Trial · NCT02019667
Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
NCT02019667, Phase 2, Completed, National Institute of Neurological Disorders and Stroke (NINDS); results first posted 2020-02-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency. Trials, record NCT02019667. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02019667
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
- NCT number
- NCT02019667
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- Conditions
- Metabolic Disease, Seizures
- Interventions
- SGS-742, Placebo
- First posted
- 2013-12-24
- Last updated
- 2020-02-24
- Results first posted
- 2020-02-24
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline on the Adaptive Behavior Assessment System (ABAS) Test at the End of the Study Drug and Placebo Treatment Periods | baseline and six months | The ABAS questionnaire was completed by the participant's parent or caregiver at the end of each six month treatment period.The ABAS provides a comprehensive picture of adaptive skills across the lifespan. The questionnaire addresses Conceptual, Social and Practical skills including communication, self-direction, use of leisure time, health, safety and self-care. The General Adaptive Composite score ranges from \<40 to \>160 with a lower score representing lower adaptive behavior. The difference between Placebo and Baseline and Study Drug and Baseline were obtained. These values were averaged across individuals to report a mean and a standard deviation of the baseline-to-treatment period change. The means for each treatment can be compared to have a baseline-adjusted treatment effect interpretation. A positive change represents an improvement in adaptive skills compared with baseline and a negative change represents a decline in adaptive skills compared with baseline. |
| Secondary | Change From Baseline of TMS Measurement of Motor Threshold at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | Transcranial Magnetic Stimulation (TMS) is a non-invasive technique which applies magnetic pulses to the brain via a coil inducing an electrical current in the brain. Stimulation is typically applied at a sufficient intensity to trigger action potentials in nearby neurons. The motor threshold is defined as the minimum percentage of the stimulator output that evoked a motor evoked potential of more than 50µV in at least 5 out of 10 trials. Motor threshold was measured at the end of the study drug period and the end of the Placebo period. The differences between Placebo and Baseline, and SGS and Baseline were obtained. A decrease from baseline value indicates increased cortical excitability and an increase from baseline value indicates reduced cortical excitability. These values were averaged across individuals to report a mean and standard deviation of this baseline-to-treatment period change. The mean for each treatment can be compared to have a baseline-adjusted treatment effect. |
| Secondary | Change From Baseline of TMS Measurement of Intracortical Facilitation at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | Transcranial Magnetic Stimulation (TMS) is a non-invasive technique which applies magnetic pulses to the brain via a coil inducing an electrical current in the brain. Stimulation is typically applied at a sufficient intensity to trigger action potentials in nearby neurons. Intracortical facilitation (ICF) and inhibition (ICI) were studied using a paired stimulus paradigm. The motor threshold (MT) was first established. The conditioning stimulus (70% MT) followed by the test stimulus (120% MT) was delivered at an interstimulus interval (ISI) of 10 ms for ICF. Each run consisted of 10 trials, and the amplitude ratio of the mean conditioned Motor Evoked Potential (MEP) to control MEP was determined. A larger amplitude ratio indicates greater cortical excitability. The differences between Placebo and Baseline, and SGS and Baseline were obtained. These values were averaged across individuals to report a mean. |
