Trial Outcomes & Findings for A Prospective Study on the Tolerability and Efficacy of the de Novo Use of Myfortic in Liver Transplant Recipients (NCT NCT00336817)

NCT ID: NCT00336817

Last Updated: 2017-03-09

Results Overview

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation). The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms. The total GSRS range of scores is 15 to 105 with higher scores meaning the worst of symptoms.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 30 participants
• Primary outcome timeframe: screening, 2, 6 and 12 weeks
• Results posted on: 2017-03-09

Participant Flow

Participant milestones

Measure	Myfortic Group Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Overall Study STARTED	15	15
Overall Study COMPLETED	14	13
Overall Study NOT COMPLETED	1	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Prospective Study on the Tolerability and Efficacy of the de Novo Use of Myfortic in Liver Transplant Recipients

Baseline characteristics by cohort

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days	Total n=29 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants	10 Participants n=7 Participants	22 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Age, Continuous	54.6 years STANDARD_DEVIATION 12.75408 • n=5 Participants	58.2 years STANDARD_DEVIATION 8.83338 • n=7 Participants	56.4333 years STANDARD_DEVIATION 10.92824 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	8 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	7 Participants n=7 Participants	19 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants	15 participants n=7 Participants	30 participants n=5 Participants

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

Population: One participant in the myfortic group was ineligible

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms. The total GSRS range of scores is 15 to 105 with higher scores meaning the worst of symptoms.

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil screening	21.80 units on a scale Standard Deviation 4.37	23.07 units on a scale Standard Deviation 6.04
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil 2 weeks	24.90 units on a scale Standard Deviation 5.47	22.14 units on a scale Standard Deviation 5.08
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil 6 weeks	28.90 units on a scale Standard Deviation 16.10	26.07 units on a scale Standard Deviation 15.72
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil 12 weeks	21.80 units on a scale Standard Deviation 7.30	25.21 units on a scale Standard Deviation 15.95

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.

The abdominal Pain subscale range is 3 to 21 with higher scores means worst symptoms

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale) screening	5.20 units on a scale Standard Deviation 1.81	5.29 units on a scale Standard Deviation 1.63
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale) 2 weeks	5.80 units on a scale Standard Deviation 1.75	4.79 units on a scale Standard Deviation 1.12
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale) 6 weeks	6.40 units on a scale Standard Deviation 3.44	5.93 units on a scale Standard Deviation 4.20
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale) 12 weeks	5.30 units on a scale Standard Deviation 2.67	5.36 units on a scale Standard Deviation 3.41

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.

Reflux subscale range is 2 to 14 with higher scores means worst symptoms

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale) screening	2.70 units on a scale Standard Deviation 1.060	3.07 units on a scale Standard Deviation 1.54
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale) 2 weeks	2.90 units on a scale Standard Deviation 1.66	2.36 units on a scale Standard Deviation 0.75
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale) 6 weeks	2.90 units on a scale Standard Deviation 1.73	3.36 units on a scale Standard Deviation 2.73
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale) 12 weeks	2.70 units on a scale Standard Deviation 1.34	3.07 units on a scale Standard Deviation 1.86

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.

The Indigestion subscale range is 4 to 28 with higher scores means worst symptoms

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale) screening	5.40 units on a scale Standard Deviation 1.174	5.86 units on a scale Standard Deviation 2.68
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale) 2 weeks	5.80 units on a scale Standard Deviation 1.75	6.43 units on a scale Standard Deviation 3.94
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale) 6 weeks	6.50 units on a scale Standard Deviation 3.84	7.50 units on a scale Standard Deviation 5.72
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale) 12 weeks	5.60 units on a scale Standard Deviation 2.75	6.86 units on a scale Standard Deviation 4.94

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.

The Diarrhea subscale range is 3 to 21 with higher scores means worst symptoms

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale) screening	4.00 units on a scale Standard Deviation 1.15	4.79 units on a scale Standard Deviation 1.85
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale) 2 weeks	5.80 units on a scale Standard Deviation 2.78	4.36 units on a scale Standard Deviation 1.39
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale) 6 weeks	6.60 units on a scale Standard Deviation 4.40	4.50 units on a scale Standard Deviation 2.31
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale) 12 weeks	3.70 units on a scale Standard Deviation 0.95	5.29 units on a scale Standard Deviation 3.29

PRIMARY outcome

Timeframe: screening, 2, 6 and 12 weeks

The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).

The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.

Constipation subscale range is 3 to 21 with higher scores means worst symptoms

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale) screening	4.50 units on a scale Standard Deviation 2.01	4.07 units on a scale Standard Deviation 1.63
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale) 2 weeks	4.60 units on a scale Standard Deviation 1.43	4.21 units on a scale Standard Deviation 1.83
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale) 6 weeks	6.50 units on a scale Standard Deviation 4.79	4.79 units on a scale Standard Deviation 3.33
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale) 12 weeks	4.50 units on a scale Standard Deviation 2.12	4.54 units on a scale Standard Deviation 3.41

PRIMARY outcome

Timeframe: 12 weeks

Number of participants with: Thrombocytopenia (<50,000 mm3), Leukopenia (< 2000 mm3), absolute neutrophils count ( <1000 mm3) or hemoglobin ( < 7.0 g/dL)

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Number of Participants With Bone Marrow Suppression	0 participants	0 participants

PRIMARY outcome

Timeframe: 12 weeks

number of participants

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Incidence of Cytomegalovirus Infection or Disease During the Study Period	0 participants	0 participants

SECONDARY outcome

Timeframe: 12 weeks

Drug discontinuation due to drug side effects regarding Cellcept and Myfortic.

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Drug Discontinuation Due to Side Effects	1 Participants	2 Participants

SECONDARY outcome

Timeframe: 12 weeks

Creatinine levels

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Number of Participants With Clinically Significant Decrease in Serum Creatinine From Baseline Through Week 12	0 participants	0 participants

SECONDARY outcome

Timeframe: 12 weeks

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Number of Participants With Neurotoxicity	0 participants	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 12 weeks

number of patients

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Incidence of Graft Loss or Death During the Study Period	0 participants	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 12 weeks

number of patients with ACR

Outcome measures

Measure	Myfortic Group n=14 Participants Subjects in the Myfortic arm will receive Myfortic 360mg or 720 mg BID for 90 days Myfortic: Myfortic 360mg or 720 mg BID for 90 days	CellCept Group n=15 Participants Subjects in the CellCept arm will receive CellCept 500mg or 1000mg BID for 90 days CellCept: CellCept 500mg or 1000mg BID for 90 days
Incidence of Biopsy-proven Acute Cellular Rejection During the Study Period	2 participants	1 participants

Adverse Events

Myfortic Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

CellCept Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Roberto Lopez-Solis

Upittsburgh

Phone: 412-647-5173

Email: lopezrc@upmc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place