Clinical trial NCT01171807
Conditions: Ulcerative Colitis
Keywords: Ulcerative colitis Steroid-dependency Steroid adverse events Erythrocytes
Sponsor: Quince Therapeutics S.p.A.
Start date: 2003-07-22; Completion date: 2007-05-15
Enrollment: 33 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
Objectives:

The primary objective of this trial was to evaluate the patients response rate at the end of the study.

Patients were considered responder if one of the following conditions occurs:

* Disease remission (Powell Tuck ≤ 3 or CDAI \< 150) and withdrawal of oral steroids therapy from at least the second treatment procedure;
* Disease marked improvement versus basal conditions (at least 5 point decrease in Powell Tuck index or 150 point decrease in CDAI score) and withdrawal of oral steroids therapy from at least the second treatment procedure.

Secondary objectives:

* to evaluate the endogenous cortisole production after receiving the study treatment
* to evaluate the inflammatory indexes (ESR and CPR) after receiving the study treatment
* to evaluate the endoscopic remission in patients suffering from mesalazine refractory Ulcerative Colitis
* to evaluate the safety of dexamethasone intra-erythrocyte therapy with particular attention to steroid-related adverse events.

Detailed description:
This was a single-center, placebo-controlled, randomised, phase II explorative study with the aim to investigate the ability of the new steroid delivery system to induce or maintain remission in steroid-dependent or mesalazine refractory patients suffering from Chron's disease (CD) or Ulcerative Colitis (UC) .

Once the patient was deemed eligible for the study, the treatment plan was selected as follows

In the Dexamethasone arm (DEX 21-P):

* steroid-dependant patients: one treatment procedure every 30 days up to a total of 6 procedures
* mesalazine refractory active UC patients: one treatment procedure every 15 days up to a total of 3 procedures.

In the placebo arm:

Patients assigned to placebo arm performed the same procedure as the patients assigned to the DEX 21-P group without loading in the Red Blood Cells the Dex 21-P.

The planned duration of individual patient participation in the study was a maximum of 6 or 28 weeks, depending from the assigned treatment scheme.

Arms: arm 1: In this arm a dose of 20 ml of Dex 2-P solution was administered every 15 or 30 days for a total of 3 or 6 treatment procedures, respectively.

Specifically, steroid-dependant IBD patients had to undergo a total of 6 procedures at one month interval, while active mesalazine refractory UC patients had to undergo a total of 3 procedures at 15 days interval.

Every procedure implies the collection and re-infusion of autologous erythrocytes previously loaded with Dex 21-P. arm 2: In this arm placebo solution was administered every 15 or 30 days for a total of 3 or 6 treatment procedures, respectively.

Specifically, steroid-dependant IBD patients had to undergo a total of 6 procedures at one month interval, while active mesalazine refractory UC patients had to undergo a total of 3 procedures at 15 days interval.

Every procedure implies the collection and re-infusion of autologous erythrocytes previously NOT loaded with Dex21-P.
Interventions: intervention 1: Dex 21-P intervention 2: Placebo
Intervention descriptions: intervention 1: At each procedure 50 ml of patient whole blood was washed with saline solution and centrifugated. The isolated erythrocytes were suspended into 2 hypotonic solutions to make their membrane permeable and incubated with Dex 21-P sodium salt up to obtain a final concentration of 10 mM. The drug loaded erythrocytes were immediately re-infused by using a suitable filter. intervention 2: Patients assigned to placebo arm performed the same procedure as the patients assigned to the DEX 21-P group without loading in the Red Blood Cells the Dex 21-P

Protocol documents: