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Clinical Trial News

DR.NOAH Biotech's AI-Driven ALS Drug, NDC-011, Receives FDA Approval for Phase 1 Trial

  • DR.NOAH Biotech received FDA approval for its Investigational New Drug (IND) application for Phase 1 clinical trial of NDC-011, a new drug combination for Amyotrophic Lateral Sclerosis (ALS).
  • The Phase 1 trial, set to begin in early 2025, will evaluate the bioavailability, safety, and tolerability of NDC-011 in 24 healthy adult participants in the U.S.
  • NDC-011, discovered through DR.NOAH's AI platform 'ARK,' showed efficacy in preclinical studies by delaying motor function deterioration and prolonging survival in ALS models.
  • The company aims for global market expansion and has partnered with Patheon for cGMP manufacturing of NDC-011, with Phase 2 trials expected to commence in 2026.

SHAM-PVI Trial Confirms Pulmonary Vein Isolation Efficacy in Atrial Fibrillation

  • The SHAM-PVI trial demonstrated that pulmonary vein isolation (PVI) significantly reduces atrial fibrillation (Afib) burden compared to a sham procedure.
  • PVI led to substantial improvements in patient-reported outcomes, including quality of life and symptom reduction, over a 6-month period.
  • The study provides evidence that the benefits of PVI in treating symptomatic Afib are not primarily due to a placebo effect.
  • Findings support the established role of PVI as an effective intervention for managing atrial fibrillation and improving patient well-being.

A Randomized Sham-controlled Study of Pulmonary Vein Isolation in Symptomatic Atrial Fibrillation

NCT04272762CompletedNot Applicable
East Sussex Hospitals NHS Trust
Posted 1/23/2020

Nipocalimab Shows Sustained Disease Control in Adolescents with Generalized Myasthenia Gravis

  • Nipocalimab, combined with standard of care, demonstrates sustained disease control in adolescents (12-17 years) with generalized myasthenia gravis (gMG) over 24 weeks.
  • The Phase 2/3 Vibrance-MG study shows a significant reduction in immunoglobulin G (IgG) levels and improvements in MG-ADL and QMG scores.
  • Nipocalimab is well-tolerated, with no serious adverse events reported, offering a potential new treatment option for adolescents with gMG.
  • Johnson & Johnson has submitted applications to the FDA and EMA seeking approval for nipocalimab for the treatment of gMG.

Efficacy and Safety Study of Nipocalimab for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

NCT05327114RecruitingPhase 2
Janssen Research & Development, LLC
Posted 9/23/2022

A Study of Nipocalimab in Adults With Primary Sjogren's Syndrome (pSS)

NCT04968912CompletedPhase 2
Janssen Research & Development, LLC
Posted 9/21/2021

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

NCT05265273RecruitingPhase 2
Janssen Research & Development, LLC
Posted 7/20/2022

A Study of Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies

NCT05379634Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 7/5/2022

A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN)

NCT05912517RecruitingPhase 3
Janssen Research & Development, LLC
Posted 12/20/2023

A Study to Evaluate the Nipocalimab and Certolizumab Combination Therapy in Participants With Active Rheumatoid Arthritis

NCT06028438CompletedPhase 2
Janssen Research & Development, LLC
Posted 8/15/2023

A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

NCT06149559RecruitingPhase 2
UCB Biopharma SRL
Posted 6/14/2024

A Study of Nipocalimab in Adult Participants With Active Systemic Lupus Erythematosus

NCT04882878CompletedPhase 2
Janssen Research & Development, LLC
Posted 8/20/2021

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis

NCT04833894RecruitingPhase 2
argenx
Posted 10/26/2021

A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis

NCT04951622RecruitingPhase 3
Janssen Research & Development, LLC
Posted 7/15/2021

Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia

NCT04119050RecruitingPhase 2
Janssen Research & Development, LLC
Posted 8/15/2019

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)

NCT03842189CompletedPhase 2
Janssen Research & Development, LLC
Posted 4/5/2018

IO Biotech's Melanoma Trial of IO102-IO103 Combination Therapy to Continue

  • IO Biotech's Phase III trial of IO102-IO103 with pembrolizumab for advanced melanoma will continue following IDMC review.
  • The IDMC found no new safety signals, supporting the ongoing evaluation of the therapeutic vaccine combination.
  • The primary endpoint, progression-free survival, is projected to be reached in the first half of 2025.
  • The trial compares IO102-IO103 plus pembrolizumab to pembrolizumab alone in previously untreated patients.

IO102-IO103 in Combination With Pembrolizumab Versus Pembrolizumab Alone in Advanced Melanoma (IOB-013 / KN-D18)

NCT05155254Active, Not RecruitingPhase 3
IO Biotech
Posted 5/17/2022

Phase II Trial of Neoadjuvant and Adjuvant IO102-IO103 and Pembrolizumab KEYTRUDA® in Patients With Resectable Tumors

NCT05280314RecruitingPhase 2
IO Biotech
Posted 12/21/2023

IO102-IO103 in Combination With Pembrolizumab as First-line Treatment for Patients With Metastatic NSCLC, SCCHN, or mUBC

NCT05077709Active, Not RecruitingPhase 2
IO Biotech
Posted 2/14/2022

Age Matters: Tailoring Treatment for Congenital Nasolacrimal Duct Obstruction in Children

  • A randomized clinical trial reveals that adding interventions like intubation or turbinate fracture to conventional probing does not significantly improve the success rate of simple Congenital Nasolacrimal Duct Obstruction (CNLDO) resolution in children aged 12-24 months.
  • In older children (24-36 months), interventions including intubation may lead to higher success rates in resolving CNLDO, suggesting age-based treatment strategies should be considered.
  • The study highlights the importance of considering patient age, surgical skills, available instruments, and parental preferences when determining the most appropriate treatment for simple CNLDO.
  • Probing alone shows high success rates (70-97%) in resolving CNLDO, aligning with the study's findings of 93.1% and 72.7% success rates for the 12-24 and 24-36 months age groups, respectively.

Childhood Leukemia Trial Shows Long-Term Remission with Targeted Therapy

  • A clinical trial (MyeChild01) testing treatment combinations for children with acute myeloid leukemia (AML) has shown promising long-term remission rates.
  • The trial included a targeted therapy that delivers chemotherapy directly into cancer cells, improving treatment outcomes for young patients.
  • One patient, Callum Kyle, diagnosed with AML at age three, has remained in remission for five years after participating in the MyeChild01 trial.
  • Cancer Research UK supports research to improve outcomes, doubling its annual spend on children's and young people's cancers in the last five years.

CAMZYOS Demonstrates Sustained Benefits in Obstructive Hypertrophic Cardiomyopathy Trial

  • Long-term follow-up data from the EXPLORER-LTE trial shows CAMZYOS (mavacamten) maintains efficacy and safety in adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
  • Patients treated with CAMZYOS experienced consistent improvements in echocardiographic measures and biomarkers, such as resting LVOT gradient and NT-proBNP levels, over 3.5 years.
  • Functional capacity also improved, with most patients achieving NYHA class I, indicating a significant reduction in symptoms and enhanced quality of life.
  • CAMZYOS is the first cardiac myosin inhibitor approved in the US for symptomatic oHCM, redefining the treatment landscape for this patient population.

RSV Vaccine Development Surges with New Approvals and Diverse Strategies

  • Recent advancements in understanding hRSV structure and immune responses have led to new vaccine approvals targeting older adults, pregnant women, and infants.
  • Several vaccine candidates are in clinical trials, employing live-attenuated, recombinant-vector, subunit, particle-based, and mRNA approaches to stimulate immunity.
  • Monoclonal antibody treatments like Nirsevimab offer immediate protection for infants, complementing vaccine strategies and addressing the high disease burden of hRSV.
  • Combination vaccines and mRNA modalities are being explored to broaden protection and improve vaccine delivery, though challenges remain in optimizing these new approaches.

Ultrasound-Localized Tuina and Nerve Blocks Show Promise for Cervicogenic Headache

  • A randomized controlled trial is set to investigate the efficacy of combining ultrasound-localized meridian sinew tuina with greater and third occipital nerve injections for cervicogenic headache (CEH).
  • The study will compare this integrative approach to ultrasound-guided nerve injections alone, assessing improvements in pain scores, headache intensity, and cervical spine mobility.
  • Medical infrared thermography will be used as an objective tool to evaluate treatment efficacy by measuring temperature variations related to nerve compression and inflammation.
  • This trial aims to provide a safe and effective manual therapy protocol, offering insights for future research and clinical management of CEH, a prevalent and challenging headache disorder.

Tolebrutinib Shows Promise in Slowing Disability Progression in Non-Relapsing Secondary Progressive MS

  • Tolebrutinib significantly delayed the onset of 6-month confirmed disability progression by 31% compared to placebo in patients with nrSPMS.
  • The proportion of patients experiencing confirmed disability improvement nearly doubled with tolebrutinib compared to placebo, indicating potential for functional gains.
  • Safety data showed liver enzyme elevations as a notable adverse event, but most cases resolved without intervention, emphasizing the need for careful monitoring.
  • HERCULES trial results support tolebrutinib as a potential treatment for nrSPMS, addressing a significant unmet need in managing disability accumulation.

A Study to Investigate the Efficacy of Fenebrutinib in Relapsing Multiple Sclerosis (RMS)

NCT05119569Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 3/1/2022

Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168)

NCT04411641CompletedPhase 3
Sanofi
Posted 9/24/2020

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 1)

NCT04410978Active, Not RecruitingPhase 3
Sanofi
Posted 6/30/2020

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 2)

NCT04410991Active, Not RecruitingPhase 3
Sanofi
Posted 6/11/2020

Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)

NCT04458051Active, Not RecruitingPhase 3
Sanofi
Posted 8/13/2020
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