A Randomized, Double-blind, Multicenter, Two-period Crossover Study to Assess the Efficacy and Tolerability of NM-BL (Burlulipase) in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis

Start Date01 May 2013

Sponsor / Collaborator

Nordmark Arzneimittel GmbH & Co. KG

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100 Clinical Results associated with Burlulipase

100 Translational Medicine associated with Burlulipase

100 Patents (Medical) associated with Burlulipase

Literatures (Medical) associated with Burlulipase

25 Dec 2023·Turkish Journal of Pharmaceutical Sciences

Development and Evaluation of Methotrexate and Baicalin Loaded Nano- Lipid Carriers for Psoriasis Treatment

Article

Author: Arshad, Saloma ; Dar, M. Junaid ; Iqbal, Kashif ; Khalid, Sidra ; Sohail, Hassan ; Dar, Muhammad Junaid ; Sohail, Sundus

ObjectivesPsoriasis is a chronic inflammatory, T-lymphocyte immune-mediated skin disease. In this study, skin-permeating nanolipid carriers (NLCs) of Methotrexate (MTX) and Baicalin (BL) were formulated. This further gave formulation of nano-lipid encapsulated carriers for dual-drug delivery of the hydrophilic and hydrophobic drugs through the liposomal gel.Materials and MethodsOptimization of the formulation of NLCs was performed and characterized by determining their particle size, drug permeation, skin irritation, drug loading capacity, stability, in vitro drug release behavior, and in vitro cellular viability. Ex vivo skin permeation and in vivo psoriatic efficiency were also evaluated and compared.ResultsResults revealed that the amount of MTX permeating the skin was 2.4 to 4.4 fold greater for dual-drug s than for single NLCs. The optimized dual-drug loaded NLCs had an average particle size (150.20 ± 3.57 nm) and polydispersity index (0.301 ± 0.01) and high entrapment (86.32 ± 2.78% w/w). The MTX nanoparticles exhibit a positive Zeta potential of 38.6 mV. The psoriasis area and severity index scoring showed the lowest skin erythema, skin thickness and scaling. MTX-BL NLCs were inhibited the expression of inflammatory cytokines (tumor necrosis factor-alpha, and interleukin-17) .ConclusionIt can be concluded that newer targeting strategies for NLCs for dual-drug delivery of nano-lipid carriers could be administered topically for the treatment of psoriasis.

01 Sep 2016·The Journal of PediatricsQ2 · MEDICINE

Safety and Efficacy of a Novel Microbial Lipase in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis: A Randomized Controlled Clinical Trial

Q2 · MEDICINE

Article

Author: Schaeffer, David ; Strausbaugh, Steven ; Sollo, Natalie ; Ahrens, Richard C ; Heubi, James E ; Forssmann, Kristin ; Jain, Raksha ; Graff, Gavin ; Witte, Stephan

OBJECTIVETo evaluate the safety and efficacy of a novel microbial lipase (NM-BL) in a liquid formulation for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF) in a phase IIa proof-of-concept study.STUDY DESIGNWe conducted a double-blind, randomized, placebo controlled crossover study in patients with cystic fibrosis and exocrine pancreatic insufficiency. Adolescent and adult patients with CF were randomized to receive NM-BL or placebo for 1 week as replacement for their usual pancreatic enzyme formulation. They were subsequently crossed-over to the alternate study treatment. The coefficient of fat absorption was evaluated as the primary endpoint. Symptoms and adverse events were evaluated as secondary endpoints.RESULTSA total of 35 patients were randomized into the study and 22 patients completed both treatment periods. During treatment with NM-BL, the coefficient of fat absorption was significantly greater (72.7%) compared with placebo (53.8%) with a difference between groups of 18.8% (P < .001). Subjective assessment of stool fat and stool consistency also improved under treatment with NM-BL. Adverse events were mostly gastrointestinal in nature and were more common in the group receiving NM-BL.CONCLUSIONSCurrently available pancreatic enzyme products are limited because of the lack of liquid formulations and being largely porcine based. The novel microbial lipase NM-BL was safe and effective in this short term trial. The trial provided clinical proof-of-concept for this novel microbial lipase as a treatment for EPI in CF. A larger phase 2 dose ranging trial is warranted.TRIAL REGISTRATIONClinicalTrials.gov: NCT01710644.

01 Feb 2011·Xi bao yu fen zi mian yi xue za zhi = Chinese journal of cellular and molecular immunology

[Effects of H2-Bl gene transfection on biological behaviour of mouse vascular smooth muscle cell].

Article

Author: Chen, Li ; Ma, Jie ; Xu, Yi-Jun ; Li, Li-Yuan ; Deng, Yong-Zhi ; Li, Hong-Fang ; Bian, Yun-Fei

AIMTo investigate the effects of H2-Bl gene on biological behaviour of mouse vascular smooth muscle cells (VSMCs) including apoptosis, proliferation and anti-cytolysis to peripheral blood mononuclear cells (PBMCs), then speculate the possible mechanism of immunological rejection after heart transplantation in maternal-fetal immune tolerance.METHODSThe mouse VSMC were transfected with 0.5 mg/L of pEGFP-N1 plasmid vector, 0.5 mg/L of pEGFP-N1-H2B1 plasmid vector and 1.0 mg/L of pEGFP-N1-H2B1 plasmid vector, respectively. The efficiency of transfection was detected, and the H2-Bl mRNA level, the apoptosis, the proliferation and cytotoxicity of VSMCs were also investigated at 24 h, 48 h and 72 h, respectively.RESULTSExpression of the H2-Bl gene was determined by real time quantitative PCR. Expressions of the H2-Bl in experimental groups were higher than that of the control group (P<0.001). VSMC apoptosis was observed after 24 h in the 0.5 mg/L and 1.0 mg/L H2-Bl experimental groups compared with control group (P<0.05, P<0.001, respectively). VSMC proliferation was also inhibited in the 0.5 mg/L and 1.0 mg/L H2-Bl gene groups at 24 h and 72 h (P<0.05, P<0.01, respectively). The cytotoxicity of PBMC in the 0.5 mg/L and 1.0 mg/L H2-Bl gene groups at 24 h was lower than the control group (P<0.005, P<0.001, respectively).CONCLUSIONTransfection of pEGFP-N1-H2B1 plasmid to mouse VSMC causes the over expression of H2-Bl mRNA, induces the apoptosis, inhibits the proliferation and attenuates the cytotoxicity of PBMC, leading to immune tolerance.

100 Deals associated with Burlulipase

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	Phase 2	US	Nordmark Arzneimittel GmbH & Co. KG	01 May 2013
Exocrine Pancreatic Insufficiency	Phase 2	US	Nordmark Arzneimittel GmbH & Co. KG	01 May 2013

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