Clinical trial NCT01026857: Propionyl-L-Carnitine in Ulcerative Colitis
Official title: Phase II, Parallel-group, Placebo Controlled, Double-blind, Randomised, Multicenter Study to Investigate the Efficacy of Two Dosages of Propionyl-L-Carnitine Colon Release Tablets in Patients Affected by Ulcerative Colitis Under Oral Stable Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Ulcerative Colitis
Lead sponsor: sigma-tau i.f.r. S.p.A.
Sponsor class: INDUSTRY
First posted: 2009-12-07
Enrollment: 121
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether Propionyl-L-carnitine administration may ameliorate the illness in patients affected by mild to moderate ulcerative colitis already in treatment with one of the standard treatments (corticosteroids excluded).

Detailed description: The trial foresees the administration of 2 different dosages of Propionyl-L-carnitine (ST261) colon release tablets, 1 g/die and 2 g/die. The primary objective of this trial is to evaluate the clinical/endoscopic response defined as a lowering of the Disease Activity Index (DAI score) of at least 3 points ot the reaching of remission, the clinical/endoscopic remission (DAI score \<= 2 with no individual subscore \> 1) and histological efficacy of the two combined dosages of PLC (i.e. PLC 1 g/die + 2 g/die) in comparison to placebo, defined as an improvement of the Histological Index (HI) of at least 1 point at the end of the study (a final HI score of \<= 1 will be defined as an histological remission).

The secondary objective is to evaluate the clinical/endoscopic and histological efficacy of the two dosages of PLC individually considered in comparison to Placebo, in order to gather information on the dosage to be used in subsequent trials. The safety and tolerability of the regimens studied will also be objectives of the study.

Eligibility criteria: Inclusion Criteria:

* Have read the Information for the Patient and signed the Informed Consent Form.
* Age comprised between 18 and 75 included.
* If female, not pregnant or nursing.
* For women of childbearing potential, willingness to avoid a pregnancy during the treatment period and for at least 1 month from the last dose of drug.
* Availability of a pancolonoscopy and histology both confirming the diagnosis of active ulcerative colitis.
* Disease Activity Index comprised between 3 and 10, inclusive, (mild to moderate ulcerative colitis).
* On one of the following treatments for ulcerative colitis prior to baseline visit:

  1. Stable background oral aminosalicylates (mesalazine, balsalazide, olsalazine) or sulfasalazine therapy for greater than or equal to 4 weeks prior to baseline assessments.
  2. Stable background mercaptopurine or azathioprine for greater than or equal to 12 weeks prior to baseline assessments.

Exclusion Criteria:

* First diagnosis of ulcerative colitis.
* Crohn's disease.
* Current or previous (in the last 10 days preceding the screening) use of systemic corticosteroids.
* Use of antibiotics in the last 10 days preceding the screening.
* Use of NSAID's in the last 10 days preceding the screening.
* Use of probiotics in the last 10 days preceding the screening
* Positive stool culture (when performed, according to Investigator's judgment, to assess possible parasitologic infection(s)).
* Significantly impaired liver, renal, pulmonary or cardiovascular function.
* History of colon resection.
* Diverticulitis.
* Diagnosis of proctitis
* Stable rectally administered therapy in the last 10 days.
* Active or chronic infection(s).
* Simultaneous participation in another clinical trial, or participation in any clinical trial involving investigational drugs within 3 months from enrolment into the present study.
* Any physical or psychological condition in a patient that could let the investigator suspect his/her poor compliance.

