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Axitinib and Long-Acting Octreotide in Advanced Extrapancreatic Neuroendocrine Tumors: A Randomized, Double-Blind, Placebo-Controlled, Phase III Clinical Trial (AXINET, GETNE 1107) - PubMed
2 months ago
- Axitinib combined with long-acting octreotide was evaluated in a phase III trial (AXINET, GETNE 1107) for advanced extrapancreatic neuroendocrine tumors (epNETs).
- The study was randomized, double-blind, and placebo-controlled, involving 256 patients with unresectable/metastatic G1-2 epNETs.
- Primary endpoint was investigator-assessed progression-free survival (PFS), with a median PFS of 17.2 months for axitinib vs. 13.1 months for placebo.
- Blinded independent central review (BICR) showed a median PFS of 16.6 months for axitinib vs. 9.9 months for placebo (HR 0.71, P=0.017).
- Objective response rate (ORR) was significantly higher with axitinib (17.5% vs. 4.6% per investigator; 12.8% vs. 3.2% per BICR).
- Common grade ≥3 toxicities included hypertension (24.0% vs. 9.2%) and diarrhea (13.6% vs. 1.5%).
- Axitinib improved PFS per BICR and ORR but did not meet the primary endpoint of investigator-assessed PFS.
Safety and feasibility of intravenous fresh adipose-derived mesenchymal stem cells in secondary progressive multiple sclerosis: phase I/IIa clinical results - PubMed
2 months ago
- Study evaluated safety and clinical effects of fresh autologous adipose-derived mesenchymal stem cells (AT-MSCs) in secondary progressive multiple sclerosis (SPMS).
- 10 female SPMS patients received high-dose intravenous AT-MSCs in two doses, seven days apart.
- No serious adverse events were reported over 9 months post-transplantation.
- AT-MSC therapy reduced T2-FLAIR lesion number and volume, improved EDSS scores, and enhanced psychological outcomes.
- Therapy increased regulatory T-cell proportions and anti-inflammatory cytokine expression while decreasing inflammatory cytokines.
- Findings suggest AT-MSCs are safe and may offer clinical benefits for SPMS patients.
Safety and efficacy of intensive task-specific training in people with recent spinal cord injury: a phase 3, pragmatic, randomised, assessor-blinded, superiority trial - PubMed
2 months ago
- Study aimed to determine if 10 weeks of intensive task-specific training plus strength training improves recovery in recent spinal cord injury (SCI) patients.
- Phase 3 randomized controlled trial conducted across 15 hospitals in multiple countries.
- 220 participants were randomly assigned to either usual care (control) or usual care plus 12 hours per week of intensive training (intervention).
- Primary outcome was Total Motor Score of the International Standards for the Neurological Classification of SCI at 10 weeks.
- No significant difference in Total Motor Scores between the intervention and control groups (mean difference 0.93, p=0.48).
- Four serious adverse events reported, including two deaths in the intervention group.
- Conclusion: Intensive task-specific training did not provide significant benefits over usual inpatient rehabilitation care.
- Funding provided by New South Wales Ministry of Health, University of Sydney, and Wings for Life.
Live biotherapeutic product IBP-9414 (L. reuteri) in very low birth weight infants: the Connection Study - PubMed
2 months ago
- The Connection Study evaluated the efficacy and safety of IBP-9414 (L. reuteri) in very low birth weight (VLBW) infants.
- IBP-9414 did not significantly reduce necrotizing enterocolitis (NEC) incidence or improve sustained feeding tolerance (SFT).
- All-cause mortality was reduced with IBP-9414 (6.2% vs. 8.5% in placebo group, RR: 0.73, p = 0.036).
- IBP-9414 was found to be safe, with no evidence of the product in blood cultures.
- The study supports the use of IBP-9414 in VLBW infants to safely reduce mortality and morbidity.
Evaluating treatment to a target of transmural healing in patients with moderately to severely active Crohn's disease: rationale, design and protocol for the randomised controlled VECTORS trial - PubM
2 months ago
- The VECTORS trial evaluates transmural healing (TMH) as a treatment target for Crohn's disease (CD).
- This phase 4, randomized controlled trial involves 304 adult patients with moderately to severely active CD.
- Patients are assigned to one of two treatment target groups: TMH plus clinical/biomarker remission or clinical/biomarker remission alone.
- All participants receive vedolizumab, with additional dosing and escalation to meet assigned targets.
- Primary outcome measures corticosteroid-free endoscopic remission at week 48, with CD-related complications assessed at week 96.
- The trial is approved by ethics committees, with findings to be published in peer-reviewed journals.
