Trial Outcomes & Findings for Intra-erythrocyte Dexamethasone Versus Placebo in Patients With Steroid-dependent Crohn's Disease (NCT NCT01277289)

Last Updated: 2024-06-03

Results Overview

Clinical remission was defined as a Crohn's Disease Activity Index (CDAI) \< 150. A therapy failure was considered to have occurred in patients with a CDAI score over 150 for \> 2 weeks and/or the need for systemic steroids (with / without surgery). Hence, the higher the index, the worse the outcome.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

51 participants

Primary outcome timeframe

after 12 months

Results posted on

2024-06-03

Participant Flow

There were 63 patients enrolled into the screening phase at 10 sites; of these, 51 patients (representing the Safety population) were randomized at 9 sites.

Participant milestones

Participant milestones
Measure	Ery-dex Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Overall Study STARTED	28	23
Overall Study COMPLETED	5	2
Overall Study NOT COMPLETED	23	21

Reasons for withdrawal

Reasons for withdrawal
Measure	Ery-dex Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Overall Study fragile vein	1	0
Overall Study Physician Decision	1	0
Overall Study therapy failure and/or Crohn's surgery	13	16
Overall Study Protocol Violation	1	1
Overall Study Adverse Event	2	1
Overall Study consent withdrawn	1	1
Overall Study premature closure of the study	4	2

Baseline Characteristics

Intra-erythrocyte Dexamethasone Versus Placebo in Patients With Steroid-dependent Crohn's Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ery-dex n=28 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Total n=51 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=93 Participants	20 Participants n=4 Participants	44 Participants n=27 Participants
Age, Categorical >=65 years	4 Participants n=93 Participants	3 Participants n=4 Participants	7 Participants n=27 Participants
Age, Continuous	47.3 years STANDARD_DEVIATION 14.9 • n=93 Participants	43.4 years STANDARD_DEVIATION 12.5 • n=4 Participants	45.5 years STANDARD_DEVIATION 13.9 • n=27 Participants
Sex: Female, Male Female	20 Participants n=93 Participants	7 Participants n=4 Participants	27 Participants n=27 Participants
Sex: Female, Male Male	8 Participants n=93 Participants	16 Participants n=4 Participants	24 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) White	28 Participants n=93 Participants	23 Participants n=4 Participants	51 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Region of Enrollment Romania	1 participants n=93 Participants	0 participants n=4 Participants	1 participants n=27 Participants
Region of Enrollment Italy	27 participants n=93 Participants	22 participants n=4 Participants	49 participants n=27 Participants
Region of Enrollment Spain	0 participants n=93 Participants	1 participants n=4 Participants	1 participants n=27 Participants

PRIMARY outcome

Timeframe: after 12 months

Population: Safety population, defined as all randomized patients who took at least one dose of the study medication.

Outcome measures

Outcome measures
Measure	Ery-dex n=28 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Percentage of Patients Maintaining Steroids-free Clinical Remission (CDAI<150) Without Surgery for 12 Months	5 Participants	2 Participants

SECONDARY outcome

Timeframe: after 12 months

Population: Safety population, defined as all randomized patients who took at least one dose of the study medication.

This evaluation of safety was made by the comparison of treatment emergent adverse events (TEAE). Treatment emergent adverse events (TEAE) are undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment.

Outcome measures

Outcome measures
Measure	Ery-dex n=28 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	13 Participants	6 Participants

SECONDARY outcome

Timeframe: after 12 months

Population: Safety population, defined as all randomized patients who took at least one dose of the study medication.

An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An adverse event (AE) can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Outcome measures

Outcome measures
Measure	Ery-dex n=28 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Percentage of Patients Interrupting the Study Because of Adverse Events	2 Participants	1 Participants

SECONDARY outcome

Timeframe: From end of the steroid therapy to relapse, up to 545 days

Population: Data reported only for 9 subjects for the EryDex group and for 13 subjects in the placebo group of the safety set. For the remaining subjects, data are not included because of no treatment with steroids, or no last date available on the use of steroids, or steroids still ongoing.

The mean time between the end of the steroid therapy and a confirmed relapse of the disease.

Outcome measures

Outcome measures
Measure	Ery-dex n=9 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=13 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Duration of the Period of Steroid-free Clinical Remission	55.3 Days Standard Deviation 73.2	84.9 Days Standard Deviation 72.9

SECONDARY outcome

Timeframe: At Baseline and at the end of the study (18 months)

Population: Safety Set defined as all randomized patients who took at least one dose of the study medication

Levels of serum cortisol were measured at baseline and following Adrenocorticotropic Hormone (ACTH) trigger.

Outcome measures

Outcome measures
Measure	Ery-dex n=28 Participants Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 Participants placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Dosing of Serum Cortisol At Baseline - before ACTH	6.8 μg/dl Standard Deviation 4.6	7.1 μg/dl Standard Deviation 6.2
Dosing of Serum Cortisol At the end of the study - before ACTH	11.3 μg/dl Standard Deviation 8.6	6.4 μg/dl Standard Deviation 5.4
Dosing of Serum Cortisol At baseline - after ACTH	10.3 μg/dl Standard Deviation 7.6	11.0 μg/dl Standard Deviation 6.5
Dosing of Serum Cortisol At the end of the study - after ACTH	18.4 μg/dl Standard Deviation 4.7	12.7 μg/dl Standard Deviation 7.6

Adverse Events

Ery-dex

Serious events: 6 serious events

Other events: 19 other events

Deaths: 0 deaths

Placebo

Serious events: 4 serious events

Other events: 16 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Ery-dex n=28 participants at risk Ery-dex (dexamethasone sodium phosphate)is administered as intra-erythrocyte drug at monthly interval Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months	Placebo n=23 participants at risk placebo comparator (sodium chloride instead of dexamethasone sodium phosphate) is administered in infusion at monthly interval. Dexamethasone: 500 mg/20 ml encapsulated in erythrocytes, every month for 12 months
Gastrointestinal disorders Crohn's disease	7.1% 2/28 • Number of events 2 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	0.00% 0/23 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).
Gastrointestinal disorders Gastrointestinal haemorrhage	3.6% 1/28 • Number of events 1 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	0.00% 0/23 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).
Gastrointestinal disorders Intestinal obstruction	3.6% 1/28 • Number of events 1 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	0.00% 0/23 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).
Gastrointestinal disorders Subileus	3.6% 1/28 • Number of events 1 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	13.0% 3/23 • Number of events 3 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).
Infections and infestations Staphylococcal infection	0.00% 0/28 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	4.3% 1/23 • Number of events 1 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).
Injury, poisoning and procedural complications Spinal fracture	3.6% 1/28 • Number of events 1 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).	0.00% 0/23 • Adverse events were collected from Visit 1 ( Day 15) up to Visit 15 (Day 600) TEAEs were defined as those events with an onset any time following the first dose of trial medication up to 30 days after last dose of trial medication or those starting before the treatment period but continuing and worsening during the treatment period or leading to treatment discontinuation (even if temporary).

Other adverse events

Additional Information

Angelo Andriulli, MD

Erydel

Phone: +39 882 410784

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place