Study of orBec® as Monotherapy in the Treatment of Patients With Upper GI Symptoms Caused by Chronic Graft Versus Host Disease (GVHD)

• Sponsor: Soligenix (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT01925950
• Collaborator: (none)
• Enrollment: 2
• Locations: 3
• Arms: 2
• Duration (Months): 17
• Patients Per Site: 0.7
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

Use of an oral topically-active glucocorticoid with limited side effects may control the gastrointestinal inflammatory process of GVHD and minimize glucocorticoid exposure.

Condition or Disease	Intervention/Treatment	Phase
Chronic Gastrointestinal Graft vs Host Disease	Drug: orBec; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Safety and Efficacy of orBec® (Oral Beclomethasone 17,21-Dipropionate) Administered as Monotherapy in the Treatment of Upper Gastrointestinal (GI) Symptoms Caused by GVHD in Patients With Chronic GI GVHD
• Study Start Date: Dec 1, 2013
• Actual Primary Completion Date: May 1, 2015
• Actual Study Completion Date: May 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Control	Drug: Placebo
Experimental: orBec Investigational drug	Drug: orBec; Other Names: oral BDP oral beclomethasone 17,21-dipropionate

Outcome Measures

Primary Outcome Measures

1. Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms [16 weeks]

   Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Receipt of allogeneic hematopoietic cell transplant >100 days prior to consent

• Documented cGVHD as defined by the NIH consensus criteria in at least one organ system other than the GI tract (for example, cGVHD of the oral cavity would qualify as a criterion)

• Endoscopic findings consistent with GI GVHD

• Must be able to swallow tablets

• Must be able to read and understand informed consent

• Adequate birth control methods for the duration of the study

Exclusion Criteria:

• 500 mL/day of diarrhea on any 1 day within 3 days prior to the first dose of study drug

• GI infection

• Multi-organ failure or other condition that, in the opinion of the investigator, would compromise the patient's ability to complete the study.

• HIV seropositivity

• Pregnant or nursing female

• Use of any investigational drug to treat chronic GVHD within 28 days of the first dose of study drug

• Evidence of recurrent or progressing malignant disorder that was the indication for HCT

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	John Theurer Cancer Center	Hackensack	New Jersey	United States	07601
2	Roswell Park Cancer Institute	Buffalo	New York	United States	14263
3	Vanderbilt-Ingram Cancer Center	Nashville	Tennessee	United States	37232

Sponsors and Collaborators

• Soligenix

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Soligenix
• ClinicalTrials.gov Identifier: NCT01925950
• Other Study ID Numbers: BDP-GVHD-08
• First Posted: Aug 20, 2013
• Last Update Posted: Nov 1, 2018
• Last Verified: Oct 1, 2018

• Keywords provided by Soligenix: BDP; orBec; GVHD; beclomethasone dipropionate; bone marrow transplant; hematopoietic cell transplant; HCT; stem cell transplant; marrow transplant; SCT; beclomethasone 17,21-dipropionate
• Additional relevant MeSH terms: Graft vs Host Disease; Beclomethasone

Study Results

Participant Flow

Arm/Group Title	Placebo	orBec
Arm/Group Description	Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. Part 1: 2 mg of placebo 4 times per day for up to 16 weeks. Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule: Weeks 17-24: 2 mg placebo 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg placebo 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg placebo daily, up to 16 weeks.	orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. Part 1: 2 mg of orBec 4 times per day for up to 16 weeks. Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule: Weeks 17-24: 2 mg orBec 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg orBec 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg orBec daily, up to 16 weeks.
Period Title: Part 1
STARTED	1	1
COMPLETED	1	1
NOT COMPLETED	0	0
Period Title: Part 1
STARTED	1	0
COMPLETED	0	0
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Placebo	orBec	Total
Arm/Group Description	Control Placebo	Investigational drug orBec	Total of all reporting groups
Overall Participants	1	1	2
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	1 100%	0 0%	1 50%
>=65 years	0 0%	1 100%	1 50%
Sex: Female, Male (Count of Participants)
Female	1 100%	0 0%	1 50%
Male	0 0%	1 100%	1 50%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%
White	1 100%	1 100%	2 100%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	1 100%	1 100%	2 100%

Outcome Measures

1. Primary Outcome

Title	Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms
Description	Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0.
Time Frame	16 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	orBec
Arm/Group Description	Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. 2 mg of placebo 4 times per day for up to 16 weeks.	orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. 2 mg of orBec 4 times per day for up to 16 weeks.
Measure Participants	1	1
Count of Participants [Participants]	1 100%	0 0%

Adverse Events

Time Frame	48-week study period
Arm/Group Title	Placebo		orBec
Arm/Group Description	Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. Part 1: 2 mg of placebo 4 times per day for up to 16 weeks. Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule: Weeks 17-24: 2 mg placebo 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg placebo 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg placebo daily, up to 16 weeks.		orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug. Part 1: 2 mg of orBec 4 times per day for up to 16 weeks. Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule: Weeks 17-24: 2 mg orBec 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg orBec 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg orBec daily, up to 16 weeks.
All Cause Mortality
	Placebo		orBec
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Placebo		orBec
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/1 (100%)		0/1 (0%)
General disorders
intractable pain	1/1 (100%)	1	0/1 (0%)	0
Hepatobiliary disorders
biliary strictures	1/1 (100%)	1	0/1 (0%)	0
Infections and infestations
fungal pneumonia	1/1 (100%)	1	0/1 (0%)	0
Respiratory, thoracic and mediastinal disorders
shortness of breath	1/1 (100%)	1	0/1 (0%)	0
Vascular disorders
pulmonary emboli	1/1 (100%)	1	0/1 (0%)	0
Other (Not Including Serious) Adverse Events
	Placebo		orBec
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/1 (0%)		0/1 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.