Design and rationale of RECOVER-AUTONOMIC: a randomized platform trial evaluating interventions for Long COVID postural orthostatic tachycardia syndrome - PubMed
2 months ago
- RECOVER-AUTONOMIC is a randomized platform trial evaluating interventions for Long COVID-related postural orthostatic tachycardia syndrome (POTS).
- The trial assesses three interventions: coordinated nonpharmacologic care, intravenous immunoglobulin (IVIG), and ivabradine.
- Primary endpoint measures change in orthostatic intolerance symptoms using the Orthostatic Hypotension Questionnaire/Orthostatic Intolerance Questionnaire.
- Secondary endpoints include quality of life, functional performance, symptom burden, and safety.
- Exploratory endpoints involve autonomic function testing, wearable sensor data, and biomarker profiling.
- The trial aims to provide evidence for treating POTS in Long COVID patients.
- Trial registration details and protocol are available on ClinicalTrials.gov and the RECOVER website.
Belantamab mafodotin, carfilzomib, lenalidomide, and dexamethasone for relapsed or refractory multiple myeloma - PubMed
2 months ago
- Belantamab mafodotin (belamaf) combined with carfilzomib, lenalidomide, and dexamethasone (KRd-b) was evaluated for relapsed/refractory multiple myeloma (RRMM).
- The study used a phase 1/2 design with belamaf administered every 8 weeks at 1.4 or 1.9 mg/kg, with 1.9 mg/kg selected as the recommended phase 2 dose.
- Overall response rate was 89.5%, with 78.9% achieving very good partial response or better, and minimal residual disease negativity in all complete responders.
- At a median follow-up of 19.3 months, 24-month progression-free survival and overall survival rates were 74.3% and 85.1%, respectively.
- Keratopathy occurred in 94.7% of patients, mostly grade 1-2, reversible, and manageable without permanent discontinuation; grade ≥3 keratopathy was 31.6%.
- Other adverse events were manageable and consistent with known profiles of KRd or belamaf.
- The combination showed deep and durable responses, including in high-risk and lenalidomide maintenance dose-refractory patients.
- The study supports further evaluation of KRd-b with extended-interval belamaf in phase 2 trials.
Colchicine in patients with chronic inflammatory cardiomyopathy: Rationale and design of the CMP-MYTHiC - PubMed
2 months ago
- The CMP-MYTHiC trial evaluates colchicine's efficacy in reducing myocardial inflammation in chronic inflammatory cardiomyopathy (Infl-CMP) patients.
- Infl-CMP, diagnosed via CMRI or FDG-PET, increases risks of ventricular arrhythmias (VA), left ventricular systolic dysfunction (LVSD), and heart failure (HF).
- Eligibility includes adult patients with VA or LVSD/HF phenotypes, excluding those with myocardial infarction, other cardiomyopathies, or autoimmune disorders.
- Primary endpoint assesses clinical, arrhythmic, and imaging improvements at 6 months, comparing colchicine to placebo.
- The trial aims for 80% power, requiring 40 patients per group, with results expected by 2029.
Phase 1 Study of INBRX-105, a TNFRSF9 (4-1BB) and PD-L1 Bispecific Antibody, in Patients with Select Solid Tumors - PubMed
2 months ago
- INBRX-105 is a tetravalent, PD-L1-targeted TNFRSF9 (4-1BB) agonist evaluated in a phase 1 study for solid tumors.
- The study included 160 patients, with 81 receiving monotherapy and 79 receiving combination therapy with pembrolizumab.
- The maximum tolerated dose of INBRX-105 was 0.3 mg/kg every 3 weeks.
- Common adverse events included fatigue, increased aspartate aminotransferase, and nausea, with notable hepatic toxicity.
- Objective response rates were low: 8.8% overall, 3.7% for monotherapy, and 13.9% for combination therapy.
- Clinical development of INBRX-105 was discontinued due to hepatotoxicity and limited efficacy.
- The study highlights the need for novel immunotherapy combinations to address tumor resistance to checkpoint inhibitors.
Coronary atherosclerotic plaque intervention with Tongxinluo capsule (TXL-CAP): a multicenter, randomized, double-blind and placebo-controlled study - PubMed
2 months ago
- The TXL-CAP study investigated the effects of Tongxinluo capsule (TXL) on coronary plaque stability in patients with acute coronary syndrome.
- This was a multicenter, randomized, double-blind, placebo-controlled clinical trial involving 220 patients with coronary thin-cap lipid-rich plaques detected by OCT.
- Patients were treated with either TXL or placebo for 12 months alongside guideline-directed therapy, including statins.
- Primary endpoint: TXL significantly increased the minimum fibrous cap thickness compared to placebo (115.0 μm vs. 80.0 μm, P < 0.001).
- Secondary outcomes: TXL also showed greater reduction in maximum lipid arc and improved angina symptoms (Seattle Angina Questionnaire and Canadian Cardiovascular Society grading).
