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Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02030015
Recruitment Status : Terminated (Efficacy)
First Posted : January 8, 2014
Results First Posted : April 14, 2021
Last Update Posted : April 14, 2021
Sponsor:
Collaborators:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Science (NCATS)
National Institute of Neurological Disorders and Stroke (NINDS)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Lysosomal Disease Network
Information provided by (Responsible Party):
University of Minnesota

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions GM1 Gangliosidoses
GM2 Gangliosidoses
Tay-Sachs Disease
Sandhoff Disease
Interventions Drug: miglustat
Other: Ketogenic Diet
Enrollment 16
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Syner-G Therapy Regimen
Hide Arm/Group Description

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.

miglustat: The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

Ketogenic Diet: The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

Period Title: Overall Study
Started 16
Completed 0
Not Completed 16
Reason Not Completed
Withdrawal by Subject             3
Death             13
Arm/Group Title Syner-G Therapy Regimen
Hide Arm/Group Description

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.

miglustat: The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

Ketogenic Diet: The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
<=18 years
16
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
6
  37.5%
Male
10
  62.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
American Indian or Alaska Native
1
   6.3%
Asian
3
  18.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
  12.5%
White
8
  50.0%
More than one race
2
  12.5%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 16 participants
16
1.Primary Outcome
Title The Duration of Survival of Each Research Subject, Measured in Months and Years
Hide Description The survival duration of patients with infantile and juvenile forms of gangliosidoses will be assessed, in order to judge the clinical impact of the Syner-G therapy regimen. This will be accomplished by recording the subject's age on the date of enrollment in this study, and the subject's age at the conclusion of this study, or on the date of their death, whichever comes first. The duration of each subject's survival, expressed in months and years, will be compared to available natural history data in order to arrive at an expert assessment of the impact of the Syner-G therapy upon patient longevity.
Time Frame From date of enrollment until 60 months thereafter, or the date of subject's death from any cause, whichever comes first, assessed up to 60 months
Hide Outcome Measure Data
Hide Analysis Population Description
No participants completed the trial. Outcome measure data was not collected due to small sample size.
Arm/Group Title Syner-G Therapy Regimen
Hide Arm/Group Description:

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.

miglustat: The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

Ketogenic Diet: The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Rate of Change in Neurocognitive Functioning
Hide Description The Bayley Scales of Infant and Toddler Development and the Vineland Adaptive Behavior Scales will be administered upon enrollment and annually thereafter for five years. Changes in these neurodevelopmental assessments will be evaluated over the duration of follow-up. Ability of the child to have these assessments yearly may be subject to patient's insurance coverage for such assessments.
Time Frame Upon Enrollment, and thereafter at 12, 24, 36, 48 and 60 months post-enrollment
Hide Outcome Measure Data
Hide Analysis Population Description
No participants completed the trial. Outcome measure data was not collected due to small sample size.
Arm/Group Title Syner-G Therapy Regimen
Hide Arm/Group Description:

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.

miglustat: The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

Ketogenic Diet: The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Participants were followed for the length of study participation; up to 60 months or 5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Syner-G Therapy Regimen
Hide Arm/Group Description

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.

miglustat: The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

Ketogenic Diet: The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

All-Cause Mortality
Syner-G Therapy Regimen
Affected / at Risk (%)
Total   13/16 (81.25%) 
Hide Serious Adverse Events
Syner-G Therapy Regimen
Affected / at Risk (%)
Total   0/16 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Syner-G Therapy Regimen
Affected / at Risk (%)
Total   0/16 (0.00%) 
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: Jeanine Jarnes, PharmD
Organization: University of Minnesota
Phone: 612-626-5131
EMail: utzx0002@umn.edu
Other Publications:
Layout table for additonal information
Responsible Party: University of Minnesota
ClinicalTrials.gov Identifier: NCT02030015    
Other Study ID Numbers: Syner_G_Regimen
U54NS065768 ( U.S. NIH Grant/Contract )
1311M46101 ( Other Identifier: Univ. of Minnesota IRB Identifier Number )
First Submitted: December 17, 2013
First Posted: January 8, 2014
Results First Submitted: February 22, 2021
Results First Posted: April 14, 2021
Last Update Posted: April 14, 2021