- |||||||||| Jardiance (empagliflozin) / Eli Lilly, Boehringer Ingelheim
Long-Term Effects of Empagliflozin in CKD (Hall D, Convention Center) - Oct 12, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4504;
Study empagliflozin continued to exert benefit on cardiorenal outcomes for a period after its discontinuation. The post-trial benefit was smaller than the benefit when taking study treatment and appeared to be temporary.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
LONG-TERM FINDINGS OF N-ACETYL-L-LEUCINE FOR NIEMANN-PICK DISEASE TYPE C (Camellia Auditorium | Level 0) - Jun 13, 2024 - Abstract #SSIEM2024SSIEM_72;
Background: The IB1001-301 clinical trial was a Phase III, double-blind, randomized, placebo-controlled trial comparing N-acetyl-L-leucine (NALL) with placebo for the treatment of neurological signs and symptoms in Niemann-Pick disease type C (NPC) after 12 weeks... In patients with NPC, treatment with NALL after 1 year was associated with a statistically and clinically significant reduction in disease progression and consistent with a neuroprotective and disease-modifying effect.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Results of a Phase III, Randomized, Placebo-controlled Crossover Trial with N-acetyl-L-leucine for Niemann-Pick Disease Type C (Colorado Convention Center | Bellco Theatre) - Feb 5, 2024 - Abstract #AAN2024AAN_1063;
P3
The secondary endpoints (CGI, SCAFI, mDRS) were also improved with IB1001 as compared to placebo (p<0.001)...NALL demonstrated a statistically significant improvement over placebo with a clinically meaningful benefit in symptoms (motor and cognitive), functioning, cognition, and quality of life. NALL was safe and well-tolerated, providing a favorable benefit-risk profile for the treatment of NPC.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - Nov 28, 2023
P2, N=33, Completed,
NALL was safe and well-tolerated, providing a favorable benefit-risk profile for the treatment of NPC. Active, not recruiting --> Completed
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - Aug 30, 2023
P2, N=39, Recruiting,
Active, not recruiting --> Completed Trial completion date: Jul 2023 --> Mar 2025 | Trial primary completion date: Jul 2023 --> Dec 2024
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Acetyl-L-Leucine for Niemann-Pick type C: A Multinational Double-blind Randomized Placebo-controlled Crossover Study (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_4292;
P3
Patients are assessed during a baseline period and then randomized (1:1) to one of two treatment sequences: IB1001 followed by placebo or vice versa...Conclusions Given the lack of global symptomatic or disease-modifying therapies for NPC and other lysosomal diseases there is an urgent need for effective and well-tolerated drug treatments. This clinical trial was designed through a collaboration between National Regulatory Agencies, leading Clinical Experts, Patient Organizations, and the industry Sponsored.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - Mar 1, 2023
P2, N=39, Recruiting,
NALL is a promising, easily administered (oral) therapeutic option for these rare, debilitating diseases with immense unmet medical needs. Trial completion date: Mar 2023 --> Jul 2023 | Trial primary completion date: Dec 2022 --> Jul 2023
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion, Trial completion date: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - Mar 1, 2023
P2, N=30, Completed,
Trial completion date: Mar 2023 --> Jul 2023 | Trial primary completion date: Dec 2022 --> Jul 2023 Active, not recruiting --> Completed | Trial completion date: Dec 2023 --> Jan 2023
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Enrollment closed: A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - Jan 3, 2023
P3, N=53, Active, not recruiting,
Active, not recruiting --> Completed | Trial completion date: Dec 2023 --> Jan 2023 Recruiting --> Active, not recruiting
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - Aug 3, 2022
P2, N=39, Active, not recruiting,
Active, not recruiting --> Completed Trial completion date: Dec 2022 --> Dec 2023 | Trial primary completion date: Jun 2022 --> Mar 2023
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Enrollment open, Trial primary completion date: A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - Jul 7, 2022
P3, N=53, Recruiting,
Trial completion date: Dec 2022 --> Dec 2023 | Trial primary completion date: Jun 2022 --> Mar 2023 Not yet recruiting --> Recruiting | Trial primary completion date: Mar 2023 --> Aug 2023
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
N-acetyl-L-leucine Improves Symptoms and Functioning in GM2 Gangliosidosis (B9) - Apr 9, 2022 - Abstract #EAN2022EAN_785;
It is a safe, well-tolerated, easily administered oral therapy, therefore offering a favorable risk/benefit profile for this serious, debilitating disorder. NALL is a new therapeutic option for the treatment of this rare disease that has no other approved therapies worldwide.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial initiation date, Trial primary completion date: A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - Mar 28, 2022
P3, N=53, Not yet recruiting,
NALL is a new therapeutic option for the treatment of this rare disease that has no other approved therapies worldwide. Trial completion date: Apr 2024 --> Sep 2024 | Initiation date: Feb 2022 --> Jun 2022 | Trial primary completion date: Dec 2022 --> Mar 2023
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
HIS BUNDLE PACING AS PRIMARY PACING MODALITY: A SINGLE CENTER REPORT ON 1001 CONSECUTIVE CASES (Poster Hall_Hall C) - Jan 28, 2022 - Abstract #ACC2022ACC_1959;
1) HBP can be safely and successfully performed as the primary modality of pacing in a large, unselected patient population referred to a tertiary care center. 2) HBP maintains QRS in normal range in patients without HPS disease and normalizes abnormal QRS parameters in patients with HPS disease 3) Procedure and fluoroscopy times remain long and further development is needed in this area.