- No significant difference in cardiovascular events was observed between the groups at 12 months.
- Conclusion: TXL enhances plaque stability and symptom relief in acute coronary syndrome patients without affecting cardiovascular event rates.
A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia - PubMed
2 months ago
- A multi-center clinical trial evaluated allogeneic hematopoietic stem cell transplantation (allo-HSCT) for transfusion-dependent thalassemia (TDT).
- The study included 823 patients, using grafts from HLA-matched sibling donors (MSDs), matched unrelated donors (MUDs), and haploidentical related donors (Haplos).
- Conditioning regimen involved busulfan, cyclophosphamide, fludarabine, and anti-thymocyte globulin.
- Graft-versus-host disease (GvHD) prophylaxis varied based on donor type.
- Primary endpoints were 2-year overall survival (OS) and event-free survival (EFS).
- 2-year OS for MSDs, MUDs, and Haplos was 97.2%, 93.1%, and 95.4% respectively.
- EFS for MSDs, MUDs, and Haplos was 97.2%, 92.9%, and 94.7% respectively.
- GvHD-free, relapse-free survival (GRFS) was higher for MSDs (91.4%) compared to MUDs (77.0%) and Haplos (75.6%).
- Incidence of acute and chronic GvHD was higher with alternative donors than MSDs.
- Transplant-related mortality was 4.4%, and graft failure rate was 0.5%.
- The findings suggest expanding the donor pool for TDT patients lacking MSDs.
Efficacy and safety of relacorilant for the treatment of patients with Cushing's syndrome (GRACE): a multicentre, phase 3, double-blind, placebo-controlled, randomised-withdrawal study - PubMed
2 months ago
- Relacorilant is a selective glucocorticoid receptor modulator designed to treat Cushing's syndrome by reducing excess cortisol activity.
- The GRACE study was a phase 3, double-blind, placebo-controlled, randomized-withdrawal trial conducted across 77 centers in 11 countries.
- Patients with endogenous hypercortisolism and hypertension or hyperglycemia were enrolled, receiving relacorilant (100-400 mg daily) in an open-label phase followed by a randomized withdrawal phase.
- The primary outcome was the proportion of patients losing hypertension control during the withdrawal phase, with relacorilant showing significantly better maintenance of control compared to placebo.
- Common adverse events included back pain, acne, arthralgia, bursitis, headache, and insomnia, with no cases of severe glucocorticoid receptor antagonism or adrenal insufficiency.
- The study supports relacorilant as a therapeutic option for managing the harmful effects of endogenous hypercortisolism.
- Funding and conflicts of interest: The study was funded by Corcept Therapeutics, with several authors reporting ties to pharmaceutical companies including Corcept, Recordati, and others.
First-Line Tislelizumab Plus Chemotherapy for Recurrent or Metastatic Nasopharyngeal Cancer: Three-Year Follow-Up of the Phase 3 RATIONALE-309 Randomized Clinical Trial - PubMed
2 months ago
- First-line tislelizumab plus chemotherapy showed improved progression-free survival (PFS) vs. placebo plus chemotherapy in recurrent or metastatic nasopharyngeal cancer (NPC).
- Median PFS was 9.6 months for tislelizumab vs. 7.4 months for placebo (HR 0.53).
- Median overall survival (OS) was 45.3 months for tislelizumab vs. 31.8 months for placebo (HR 0.73).
- Greater OS benefit was observed in participants with high B-cell gene expression (HR 0.41).
- Treatment-emergent adverse events were common but manageable, with higher immune-mediated events in the tislelizumab group.
- The study supports tislelizumab plus chemotherapy as a viable first-line treatment for recurrent or metastatic NPC.
Morphine Plus Placebo vs Morphine Plus Acetaminophen for Acute Pain in the Emergency Department: A Randomized Clinical Trial - PubMed
2 months ago
- Study compares morphine plus placebo vs morphine plus acetaminophen for acute pain in emergency departments (EDs).
- Multicenter, double-blind, randomized, placebo-controlled noninferiority clinical trial conducted in 11 French EDs.
- Participants were adults (≥18 years) with severe acute pain (NRS score ≥5).
- Primary outcome: mean change in NRS pain score from baseline to 30 minutes.
- Noninferiority margin set at 1 point.
- Results show morphine plus placebo did not meet noninferiority criterion compared to morphine plus acetaminophen.
- Acetaminophen may have potential benefit as an adjunct to morphine for acute pain in EDs.
Amivantamab-Lazertinib in EGFR-Mutated Advanced NSCLC - PubMed
2 months ago
- Study evaluates amivantamab-lazertinib combination in EGFR-mutated advanced NSCLC.
- Clinical trial results published in the New England Journal of Medicine.
- Findings indicate potential benefits in overall survival for patients.
- Multinational research effort involving numerous investigators and institutions.
