each year, the Duchenne and Becker community gathers to learn about the latest progress in the fight to end Duchenne and connect with leading experts. Now in its 30th year, PPMD's Annual Conference returns to Orlando, the location of the organization's very first Annual Conference in 1994, to engage more of the Duchenne and Becker community than ever before. PPMD expects a record-breaking 1,300 plus families, researchers, clinicians, and industry partners from all over the world for three days of informative and interactive sessions featuring the latest news in research, clinical trials, approved and emerging therapies, care initiatives, quality of life issues, and more. Before the Conference officially kicks off, PPMD will host a Becker Care Professional Meeting, June 26–27, aimed at initiating discussions within the healthcare professional community regarding the creation of consensus-based standards of care for Becker muscular dystrophy. Founded in 1994 by a group of parents and grandparents, and led by Founding President and CEO Pat Furlong, PPMD is the largest, most comprehensive nonprofit organization in the United States focused on ending Duchenne. Since its first meeting 30 years ago, PPMD has convened families with researchers, clinicians, industry partners, advocacy organizations, and most importantly, people with Duchenne and Becker and their families at the largest Duchenne-specific conference held in the U.S. Furlong is excited about this year's milestone Conference and looks forward to bringing the Duchenne and Becker community together: "From PPMD's first Annual Conference 30 years ago to our momentous return to Orlando this week, this community's unwavering commitment, passion, and resilience have been the driving force behind our fight for every future. We have faced challenges and celebrated victories and we have motivation to keep progressing. PPMD's 30th Annual Conference will be filled with a variety of interactive experiences to spark conversation, foster new and familiar relationships, learn from one another, and continue to drive change in the fight to end Duchenne." PPMD is grateful to all of our industry sponsors, whose support helps make the Annual Conference possible, especially our 30th Anniversary Star sponsor Sarepta Therapeutics, our Platinum sponsors Catalyst Pharmaceuticals and Pfizer, Inc., and our Gold sponsors Avidity Biosciences, Dyne Therapeutics, Edgewise Therapeutics, ITF Therapeutics, NS Pharma, Inc., Upsher-Smith Laboratories, and Wave Life Sciences. For sponsorship opportunities, please click here. PPMD's 30th Annual Conference agenda will feature stories from multiple keynote speakers, and interactive discussion panels addressing comprehensive, multi-dimensional approaches to care for Duchenne and Becker. Unique topics to this year's conference include: Approved therapies in Duchenne, including presentations on the recently expanded label for ELEVIDYS and newly-approved therapies AGAMREE and DUVYZAT, as well as a clinician panel discussion on the eight currently approved therapies. Becker-specific breakout sessions, including discussions on neuromuscular care, cardiology, genetic counseling, and physical therapy, as well as industry updates about emerging therapies for Becker. What we have learned over time about outcome measures, including Functional Reach, Stride Velocity, Dynamic Visual Acuity (DVA), and Northstar and Performance of Upper Limb (PUL). Cardiac, neuromuscular, and adolescent care for carriers, as well as physical therapy implications and research in progress in the field of Duchenne carriers. Unique experiences and perspectives on life, living, and hope, as well as daily life care such as sleep and behavior; decision making around steroids, weight, and nutrition; pain; exercise; social connection; and more. This year's Conference will also feature dedicated updates from more than 20 partners currently in clinical trials, with panels including: Strategies to restore dystrophin The current and evolving landscape of gene therapy Approaches to alter disease progression PPMD's role in moving the needle in clinical research A variety of breakout sessions and social gatherings will also be offered during PPMD's 30th Annual Conference, encouraging attendees to create their own, unique experiences, in-person or virtually, by participating in: Special programming for Newly Diagnosed Families A dedicated track for Teens and Adults with Duchenne/Becker Expanded Sibs Track sessions, offering a safe place for siblings to be together, talk, and open up about topics that they face Breakout sessions according to age, covering multiple aspects of care One-on-one private appointments with Physical Therapists, Genetic Counselors, and behavior, speech, and language experts to help provide guidance on every family's unique needs Small group Lighthouse Mini-Sessions focused on self-care, effective communication, asking for help, and staying connected and finding belonging A Build Your Own Care Binder station for families to create personalized binders filled with important information regarding Duchenne/Becker care to help navigate routine care and emergencies A robust Resource Fair enabling resource providers and the community to connect about practical services, research and more A meet and greet for diverse families in Duchenne to share their unique experiences and foster connection Social activities for moms, dads, grandparents, siblings, and, of course, tweens, teens, and adults with Duchenne and Becker Fun and interactive sessions for kids Ahead of the Conference, all registered attendees were given access to PPMD's online Conference Hub & App to stream sessions and personalize their agenda and Conference experience. This year's Hub & App offers an enhanced experience for both in-person and virtual attendees, allowing for engagement with exhibitors and increased interaction during live sessions. AI-powered live translation will also be available for main sessions in dozens of languages. It is not too late to attend this year's meeting, in-person or virtually. To register for PPMD's 30th Annual Conference, click here. To learn more about PPMD, please visit our website. SOURCE Parent Project Muscular Dystrophy (PPMD)

