Trial record 1 of 1 for: CellCept (mycophenolate) | Batten Disease

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Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)

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ClinicalTrials.gov Identifier: NCT01399047

Recruitment Status : Completed

First Posted : July 21, 2011

Results First Posted : January 23, 2017

Last Update Posted : May 21, 2019

Sponsor:

Collaborator:

Batten Disease Support and Research Assocation (BDSRA)

Information provided by (Responsible Party):

Erika Augustine, University of Rochester

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition	Juvenile Neuronal Ceroid Lipofuscinosis
Interventions	Drug: Mycophenolate mofetil Drug: Liquid Placebo
Enrollment	19

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Group A: Mycophenolate to Placebo Crossover	Group B: Placebo to Mycophenolate Crossover
Arm/Group Description	Subjects received one treatment per...	Subjects received one treatment per...
Arm/Group Description	Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.	Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.
Period Title: Overall Study
Started	10	9
Completed	10	8
Not Completed	0	1
Reason Not Completed
Withdrawal by Subject	0	1

Baseline Characteristics

Arm/Group Title		Group A: Mycophenolate to Placebo Crossover	Group B: Placebo to Mycophenolate Crossover	Total
Arm/Group Description		Subjects received one treatment per...	Subjects received one treatment per...	Total of all reporting groups
Arm/Group Description		Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.	Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.	Total of all reporting groups
Overall Number of Baseline Participants		10	9	19
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	10 participants	9 participants	19 participants
		10.50 (5.99)	14.78 (2.44)	12.53 (5.04)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	10 participants	9 participants	19 participants
	Female	2 20.0%	2 22.2%	4 21.1%
	Male	8 80.0%	7 77.8%	15 78.9%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants
Race	Number Analyzed	10 participants	9 participants	19 participants
	White/Caucasian	9 90.0%	8 88.9%	17 89.5%
	More than one Race	1 10.0%	1 11.1%	2 10.5%
Region of Enrollment Measure Type: Number Unit of measure: Participants
United States	Number Analyzed	10 participants	9 participants	19 participants
United States		10	9	19

Outcome Measures

1.Primary Outcome


Title	Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose
Description	The primary outcome measure is tolerability, defined as the completion...
Description	The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
Time Frame	Baseline to 8 weeks

Outcome Measure Data

Analysis Population Description

All subjects.


Arm/Group Title	Mycophenolate	Placebo
Arm/Group Description:	Includes data from all subjects whi...	Includes data from all subjects whi...
Arm/Group Description:	Includes data from all subjects while on mycophenolate, regardless of treatment order.	Includes data from all subjects while on placebo, regardless of treatment order.
Overall Number of Participants Analyzed	19	19
Measure Type: Count of Participants Unit of Measure: Participants
	17 89.5%	19 100.0%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Mycophenolate, Placebo
	Comments	The primary outcome variable was tolerability, defined as the proportion of subjects able to complete 8 weeks on the assigned treatment. Tolerability was compared among the treatment groups using Prescott's test. A 95% confidence interval was computed for the tolerability of mycophenolate, placebo, and their difference.
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.21
	Comments	[Not Specified]
	Method	Prescott's test
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Risk Difference (RD)
	Estimated Value	0.105
	Confidence Interval	(2-Sided) 95% -0.033 to 0.243
	Estimation Comments	Mycophenolate was tolerated in 17/19 subjects (89.5%, 95% CI: 66.9% - 98.7%), while placebo was tolerated in 19/19 subjects (100%, 95% CI: 82.4% - 100%). The difference in tolerability rates was 10.5% (95% CI: -3.3% - 24.3%, p=0.21).

2.Secondary Outcome


Title	Unified Batten Disease Rating Scale Physical Subscale Change
Description	The Unified Batten Disea...
Description	The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome. The overall treatment effect was determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Time Frame	Baseline to 8 weeks

Outcome Measure Data

Analysis Population Description

All subjects who completed 8 weeks of the respective treatment arm assignment.