Tecovirimat for the Treatment of Mpox - PubMed
2 months ago
- Tecovirimat was evaluated in a phase 3 trial for treating clade II mpox (monkeypox).
- The study was a double-blind, randomized, placebo-controlled trial involving 412 participants.
- Participants received either tecovirimat or placebo for 14 days, with 344 having confirmed mpox.
- Primary outcome was clinical resolution; secondary outcomes included pain reduction and viral DNA clearance.
- Results showed no significant difference in clinical resolution between tecovirimat (83%) and placebo (84%).
- No substantial differences were observed in pain reduction, lesion healing, or viral clearance.
- Adverse events of grade 3 or higher were similar in both groups (4% tecovirimat vs. 3% placebo).
- Conclusion: Tecovirimat did not shorten clinical resolution time, reduce pain, or enhance viral clearance in mpox patients.
Nerandomilast in idiopathic pulmonary fibrosis: data from the whole follow-up period of the FIBRONEER-IPF trial - PubMed
2 months ago
- Nerandomilast met the primary endpoint in the FIBRONEER-IPF trial by reducing decline in forced vital capacity at week 52 in patients with idiopathic pulmonary fibrosis.
- The study assessed the effects of nerandomilast over the full follow-up period, focusing on time to first acute exacerbation, hospitalization for respiratory cause, or death.
- Hazard ratios for the key secondary endpoint and death showed no significant effect for nerandomilast 9 mg bid, but nerandomilast 18 mg bid was associated with a numerically lower risk of death.
- Adverse events leading to treatment discontinuation were slightly higher in the nerandomilast groups compared to placebo.
- Nerandomilast demonstrated a favorable safety profile with a low rate of discontinuation due to adverse events.
Switching to twice-yearly depemokimab from mepolizumab/benralizumab in severe asthma: A multicenter, randomized, double-blind, Phase 3A Clinical Trial (NIMBLE) - PubMed
2 months ago
- Depemokimab is an ultra-long-acting biologic with high interleukin-5 binding affinity, enabling twice-yearly dosing.
- The NIMBLE trial investigated switching severe asthma patients from mepolizumab/benralizumab to depemokimab.
- Primary endpoint: Annualized rate of clinically significant exacerbations over 52 weeks (non-inferiority margin: 1.28).
- Non-inferiority was not met (rate ratio: 1.16 [0.98 to 1.38]).
- Exacerbation rates were low in both groups, with symptom control and lung function maintained.
- Adverse events were comparable between depemokimab and active comparator groups.
- First randomized controlled switch trial suggests safe transition to twice-yearly depemokimab.
Phase 2 study of relmacabtagene autoleucel (CD19 CAR-T) for relapsed/refractory mantle cell lymphoma in Chinese adults - PubMed
2 months ago
- Relmacabtagene autoleucel (relma-cel) is an anti-CD19 CAR-T therapy approved in China for certain lymphomas.
- Phase 2 study evaluated relma-cel in 70 Chinese adults with relapsed/refractory mantle cell lymphoma (MCL).
- 59 patients received a single infusion of 100 × 10^6 CAR-positive T cells.
- At 3 months, the objective response rate (ORR) was 71.19%, with a complete response rate of 59.32%.
- Median duration of response (DOR) was 18.1 months, progression-free survival (PFS) was 15.5 months, and overall survival (OS) was 19.5 months.
- Common grade 3 or higher adverse events included neutropenia (76.3%), leukopenia (69.5%), and lymphopenia (47.5%).
- Severe cytokine release syndrome and neurotoxicity each occurred in 6.8% of patients, with all cases resolving fully.
- No fatal events were reported.
- Relma-cel demonstrated high response rates, durable remissions, and manageable safety in R/R MCL patients.
Sequential or upfront triple combination with durvalumab, tremelimumab, and bevacizumab for patients with unresectable hepatocellular carcinoma: the MONTBLANC trial protocol (AIO-HEP-0325/ass) - PubMe
2 months ago
- The MONTBLANC trial investigates a triple combination therapy for unresectable hepatocellular carcinoma (HCC) using durvalumab, tremelimumab, and bevacizumab.
- This phase II, randomized, open-label study compares upfront triple-agent therapy versus sequential doublet therapy (durvalumab + tremelimumab) followed by bevacizumab upon progression.
- Primary endpoint is overall response rate; secondary endpoints include overall survival, progression-free survival, safety, and patient-reported outcomes.
- The study aims to enhance efficacy in advanced HCC, where current immune checkpoint inhibitor therapies benefit only a subset of patients.
- Ethical approval has been obtained, and the trial adheres to Declaration of Helsinki and Good Clinical Practice standards.
- Trial registered at ClinicalTrials.gov (NCT05844046) and EU Clinical Trials Register (2022-001201-48).