- |||||||||| Ixinity (trenonacog alfa) / Medexus
Enrollment closed, Trial completion date, Trial primary completion date: Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B (clinicaltrials.gov) - Aug 31, 2021
P3, N=21, Active, not recruiting,
3) Reintervention rate was 4% which included the initial learning period. Recruiting --> Active, not recruiting | Trial completion date: Sep 2021 --> Jul 2022 | Trial primary completion date: Aug 2021 --> Jul 2022
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Clinical, Retrospective data, Journal: Ten-year outcomes of uveal melanoma based on The Cancer Genome Atlas (TCGA) classification in 1001 cases. (Pubmed Central) - Jul 2, 2021
By comparison, increasing category (A vs. B vs. C vs. D) was associated with features of older age at presentation (56.8 vs. 52.8 vs. 61.1 vs. 63.5 years, P < 0.001), less often visual acuity of 20/20-20/50 (80% vs. 67% vs. 70% vs. 65%, P = 0.001), tumor location further from the optic disc (P < 0.001) and foveola (P < 0.001), and greater median tumor basal diameter (10.0 vs. 13.0 vs. 14.0 vs. 16.0 mm, P < 0.001) and tumor thickness (3.5 vs. 5.2 vs. 6.0 vs. 7.1 mm, P < 0.001). The Kaplan-Meier (5-year/10-year) rate of metastasis was 4%/6% for Group A, 12%/20% for Group B, 33%/49% for Group C, and 60%/not available for Group D. A simplified 4-category classification of uveal melanoma using TCGA, based on tumor DNA, is highly predictive of risk for metastatic disease.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Clinical, Journal: A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. (Pubmed Central) - Jun 22, 2021
P2
The novel CI-CS assessment, developed through a collaboration of all stakeholders, is advantageous in that it better ensures the primary endpoint is functionally relevant for each patient, is able to capture small but meaningful clinical changes critical to the patients' quality of life (fine-motor skills; gait), and blinds the primary outcome assessment. The results of these three trials will inform whether N-acetyl-L-leucine is an effective treatment for NPC, GM2, and A-T and can also serve as a new therapeutic paradigm for the development of future treatments for other orphan diseases.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Enrollment closed: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - May 24, 2021
P2, N=39, Active, not recruiting,
The results of these three trials will inform whether N-acetyl-L-leucine is an effective treatment for NPC, GM2, and A-T and can also serve as a new therapeutic paradigm for the development of future treatments for other orphan diseases. Recruiting --> Active, not recruiting
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - Apr 22, 2021
P2, N=39, Recruiting,
Recruiting --> Active, not recruiting Trial completion date: Dec 2021 --> Dec 2022 | Trial primary completion date: Mar 2021 --> Jun 2022
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - Apr 22, 2021
P2, N=39, Recruiting,
Trial completion date: Dec 2021 --> Dec 2022 | Trial primary completion date: Mar 2021 --> Jun 2022 Trial completion date: Apr 2022 --> Mar 2023 | Trial primary completion date: Apr 2021 --> Dec 2022
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial primary completion date: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - Feb 9, 2021
P2, N=39, Recruiting,
Uveal melanoma diagnosis at older age is associated with more advanced TCGA classification, larger tumor size and higher metastasis rate. Trial primary completion date: Dec 2020 --> Mar 2021
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - Feb 9, 2021
P2, N=34, Active, not recruiting,
Trial primary completion date: Dec 2020 --> Mar 2021 Trial completion date: Dec 2021 --> Dec 2022 | Trial primary completion date: Nov 2020 --> Nov 2022
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - Dec 17, 2020
