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Clinical Trial News

Intravenous Rehydration Shows Safety in Severely Malnourished Children, Challenging WHO Guidelines

  • A randomized controlled trial involving 292 children across four African countries found that intravenous rehydration was as safe as oral rehydration for children with severe acute malnutrition and gastroenteritis.
  • The study revealed no cases of heart failure or fluid overload with IV treatment, contradicting long-held concerns about cardiac complications in malnourished children.
  • Children receiving standard oral rehydration experienced more complications, including increased need for nasogastric tubes, vomiting episodes, and emergency IV fluids for shock.
  • The findings could prompt a review of WHO guidelines that currently distinguish between rehydration strategies for malnourished and non-malnourished children.

FDA Delays Sebetralstat Approval Decision for Hereditary Angioedema Treatment Due to Resource Constraints

Drug ApprovalRare Disease
  • The FDA will not meet the June 17, 2025 PDUFA goal date for KalVista's sebetralstat NDA due to heavy workload and limited resources, with a decision expected within four weeks.
  • Sebetralstat represents a potential breakthrough as the first oral on-demand treatment for hereditary angioedema attacks in patients aged 12 and older.
  • The FDA has not requested additional data or raised safety concerns, with only labeling finalization remaining under review according to KalVista.
  • The delay affects patients with hereditary angioedema who currently rely on intravenous or subcutaneous treatments for managing painful and potentially life-threatening attacks.

Olutasidenib Maintenance Therapy Demonstrates Sustained Remission in IDH1-Mutated AML Patients

  • Olutasidenib maintenance therapy achieved an 83% relapse-free survival rate at 4 months and 71% at 12 months in IDH1-mutated AML patients who achieved complete remission following induction therapy.
  • The phase 2 trial enrolled 18 patients with a median treatment duration of 19.6 months, demonstrating clinically meaningful activity as a switch maintenance strategy for patients with persistent minimal residual disease.
  • Overall survival rates remained high at 89% for both 12 and 24 months, with a manageable safety profile and no treatment discontinuations due to adverse events.
  • Two patients with prior venetoclax exposure converted from incomplete remission to complete remission during olutasidenib treatment, supporting its therapeutic potential in this patient population.

Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation

NCT02719574CompletedPhase 1
Forma Therapeutics, Inc.
Posted 4/30/2016

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

NCT03173248Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 6/26/2017

Thermo Fisher Scientific Explores $4 Billion Diagnostics Unit Sale to Streamline Portfolio

  • Thermo Fisher Scientific is reportedly planning to divest portions of its diagnostics business valued at approximately $4 billion to offload lower-growth assets.
  • The company has enlisted advisers to explore private equity interest in parts of its diagnostics unit, including the microbiology division that produces infectious disease testing equipment.
  • This strategic move comes amid healthcare sector volatility and follows the company's recent $4.1 billion acquisition of Solventum's purification and filtration business.
  • The auction process does not guarantee a sale, as Thermo Fisher may ultimately decide to retain these assets depending on market conditions and strategic considerations.

NeuroEM Therapeutics Secures $1.33 Million in Series A Funding to Advance Drug-Free Alzheimer's Treatment

  • NeuroEM Therapeutics has closed initial rounds totaling $1.33 million of a $5 million Series A equity raise led by BlueLake.vc to commercialize its Transcranial Electromagnetic Treatment (TEMT-RF) technology.
  • The company's innovative approach uses radio frequencies delivered through a lightweight cap worn twice daily to target cognitive decline and restore brain function in Alzheimer's patients.
  • NeuroEM received the first FDA Breakthrough Device status for treating Alzheimer's disease and has demonstrated safety and cognitive enhancement in early clinical studies.
  • The funding will support continued progress toward commercialization of the drug-free treatment, which aims to prevent and treat cognitive decline caused by aging and neurodegenerative diseases.