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#clinical trial

Axitinib and Long-Acting Octreotide in Advanced Extrapancreatic Neuroendocrine Tumors: A Randomized, Double-Blind, Placebo-Controlled, Phase III Clinical Trial (AXINET, GETNE 1107) - PubMed

Safety and feasibility of intravenous fresh adipose-derived mesenchymal stem cells in secondary progressive multiple sclerosis: phase I/IIa clinical results - PubMed

Safety and efficacy of intensive task-specific training in people with recent spinal cord injury: a phase 3, pragmatic, randomised, assessor-blinded, superiority trial - PubMed

Live biotherapeutic product IBP-9414 (L. reuteri) in very low birth weight infants: the Connection Study - PubMed

Evaluating treatment to a target of transmural healing in patients with moderately to severely active Crohn's disease: rationale, design and protocol for the randomised controlled VECTORS trial - PubM

Design and rationale of RECOVER-AUTONOMIC: a randomized platform trial evaluating interventions for Long COVID postural orthostatic tachycardia syndrome - PubMed

Belantamab mafodotin, carfilzomib, lenalidomide, and dexamethasone for relapsed or refractory multiple myeloma - PubMed

Colchicine in patients with chronic inflammatory cardiomyopathy: Rationale and design of the CMP-MYTHiC - PubMed

Phase 1 Study of INBRX-105, a TNFRSF9 (4-1BB) and PD-L1 Bispecific Antibody, in Patients with Select Solid Tumors - PubMed

Coronary atherosclerotic plaque intervention with Tongxinluo capsule (TXL-CAP): a multicenter, randomized, double-blind and placebo-controlled study - PubMed

A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia - PubMed

Efficacy and safety of relacorilant for the treatment of patients with Cushing's syndrome (GRACE): a multicentre, phase 3, double-blind, placebo-controlled, randomised-withdrawal study - PubMed

First-Line Tislelizumab Plus Chemotherapy for Recurrent or Metastatic Nasopharyngeal Cancer: Three-Year Follow-Up of the Phase 3 RATIONALE-309 Randomized Clinical Trial - PubMed

Morphine Plus Placebo vs Morphine Plus Acetaminophen for Acute Pain in the Emergency Department: A Randomized Clinical Trial - PubMed

Amivantamab-Lazertinib in EGFR-Mutated Advanced NSCLC - PubMed

Tecovirimat for the Treatment of Mpox - PubMed

Nerandomilast in idiopathic pulmonary fibrosis: data from the whole follow-up period of the FIBRONEER-IPF trial - PubMed

Switching to twice-yearly depemokimab from mepolizumab/benralizumab in severe asthma: A multicenter, randomized, double-blind, Phase 3A Clinical Trial (NIMBLE) - PubMed

Phase 2 study of relmacabtagene autoleucel (CD19 CAR-T) for relapsed/refractory mantle cell lymphoma in Chinese adults - PubMed

Sequential or upfront triple combination with durvalumab, tremelimumab, and bevacizumab for patients with unresectable hepatocellular carcinoma: the MONTBLANC trial protocol (AIO-HEP-0325/ass) - PubMe