Trial Outcomes & Findings for Synergistic Enteral Regimen for Treatment of the Gangliosidoses (NCT NCT02030015)

NCT ID: NCT02030015

Last Updated: 2021-04-14

Results Overview

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.

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 16 participants
• Primary outcome timeframe: 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
• Results posted on: 2021-04-14

Participant Flow

Participant milestones

Measure	Syner-G Therapy Regimen 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 Study STARTED	16
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	16

Reasons for withdrawal

Measure	Syner-G Therapy Regimen 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 Study Withdrawal by Subject	3
Overall Study Death	13

Baseline Characteristics

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Baseline characteristics by cohort

Measure	Syner-G Therapy Regimen n=16 Participants 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.
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 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

Population: No participants completed the trial. Outcome measure data was not collected due to small sample size.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Upon Enrollment, and thereafter at 12, 24, 36, 48 and 60 months post-enrollment

Population: No participants completed the trial. Outcome measure data was not collected due to small sample size.

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.

Outcome measures

Outcome data not reported

Adverse Events

Syner-G Therapy Regimen

Serious events: 0 serious events

Other events: 0 other events

Deaths: 13 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Jeanine Jarnes, PharmD

University of Minnesota

Phone: 612-626-5131

Email: utzx0002@umn.edu

Results disclosure agreements

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