P2, N=39, Recruiting,
Trial completion date: Dec 2021 --> Dec 2022 | Trial primary completion date: Nov 2020 --> Nov 2022 Trial completion date: Feb 2021 --> Apr 2022 | Trial primary completion date: Dec 2020 --> Apr 2021
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Clinical, Journal: Hereditary Ataxias in Cuba: A Nationwide Epidemiological and Clinical Study in 1001 Patients. (Pubmed Central) - Nov 25, 2020
The spreading of SCA2 to whole country and the documented raising of its prevalence set the rationales for higher-scope medical care and research strategies, supported in collaborative research networks. The wide epidemiological, clinical, and genetic characterization of this founder SCA2 population identifies this homogeneous cohort as an attractive source for the development of future clinical-genetic and therapeutic researches.
- |||||||||| Trial completion: ATHN 2: Factor Switching Study (clinicaltrials.gov) - Aug 19, 2020
P=N/A, N=310, Completed,
Trial completion date: Feb 2021 --> Dec 2021 Active, not recruiting --> Completed
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - Aug 13, 2020
P2, N=34, Active, not recruiting,
Active, not recruiting --> Completed Trial completion date: Dec 2020 --> Dec 2021
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - Aug 8, 2020
P2, N=34, Active, not recruiting,
Trial completion date: Dec 2020 --> Dec 2021 Trial completion date: Jul 2020 --> Dec 2020 | Trial primary completion date: Jun 2020 --> Nov 2020
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (clinicaltrials.gov) - Aug 8, 2020
P2, N=39, Recruiting,
Trial completion date: Jul 2020 --> Dec 2020 | Trial primary completion date: Jun 2020 --> Nov 2020 Trial completion date: Jul 2020 --> Feb 2021 | Trial primary completion date: Apr 2020 --> Dec 2020
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Trial completion date, Trial primary completion date: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - Aug 8, 2020
P2, N=39, Recruiting,
Trial completion date: Jul 2020 --> Feb 2021 | Trial primary completion date: Apr 2020 --> Dec 2020 Trial completion date: Sep 2020 --> Feb 2021 | Trial primary completion date: Jul 2020 --> Dec 2020
- |||||||||| Ixinity (trenonacog alfa) / Aptevo, Emergent Biosolutions
[VIRTUAL] N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C, GM2-Gangliosidosis and Ataxia telangiectasia: Three multinational, multicenter, open-label, rater-blinded phase II clinical trials (Room Istanbul) - Apr 9, 2020 - Abstract #EAN2020EAN_925;
Background and aims: Phase II trials are investigating the effects of N-Acetyl-L-Leucine (IB1001/ALL) in 3 ultra- rare, autosomal-recessive, neurodegenerative disorders: Niemann-Pick disease, type C (NPC), Ataxia-telangiectasia (A-T) and GM2-Gangliosidosis (GM2)... A novel primary endpoint is being implemented to better demonstrate the clinically meaningful effect of ALL treatment in three ultra-rare neurodegenerative diseases with overlapping phenotypes.
- |||||||||| Ixinity (trenonacog alfa) / Medexus, Emergent Biosolutions
Enrollment closed: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - Feb 7, 2020
P2, N=34, Active, not recruiting,
A novel primary endpoint is being implemented to better demonstrate the clinically meaningful effect of ALL treatment in three ultra-rare neurodegenerative diseases with overlapping phenotypes. Recruiting --> Active, not recruiting
- |||||||||| Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date: ATHN 2: Factor Switching Study (clinicaltrials.gov) - Feb 5, 2020
P=N/A, N=310, Active, not recruiting,
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting | N=600 --> 310 | Trial completion date: Jan 2022 --> Jun 2020 | Trial primary completion date: Aug 2021 --> Jun 2020
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