| Secondary | Change From Baseline of TMS Measurement of Short Interval Intracortical Inhibition (Short ICI) at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | Transcranial Magnetic Stimulation (TMS) is a non-invasive technique which applies magnetic pulses to the brain via a coil inducing an electrical current in the brain. Stimulation is typically applied at a sufficient intensity to trigger action potentials in nearby neurons. Intracortical facilitation and inhibition were studied using a paired stimulus paradigm. The motor threshold (MT) was first established. The conditioning stimulus (70% MT) followed by the test stimulus (120% MT) was delivered at an interstimulus interval (ISI) of 2 ms for short ICI. Each run consisted of 10 trials, and the amplitude ratio of the mean conditioned Motor Evoked Potential (MEP) to control MEP was determined. A larger amplitude ratio indicates greater cortical excitability. The differences between Placebo and Baseline, and SGS and Baseline were obtained. These values were averaged across individuals to report a mean. |
| Secondary | Change From Baseline of TMS Measurement of Long Interval Intracortical Inhibition (Long ICI) at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | Transcranial Magnetic Stimulation (TMS) is a non-invasive technique which applies magnetic pulses to the brain via a coil inducing an electrical current in the brain. Stimulation is typically applied at a sufficient intensity to trigger action potentials in nearby neurons.Intracortical facilitation and inhibition were studied using a paired stimulus paradigm. The motor threshold (MT) was first established. The conditioning stimulus (70% MT) followed by the test stimulus (120% MT) was delivered at 100 ms for long ICI. Each run consisted of 10 trials, and the amplitude ratio of the mean conditioned Motor Evoked Potential (MEP) to control MEP was determined. A larger amplitude ratio indicates greater cortical excitability. The differences between Placebo and Baseline, and SGS and Baseline were obtained. These values were averaged across individuals to report a mean. |
| Secondary | Results of Physical Examination at the End of the Study Drug and Placebo Treatment Periods | Six months | A physical examination was administered by a physician to subjects at the end of each six month treatment period, i.e., following completion of a six month period on SGS-742 or Placebo. Results of the examination ranged from 0-4 with scores defined as follows: 0=No observation; 1=Stable baseline findings; 2=New asymptomatic finding; 3=Patient reports some worsening of a baseline daily function associated with new finding; 4=Patient unable to carry out a baseline daily function associated with new finding |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline on the Adaptive Behavior Assessment System (ABAS) Test at the End of the Study Drug and Placebo Treatment Periods | baseline and six months | The ABAS was not able to be completed for all participants. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline of TMS Measurement of Motor Threshold at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | We were unable to obtain Motor Thresholds for all participants at all three timepoints, resulting in different N for each time period. All available data is reported. | percentage of stimulator output | MEAN |
| SECONDARY | Change From Baseline of TMS Measurement of Intracortical Facilitation at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | We were unable to obtain Motor Thresholds for all participants at all three timepoints, resulting in different N for each time period. All available data is reported. | ratio of MEP amplitude | MEAN |
| SECONDARY | Change From Baseline of TMS Measurement of Short Interval Intracortical Inhibition (Short ICI) at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | We were unable to obtain Motor Thresholds for all participants at all three timepoints, resulting in different N for each time period. All available data is reported. | ratio of MEP amplitude | MEAN |
| SECONDARY | Change From Baseline of TMS Measurement of Long Interval Intracortical Inhibition (Long ICI) at the End of the Study Drug and Placebo Treatment Periods | Baseline and Six months | We were unable to obtain Motor Thresholds for all participants at all three timepoints, resulting in different N for each time period. All available data is reported. | ratio of MEP amplitude | MEAN |
| SECONDARY | Results of Physical Examination at the End of the Study Drug and Placebo Treatment Periods | Six months | One subject withdrew prior to end of Phase 1 | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline on the Adaptive Behavior Assessment System (ABAS) Test at the End of the Study Drug and Placebo Treatment Periods | Placebo | 5.2 | Standard Deviation 7.1 | scores on a scale | |
| Change From Baseline on the Adaptive Behavior Assessment System (ABAS) Test at the End of the Study Drug and Placebo Treatment Periods | Study Drug | 4.5 | Standard Deviation 7.5 | scores on a scale | |