Primary outcomes:
measure: Disease Activity Index score
timeFrame: baseline, week 4
Secondary outcomes:
measure: Adverse Events
timeFrame: Throughout the study
Other outcomes:
Locations:
city: Forlì
country: Italy
facility: Hospital Morgagni - Pierantoni
city: Milan
country: Italy
facility: Ospedale "L. Sacco"
city: Naples
country: Italy
facility: University Federico II
city: Rome
country: Italy
facility: Hospital S. Filippo Neri
city: Rome
country: Italy
facility: Policlinico "A. Gemelli"
city: Kaunas
country: Lithuania
facility: Kaunas Medical University Clinic
city: Vilnius
country: Lithuania
facility: Vilnius University Santariskiu Hospital
city: Lodz
country: Poland
facility: Klinika Przewodu Pokarmowego Szpital Kliniczny Nr 1
city: Wroclaw
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny Nr 3 Katedra i Klinika Gastroenterologii AM
city: Wroclaw
country: Poland
facility: Wojewódzki Szpital Specjalistyczny Klinika Gastroenterologii
city: Moscow
country: Russian Federation
facility: Federal State Institution "7-th Central Military Clinical Aviation Hospital of Ministry of Defence of Russia"
city: Moscow
country: Russian Federation
facility: Federal State Institution "State Scientific Centre of Coloproctology of Rosmedtechnologij"
city: Saint Petersburg
country: Russian Federation
facility: State Institution "Regional Military Clinical Hospital № 442 named after Z.P. Solovyov of Ministry of Defence of Russia"
city: Saint Petersburg
country: Russian Federation
facility: Federal State Healthcare Institution "Clinical Hospital № 122 named after L.G. Sokolov of Federal Medical and Biological Agency"
city: Saint Petersburg
country: Russian Federation
facility: St-Petersburg State Healthcare Institution "Municipal Hospital № 26"
Study officials:
affiliation: Policlinico Universitario "A. Gemelli" - Rome - Italy
name: Giovanni B. Gasbarrini, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Ospedale "L. Sacco" - Milan - Italy
name: Gabriele Bianchi Porro, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: A.O. Universitaria Federico II - Naples - Italy
name: Agesilao D'Arienzo, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: P.O. Morgagni Pierantoni - Forlì - Italy
name: Daniela Valpiani, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Ospedale "S. Filippo Neri" - Rome - Italy
name: Maurizio Koch, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Klinika Przewodu Pokarmowego Szpital Kliniczny Nr 1 - Lodz - Poland
name: Ewa Malecka-Panas, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Samodzielny Publiczny Szpital Kliniczny Nr 3 - Wroclaw - Poland
name: Leszek Paradowski, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Wojewodzki Szpital - Wroclaw - Poland
name: Konrad Lesniakowski, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Kaunas Medical University Clinic - Kaunas - Lithuania
name: Limas Kupcinskas, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Vilnius University Santariskiu Hospital - Vilnius - Lithuania
name: Goda Denapiene, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: State Institution "Regional Military Clinical Hospital N°422 named after Solovyov of Ministry of Defence of Russia - Saint Petersburg - Russia
name: Vladimir B. Grinevich, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Federal State Healthcare Institution "Clinical Hospital N°122 named after L.G. Sokolov of Federal Medical and Biological Agency" - Saint Petersburg - Russia
name: Elena Sishkova, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: St Petersburg State Healthcare Institution "Municipal Hospital N°26" - Saint Petersburg - Russia
name: Konstantin P. Zhidkov, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Federal State Institution "7th Central Military Clinical Aviation Hospital of Ministry of Defence of Russia" - Moscow - Russia
name: Igor G. Bakulin, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Federal State Institution "State Scientific Centre of Coloproctology of Rosmedtechnologij" - Moscow - Russia
name: Tatiana L. Mikhailova, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Mikhailova TL, Sishkova E, Poniewierka E, Zhidkov KP, Bakulin IG, Kupcinskas L, Lesniakowski K, Grinevich VB, Malecka-Panas E, Ardizzone S, D'Arienzo A, Valpiani D, Koch M, Denapiene G, Vago G, Fociani P, Zerbi P, Ceracchi M, Camerini R, Gasbarrini G. Randomised clinical trial: the efficacy and safety of propionyl-L-carnitine therapy in patients with ulcerative colitis receiving stable oral treatment. Aliment Pharmacol Ther. 2011 Nov;34(9):1088-97. doi: 10.1111/j.1365-2036.2011.04844.x. Epub 2011 Sep 19.