Ponatinib Shows Superior Survival Outcomes in Ph+ ALL Patients with Persistent MRD After Induction

Drug Approval
  • Post hoc analysis of the PhALLCON trial demonstrates that ponatinib achieved higher rates of deep molecular responses compared to imatinib in newly diagnosed Ph+ ALL patients who remained MRD-positive after induction therapy.
  • Patients with persistent MRD who continued ponatinib beyond cycle 3 showed significantly improved event-free survival with a median not reached versus 24.8 months for imatinib (HR 0.198, P = 0.0004).
  • The analysis supports continuing ponatinib treatment in Ph+ ALL patients who fail to achieve MRD negativity after initial induction, though researchers caution about the small patient numbers and potential selection bias.

A Study of Ponatinib Versus Imatinib in Adults With Acute Lymphoblastic Leukemia

NCT03589326Active, Not RecruitingPhase 3
Takeda
Posted 10/4/2018

Nectero Medical Secures CPT Codes for Investigational AAA Treatment as Phase 2/3 Trial Shows Promise

  • Nectero Medical received two Category III CPT codes from the American Medical Association for its investigational EAST system, facilitating future billing and reimbursement for treating small- to medium-sized abdominal aortic aneurysms.
  • The EAST system delivers pentagalloylglucose directly into aneurysmal walls via a dual-balloon catheter, designed as a one-time endovascular treatment to slow or stabilize AAA growth.
  • One-year follow-up data from the stAAAble trial showed 91% of patients experienced aneurysm growth rates below expected levels compared to historical controls.
  • The approval represents a significant milestone for addressing an unmet medical need, as no proven therapeutic interventions currently exist for small- to medium-sized AAAs.

Cyclophosphamide Plus Cyclosporin Emerges as New Standard of Care for GVHD Prevention in Blood Cancer Transplants

  • The phase 3 BM12 CAST trial demonstrated that cyclophosphamide plus cyclosporin significantly improved graft-versus-host disease-free relapse-free survival compared to standard cyclosporin/methotrexate prophylaxis in blood cancer patients.
  • Median GVHD-free relapse-free survival reached 26.2 months with the new combination versus 6.4 months with standard therapy, representing a 58% reduction in risk.
  • At three years, 49.1% of patients receiving cyclophosphamide/cyclosporin remained free from GVHD compared to only 14.2% with standard treatment.
  • The study enrolled 134 patients with acute leukemia or myelodysplastic syndrome across 10 transplant centers in Australia and New Zealand.

Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

NCT05316701Active, Not RecruitingPhase 3
Orca Biosystems, Inc.
Posted 6/21/2022

Precision-T: A Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

NCT04013685Active, Not RecruitingPhase 1
Orca Biosystems, Inc.
Posted 11/21/2019

Combination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia

NCT02003222Active, Not RecruitingPhase 3
National Cancer Institute (NCI)
Posted 5/19/2014

Innocan Pharma Advances Liposomal CBD Platform for Chronic Pain as Peer-Reviewed Study Highlights Non-Opioid Alternative

  • A peer-reviewed narrative review published in Cureus journal highlights the therapeutic potential of long-acting synthetic cannabidiol (CBD) for chronic pain management as a non-opioid alternative.
  • Innocan Pharma's proprietary LPT-CBD platform delivers sustained CBD release for up to four weeks through injectable liposomal formulation, supported by animal studies showing prolonged pain relief.
  • The company has initiated regulatory submissions to advance LPT-CBD into human clinical trials, addressing the urgent need for safer pain therapies amid a global opioid crisis causing over 100,000 deaths annually.
  • Leading pain specialists from Johns Hopkins University and NYU School of Medicine co-authored the review, emphasizing the potential paradigm shift away from addictive opioid medications.

EMA Recommends First EU Vaccine Against Swine Dysentery, Targeting Disease with 30-50% Mortality Rate

Drug Approval
  • The European Medicines Agency has issued a positive opinion for Biobhyo, the first vaccine authorized in the EU to protect pigs against swine dysentery disease.
  • Swine dysentery affects up to 90% of pigs in infected herds with a mortality rate of 30-50%, caused by the bacterium Brachyspira hyodysenteriae.
  • Clinical studies demonstrated vaccine efficacy when administered at five and eight weeks of age, showing reduced incidence of dysenteric diarrhea in commercial farm settings.
  • The Committee for Medicinal Products for Veterinary Use opinion will now proceed to the European Commission for final EU-wide marketing authorization.

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