Lancovutide (Moli1901) Inhalation Solution Study in Adolescents and Adults With Cystic Fibrosis

NCT00671736 — Phase 2, Completed, 160 participants, AOP Orphan Pharmaceuticals AG

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A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults With Cystic Fibrosis

Registry	NCT00671736
Status	Completed
Phase	Phase 2
Study type	Interventional; randomized, parallel, masking: quadruple, treatment
Enrollment	160 (Estimated)
Sponsor	AOP Orphan Pharmaceuticals AG (Industry)
Conditions	Cystic Fibrosis
Interventions	Moli1901, Moli1901 placebo (Drug)
Dates	start 2007-10-01, primary completion 2009-07-01, completion 2009-07-01, first posted 2008-05-05, last updated 2009-08-27
Locations	1 site in Austria

-Summary

This is a dose-finding study for the investigational product Lancovutide (Moli1901) in the exploratory phase IIb to establish minimum effective dose, optimal dose, and maximum safe dose. Additionally, the tolerability of Moli1901 shall be investigated.

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+Detailed description

Study Moli1901-010B is a multi center, parallel group, placebo controlled, double-blind efficacy and safety evaluation of three different dosage schedules of aerosolized Moli1901 in adolescents (12 years of age or older), and adults. Study will start with a screening period (visit 1) followed by an 8 weeks double blind comparative treatment period (visit 2-7). Thereafter, subjects will be observed for additional 4 weeks without treatment (follow-up period and visit 8). Spirometry, pulse oximetry and other safety measurements will be performed up to 120 min after study medication inhalation. The study will be conducted in 30 sites in 9 European countries.

+Arms and interventions

Arm	Type	Interventions	Description
1	Experimental	Drug: Moli1901	daily inhalation
2	Experimental	Drug: Moli1901	inhalation every other day
3	Experimental	Drug: Moli1901	inhalation twice a week
4	Placebo comparator	Drug: Moli1901 placebo	daily inhalation

Moli1901 (Drug), also known as Lancovutide, 2622U90, duramycin: 2,5 ml inhalation solution, 8 weeks treatment period
Moli1901 placebo (Drug): 2,5 ml placebo solution, 8 weeks treatment period

+Outcome measures

Primary outcomes

The change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value [at every study visit]

Secondary outcomes

The ensemble of two subject reported clinical scores, which are physical dimensions of the self-report, disease-specific quality of life test, the cystic fibrosis questionnaire in the revised version [every 2 weeks]

+Eligibility

Sex: All, ages: 12 Years to any

Inclusion Criteria:

* Body mass index
* Confirmed diagnosis of cystic fibrosis
* FEV1 between 50% and 85% of predicted
* Oxygen saturation level measured by pulse oximetry (SpO2) >90 % on room air

Exclusion Criteria:

* Bronchial hyperresponsiveness
* Unstable lung function
* Pulmonary disease such as pneumonia, tuberculosis, or lung cancer
* Acute upper respiratory tract infection within the last 2 weeks
* Acute lower respiratory tract infection (requiring antibiotics or hospitalization) within the last 4 weeks
* Pulmonary exacerbation within the last 4 weeks
* Changes from routine maintenance therapy within the last 4 weeks
* Scheduled changes to inhaled antibiotics regimen during the course of the study
* Receive or are planned to receive any treatment via "on-off" regimen (e.g. Tobramycin - TOBI®); last dose of any "on-off" treatment within the last 6 weeks
* Any clinically significant liver, renal, cardiac, neurological, or hematological disease
* ABPA or colonization with Burkholderia cepacia
* Poorly controlled diabetes mellitus

+Locations (1)

Facility	City	Country	Status
Medical University	Graz	Austria

+Publications

Grasemann H, Stehling F, Brunar H, Widmann R, Laliberte TW, Molina L, Doring G, Ratjen F. Inhalation of Moli1901 in patients with cystic fibrosis. Chest. 2007 May;131(5):1461-6. doi: 10.1378/chest.06-2085. PMID 17494794
Zeitlin PL, Boyle MP, Guggino WB, Molina L. A phase I trial of intranasal Moli1901 for cystic fibrosis. Chest. 2004 Jan;125(1):143-9. doi: 10.1378/chest.125.1.143. PMID 14718433
Rickert DE, Dingley K, Ubick E, Dix KJ, Molina L. Determination of the tissue distribution and excretion by accelerator mass spectrometry of the nonadecapeptide 14C-Moli1901 in beagle dogs after intratracheal instillation. Chem Biol Interact. 2005 Jun 30;155(1-2):55-61. doi: 10.1016/j.cbi.2005.04.002. PMID 15893299
McNulty MJ, Hutabarat RH, Findlay JW, Devereux K, Knick VC, Harvey RJ, Molina L. Pharmacokinetics and tissue distribution of the nonadecapeptide Moli1901 in rats and mice. Xenobiotica. 2003 Feb;33(2):197-210. doi: 10.1080/0049825021000022320. PMID 12623761
Zebedin E, Koenig X, Radenkovic M, Pankevych H, Todt H, Freissmuth M, Hilber K. Effects of duramycin on cardiac voltage-gated ion channels. Naunyn Schmiedebergs Arch Pharmacol. 2008 Mar;377(1):87-100. doi: 10.1007/s00210-007-0248-5. Epub 2008 Jan 5. PMID 18176799
Results publication: Eber E, Trawinska-Bartnicka M, Sands D, Bellon G, Mellies U, Bolbas K, Quattrucci S, Mazurek H, Widmann R, Schoergenhofer C, Jilma B, Ratjen F. Aerosolized lancovutide in adolescents (>/=12 years) and adults with cystic fibrosis - a randomized trial. J Cyst Fibros. 2021 Jan;20(1):61-67. doi: 10.1016/j.jcf.2020.08.014. Epub 2020 Sep 1. PMID 32888826

+Investigators

Ernst Eber, Prof Dr, Principal investigator, Medical University of Graz