| Change From Baseline of TMS Measurement of Motor Threshold at the End of the Study Drug and Placebo Treatment Periods | Placebo | -2 | Standard Deviation 7.1 | percentage of stimulator output | |
| Change From Baseline of TMS Measurement of Motor Threshold at the End of the Study Drug and Placebo Treatment Periods | Study Drug | -0.5 | Standard Deviation 6.8 | percentage of stimulator output | |
| Change From Baseline of TMS Measurement of Intracortical Facilitation at the End of the Study Drug and Placebo Treatment Periods | Placebo | 49.9 | Standard Deviation 61.9 | ratio of MEP amplitude | |
| Change From Baseline of TMS Measurement of Intracortical Facilitation at the End of the Study Drug and Placebo Treatment Periods | Study Drug | 40.5 | Standard Deviation 50.2 | ratio of MEP amplitude | |
| Change From Baseline of TMS Measurement of Short Interval Intracortical Inhibition (Short ICI) at the End of the Study Drug and Placebo Treatment Periods | Placebo | 35.5 | Standard Deviation 123.4 | ratio of MEP amplitude | |
| Change From Baseline of TMS Measurement of Short Interval Intracortical Inhibition (Short ICI) at the End of the Study Drug and Placebo Treatment Periods | Study Drug | -11.0 | Standard Deviation 54.8 | ratio of MEP amplitude | |
| Change From Baseline of TMS Measurement of Long Interval Intracortical Inhibition (Long ICI) at the End of the Study Drug and Placebo Treatment Periods | Placebo | -9.3 | Standard Deviation 112.0 | ratio of MEP amplitude | |
| Change From Baseline of TMS Measurement of Long Interval Intracortical Inhibition (Long ICI) at the End of the Study Drug and Placebo Treatment Periods | Study Drug | 0.3 | Standard Deviation 98.6 | ratio of MEP amplitude |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| First Intervention (6 Months) | STARTED | Placebo First, Then SGS-742 | 10 | |
| First Intervention (6 Months) | STARTED | SGS-742 First, Then Placebo | 9 | |
| First Intervention (6 Months) | COMPLETED | Placebo First, Then SGS-742 | 10 | |
| First Intervention (6 Months) | COMPLETED | SGS-742 First, Then Placebo | 8 | |
| First Intervention (6 Months) | NOT COMPLETED | Placebo First, Then SGS-742 | 0 | |
| First Intervention (6 Months) | NOT COMPLETED | SGS-742 First, Then Placebo | 1 | |
| Washout Period (9 Weeks) | STARTED | Placebo First, Then SGS-742 | 10 | |
| Washout Period (9 Weeks) | STARTED | SGS-742 First, Then Placebo | 8 | |
| Washout Period (9 Weeks) | COMPLETED | Placebo First, Then SGS-742 | 10 | |
| Washout Period (9 Weeks) | COMPLETED | SGS-742 First, Then Placebo | 8 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events204
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal Pain | Gastrointestinal disorders | Placebo | 2 | 18 | 3 |
| other | Abdominal Pain | Gastrointestinal disorders | Washout Period Following Placebo | 0 | 16 | 0 |
| other | Abdominal Pain | Gastrointestinal disorders | Study Drug | 4 | 19 | 6 |
| other | Abdominal Pain | Gastrointestinal disorders | Washout Period Following Study Drug | 1 | 18 | 1 |
| other | Abnormal Urinary Analysis results | Renal and urinary disorders | Placebo | 0 | 18 | 0 |
| other | Abnormal Urinary Analysis results | Renal and urinary disorders | Washout Period Following Placebo | 0 | 16 | 0 |
| other | Abnormal Urinary Analysis results | Renal and urinary disorders | Study Drug | 1 | 19 | 1 |
| other | Abnormal Urinary Analysis results | Renal and urinary disorders | Washout Period Following Study Drug | 0 | 18 | 0 |
| other | Acne | Skin and subcutaneous tissue disorders | Placebo | 1 | 18 | 1 |
| other | Acne | Skin and subcutaneous tissue disorders | Washout Period Following Placebo | 0 | 16 | 0 |
1 to 10 of 204Source ClinicalTrials.gov
See all 204 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 18 |
| Total, all-cause mortality | Washout Period Following Placebo | 0 | 16 |
| Total, all-cause mortality | Study Drug | 0 | 19 |
| Total, all-cause mortality | Washout Period Following Study Drug | 0 | 18 |
| Total, other adverse events | Placebo | 16 | 18 |
| Total, other adverse events | Washout Period Following Placebo | 3 | 16 |
| Total, other adverse events | Study Drug | 18 | 19 |
| Total, other adverse events | Washout Period Following Study Drug | 6 | 18 |
| Total, serious adverse events | Placebo | 0 | 18 |
| Total, serious adverse events | Washout Period Following Placebo | 0 | 16 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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