Arm/Group Title	Mycophenolate	Placebo
Arm/Group Description:	All data from subjects while on myc...	All data from subjects while on pla...
Arm/Group Description:	All data from subjects while on mycophenolate, regardless of treatment order.	All data from subjects while on placebo, regardless of treatment order.
Overall Number of Participants Analyzed	17	18
Mean (95% Confidence Interval) Unit of Measure: units on a scale
	-1.18 (-4.39 to 2.04)	2.17 (-1.02 to 5.35)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Mycophenolate, Placebo
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.72
	Comments	[Not Specified]
	Method	Prescott's test
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-0.69
	Confidence Interval	(2-Sided) 95% -4.67 to 3.28
	Estimation Comments	[Not Specified]

3.Secondary Outcome


Title	Unified Batten Disease Rating Scale Seizure Subscale Change
Description	The Unified Batten Disea...
Description	The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome. The overall treatment effect was determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Time Frame	Baseline to 8 weeks

Outcome Measure Data

Analysis Population Description

All subjects who completed 8 weeks of the respective treatment arm assignment.


Arm/Group Title	Mycophenolate	Placebo
Arm/Group Description:	All data from subjects while on myc...	All data from subjects while on pla...
Arm/Group Description:	All data from subjects while on mycophenolate, regardless of treatment order.	All data from subjects while on placebo, regardless of treatment order.
Overall Number of Participants Analyzed	17	18
Mean (95% Confidence Interval) Unit of Measure: units on a scale
	-0.24 (-1.15 to 0.68)	-1.44 (-3.03 to 0.14)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Mycophenolate
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.78
	Comments	[Not Specified]
	Method	Prescott's test
	Comments	[Not Specified]

4.Secondary Outcome


Title	Unified Batten Disease Rating Scale Behavior Subscale Change
Description	The Unified Batten Disea...
Description	The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome. The overall treatment effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Time Frame	Baseline to 8 weeks

Outcome Measure Data

Analysis Population Description

All subjects who completed 8 weeks of the respective treatment arm assignment.


Arm/Group Title	Mycophenolate	Placebo
Arm/Group Description:	All data from subjects while on myc...	All data from subjects while on pla...
Arm/Group Description:	All data from subjects while on mycophenolate, regardless of treatment order.	All data from subjects while on placebo, regardless of treatment order.
Overall Number of Participants Analyzed	17	18
Mean (95% Confidence Interval) Unit of Measure: units on a scale
	-1.28 (-4.12 to 1.56)	-0.37 (-2.94 to 2.20)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Mycophenolate
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.98
	Comments	[Not Specified]
	Method	Prescott's test
	Comments	[Not Specified]

5.Secondary Outcome


Title	Unified Batten Disease Rating Scale Capability Subscale Change
Description	The Unified Batten Disea...
Description	The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome. The overall treatment effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Time Frame	Baseline to 8 weeks

Outcome Measure Data

Analysis Population Description

All subjects who completed 8 weeks of the respective treatment arm assignment.


Arm/Group Title	Mycophenolate	Placebo
Arm/Group Description:	All data from subjects while on myc...	All data from subjects while on pla...
Arm/Group Description:	All data from subjects while on mycophenolate, regardless of treatment order.	All data from subjects while on placebo, regardless of treatment order.
Overall Number of Participants Analyzed	17	18
Mean (95% Confidence Interval) Unit of Measure: units on a scale
	0.33 (-0.18 to 0.85)	0.47 (-0.02 to 0.97)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Mycophenolate
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.26
	Comments	[Not Specified]
	Method	Wilcoxon (Mann-Whitney)
	Comments	[Not Specified]

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).