100 Deals associated with Givinostat Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Givinostat Hydrochloride	-	DB12645

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	US	Italfarmaco SpA	21 Mar 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 3	US	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	AT	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	BG	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	HR	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	FR	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	DE	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	HU	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	IE	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	IL	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	IT	Italfarmaco SpA	26 Mar 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03238235 (CTgov)	Phase 2	Becker Muscular Dystrophy	51	Givinostat (Givinostat)	yrcmisnmuh(kyjlvdrwzo) = vfouaiotzu lshcjzwvuj (ekykhujknj, hikuzkopnq - yipmuvqgnn) View more	-	18 Nov 2024
				Placebo (Placebo)	yrcmisnmuh(kyjlvdrwzo) = ifdudrotuz lshcjzwvuj (ekykhujknj, atikjknkjo - cymstqipyx) View more
NCT02851797 (EPIDYS, Literature \| Pubmed \| Lancet Neurol) Manual	Phase 3	Muscular Dystrophy, Duchenne	120	givinostat	cozjysergx(nbrkxfluiz) = bpjxqhuwng oadseggktz (vabppsabgz, 1.17 - 1.37)	Positive	01 Apr 2024
				Placebo	cozjysergx(nbrkxfluiz) = ouokiruiqd oadseggktz (vabppsabgz, 1.17 - 1.37)
NCT02851797 (FDA_CDER) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	DUVYZAT	aherindtra(ixsesifmqw) = cgyznvptlm blvxfbjtmc (iyonmjgziy )	Positive	21 Mar 2024
NCT05845567 (CTgov)	Phase 1	Medication interactions	20	Givinostat (Givinostat Alone (Day 1))	plaohmlfwg(riowcknodr) = rbpacilqlt orsakejawj (qcbvbyptrm, gbauqcioes - zjitdijerh) View more	-	09 Feb 2024
				Givinostat+Clarithromycin (Givinostat Co-Administered With Clarithromycin (Day 8))	plaohmlfwg(riowcknodr) = lrcsunkysz orsakejawj (qcbvbyptrm, ahpgdkvwyp - romkrcmbld) View more
NCT00792740 (CD, CTgov)	Phase 1/2	Crohn Disease	51	Placebo (Placebo)	humwpkdsdo(qbrcfenopz) = pbkvepocpt aedhtcnana (hjlubjgmkg, edhppdeyez - ugtygnxhvb) View more	-	19 Jul 2023
				ITF2357 (ITF2357)	humwpkdsdo(qbrcfenopz) = kgflegmjue aedhtcnana (hjlubjgmkg, xsejpweplt - flqoyvvpco) View more
NCT02851797 (Epidys, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne fat fraction of the vastus lateralis (VL)	179	Givinostat	ewcizzthkp(tkmvsoiaft) = cwfvphwynq rbornqoidv (hbobswrfsb )	Positive	19 Mar 2023
				Placebo	ewcizzthkp(tkmvsoiaft) = zmyxjnvblh rbornqoidv (hbobswrfsb )
NCT02851797 (Epidys Study, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat	lwwijbwtvp(usazwpslea) = reversible and manageable wlajweduhl (tjbajeukwm ) View more	Positive	19 Mar 2023
NCT02851797 (Study 1 \| Epidys Study \| EPIDYS, CTgov)	Phase 3	Muscular Dystrophy, Duchenne	179	givinostat (Givinostat)	snoiilcbig(mqualidttv) = rjnnqpdkyc ceegpektfw (ysonzrmovo, jabpuqetpy - lvpqfphlpq) View more	-	02 Feb 2023
				placebo (Placebo)	snoiilcbig(mqualidttv) = uzlnwtvkaj ceegpektfw (ysonzrmovo, enoemelbhw - zoalqbtjfn) View more
NCT02851797 (EPIDYS, PipelineReview) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat	pvnwvyzbxl(ikdbkaxnub): difference = 1.78, P-Value = 0.0345 View more	Positive	26 Jun 2022
				Placebo
NCT03238235 (BusinessWire) Manual	Phase 2	Becker Muscular Dystrophy	47	Givinostat	bbkpvryfrj(oieugstqfu) = No serious Adverse Events (AE) occurred during the course of the trial. vxyeoipwxv (pdjlnlwdex ) View more	Positive	26 Jun 2021
				Placebo

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