Arm/Group Title	Mycophenolate		Placebo
Arm/Group Description	Includes data from all subjects whi...		Includes data from all subjects whi...
Arm/Group Description	Includes data from all subjects while on mycophenolate, regardless of order.		Includes data from all subjects while on placebo, regardless of order.
All-Cause Mortality
	Mycophenolate		Placebo
	Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--
Serious Adverse Events Serious Adverse Events
	Mycophenolate		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/19 (5.26%)		0/19 (0.00%)
Metabolism and nutrition disorders
Hyperkalemia ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
† Indicates events were collected by systematic assessment 1 Term from vocabulary, MedDRA
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Mycophenolate		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	14/19 (73.68%)		17/19 (89.47%)
Cardiac disorders
ECG Abnormality ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Ear and labyrinth disorders
Haemotympanum ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Ear pain ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Endocrine disorders
Weight decreased ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Eye disorders
Photophobia ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Gastrointestinal disorders
Vomiting ^† 1	6/19 (31.58%)	8	2/19 (10.53%)	3
Diarrhea ^† 1	3/19 (15.79%)	3	2/19 (10.53%)	2
Abdominal pain upper ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Gastroesophageal reflux disease ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Retching ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Abdominal Discomfort ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Abdominal Pain ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
General disorders
Oedema peripheral ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Chest pain ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Fatigue ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Infections and infestations
Otitis media ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Strep pharyngitis ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Sinusitis ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Upper Respiratory Infection ^† 1	2/19 (10.53%)	2	0/19 (0.00%)	0
Conjunctivitis ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Gingivitis ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Ear Infection ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Otitis externa ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Investigations
Blood prolactin increased ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Weight increased ^† 1	2/19 (10.53%)	2	1/19 (5.26%)	1
Blood calcium decreased ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Cardiac murmur ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Electrocardiogram abnormality ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Electrocardiogram T wave inversion ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Protein urine present ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Metabolism and nutrition disorders
Decreased appetite ^† 1	0/19 (0.00%)	0	2/19 (10.53%)	2
Hypoglycemia ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Musculoskeletal and connective tissue disorders
Arthralgia ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Muscle Stiffness ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Muscle twitching ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Back pain ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Musculoskeletal pain ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Pain in extremity ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Nervous system disorders
Headache ^† 1	3/19 (15.79%)	3	3/19 (15.79%)	3
Tremor ^† 1	2/19 (10.53%)	2	0/19 (0.00%)	0
Dyskinesia ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Insomnia ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Aphasia ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Convulsion ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Disorientation ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Dysarthria ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Motor dysfunction ^† 1	0/19 (0.00%)	0	2/19 (10.53%)	2
Movement disorder ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Seizure ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Speech disorder ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Dizziness ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Ataxia ^† 1	2/19 (10.53%)	2	0/19 (0.00%)	0
Psychiatric disorders
Abnormal behavior ^† 1	1/19 (5.26%)	1	1/19 (5.26%)	1
Mental status changes ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Mood altered ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Aggression ^† 1	0/19 (0.00%)	0	2/19 (10.53%)	2
Anger ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Anxiety ^† 1	0/19 (0.00%)	0	2/19 (10.53%)	2
Confusional state ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Irritability ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Nightmare ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Perseveration ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Renal and urinary disorders
Haematuria ^† 1	1/19 (5.26%)	1	0/19 (0.00%)	0
Urinary incontinence ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
Respiratory, thoracic and mediastinal disorders
Cough ^† 1	3/19 (15.79%)	3	1/19 (5.26%)	1
Oropharyngeal pain ^† 1	1/19 (5.26%)	1	2/19 (10.53%)	2
Rhinorrhea ^† 1	0/19 (0.00%)	0	1/19 (5.26%)	1
† Indicates events were collected by systematic assessment 1 Term from vocabulary, MedDRA

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Erika Augustine, MD, MS
Organization:	University of Rochester Medical Center
Phone:	585-275-2808
EMail:	erika_augustine@urmc.rochester.edu

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Adams HR, Defendorf S, Vierhile A, Mink JW, Marshall FJ, Augustine EF. A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder. Clin Trials. 2019 Oct;16(5):555-560. doi: 10.1177/1740774519855715. Epub 2019 Jun 11.

Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013 Sep;28(9):1142-50. doi: 10.1177/0883073813495959.

Layout table for additonal information

Responsible Party:	Erika Augustine, University of Rochester
ClinicalTrials.gov Identifier:	NCT01399047
Other Study ID Numbers:	3908
First Submitted:	July 5, 2011
First Posted:	July 21, 2011
Results First Submitted:	November 28, 2016
Results First Posted:	January 23, 2017
Last Update Posted:	May 21, 2019

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