Trial Outcomes & Findings for Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) (NCT NCT02599961)

Last Updated: 2020-06-11

Results Overview

An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Results posted on

2020-06-11

Participant Flow

Study enrolled pediatric, adolescent, and adult glucose transporter type 1 deficiency syndrome (Glut1 DS) participants who completed the UX007G-CL201 study (NCT01993186; rollover participants). No non-rollover participants (those from other clinical studies, investigator sponsored trials, or expanded access/compassionate use treatment) enrolled.

For continuing UX007G-CL201 participants, the Week 52 visit of that study may have been conducted in conjunction with the Baseline visit for this study to avoid duplication of assessments.

Participant milestones

Participant milestones
Measure	UX007 (Triheptanoin) UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Study STARTED	15
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	15

Reasons for withdrawal

Reasons for withdrawal
Measure	UX007 (Triheptanoin) UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Study Withdrawal by Subject	1
Overall Study Subject Non-Compliance	2
Overall Study Discontinuation of Study by Sponsor	9
Overall Study Other, Not Specified	3

Baseline Characteristics

participants with a baseline assessment

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	UX007 (Triheptanoin) n=15 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Age, Continuous	15.24 years STANDARD_DEVIATION 6.106 • n=15 Participants
Age, Customized 2 to < 12 years	5 Participants n=15 Participants
Age, Customized 12 to < 18 years	5 Participants n=15 Participants
Age, Customized 18 to < 65 years	5 Participants n=15 Participants
Sex: Female, Male Female	11 Participants n=15 Participants
Sex: Female, Male Male	4 Participants n=15 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=15 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=15 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=15 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=15 Participants
Race (NIH/OMB) Asian	1 Participants n=15 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=15 Participants
Race (NIH/OMB) Black or African American	1 Participants n=15 Participants
Race (NIH/OMB) White	11 Participants n=15 Participants
Race (NIH/OMB) More than one race	0 Participants n=15 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=15 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=15 Participants
Race/Ethnicity, Customized Asian	1 Participants n=15 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=15 Participants
Race/Ethnicity, Customized White	11 Participants n=15 Participants
Race/Ethnicity, Customized Other, Not Specified	1 Participants n=15 Participants
Overall Seizure Frequency Per 4 Weeks	312.17 seizures per 4 weeks STANDARD_DEVIATION 528.057 • n=15 Participants
Baseline (NCT01993186) Columbia Neurological Score (CNS) Total Score	38.63 score on a scale STANDARD_DEVIATION 25.428 • n=12 Participants • participants with a baseline assessment
Baseline (NCT01993186) Short Form (SF) 10 (SF-10) Health Survey for Children Physical Summary Score	32.63 T-score STANDARD_DEVIATION 17.027 • n=10 Participants • participants with a baseline assessment
Baseline (NCT01993186) SF-10 Health Survey for Children Psychosocial Summary Score	48.29 T-score STANDARD_DEVIATION 10.196 • n=10 Participants • participants with a baseline assessment
Baseline (NCT01993186) SF12 Version 2 (SF-12v2) Health Survey Physical Component Summary (PCS) Score	39.37 T-score STANDARD_DEVIATION 0.523 • n=2 Participants • participants with a baseline assessment
Baseline (NCT01993186) SF-12v2 Health Survey MCS Score	43.49 T-score STANDARD_DEVIATION 4.356 • n=2 Participants • participants with a baseline assessment

PRIMARY outcome

Timeframe: From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=15 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths TEAEs	13 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths Serious TEAEs	2 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths Related TEAEs	10 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths Serious and Related TEAEs	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths Grade 3 or 4 TEAEs	1 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths Gastrointestinal TEAEs	9 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths TEAEs Leading to Treatment Discontinuation	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths TEAEs Leading to Study Discontinuation	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths TEAEs Leading to Death	0 Participants

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36

Population: Participants with a baseline and postbaseline assessment at given time point.

The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=15 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 0-3	-64.22 seizures per 4 weeks Standard Deviation 185.564
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 4-6	-64.19 seizures per 4 weeks Standard Deviation 142.460
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 7-9	-61.81 seizures per 4 weeks Standard Deviation 164.770
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 10-12	-91.93 seizures per 4 weeks Standard Deviation 216.834
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 13-18	-75.56 seizures per 4 weeks Standard Deviation 206.406
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 19-24	-110.12 seizures per 4 weeks Standard Deviation 233.492
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 25-30	-135.60 seizures per 4 weeks Standard Deviation 249.871
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks Change at Month 31-36	-51.88 seizures per 4 weeks Standard Deviation 101.378

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36

Population: Participants with a baseline and postbaseline assessment at given time point.

The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance \& Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone \& Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=12 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in CNS Total Score Change at Month 0	11.85 score on a scale Standard Deviation 20.331
Change From Baseline Over Time in CNS Total Score Change at Month 6	9.36 score on a scale Standard Deviation 18.495
Change From Baseline Over Time in CNS Total Score Change at Month 12	13.64 score on a scale Standard Deviation 21.371
Change From Baseline Over Time in CNS Total Score Change at Month 24	3.38 score on a scale Standard Deviation 3.092
Change From Baseline Over Time in CNS Total Score Change at Month 36	0.00 score on a scale Standard Deviation NA 1 participant assessed

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

Population: Pediatric participants with a baseline and postbaseline assessment at given time point.

The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=10 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 0	0.25 T-score Standard Deviation 16.917
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 6	9.56 T-score Standard Deviation 21.522
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 12	-2.67 T-score Standard Deviation 9.475
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 18	-9.35 T-score Standard Deviation 12.702
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 24	-8.96 T-score Standard Deviation 20.075
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score Change at Month 30	7.74 T-score Standard Deviation 2.273

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

Population: Pediatric participants with a baseline and postbaseline assessment at given time point.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=10 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 0	0.59 T-score Standard Deviation 11.049
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 6	-2.29 T-score Standard Deviation 12.366
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 12	-7.43 T-score Standard Deviation 15.514
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 18	-6.60 T-score Standard Deviation 14.987
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 24	-8.25 T-score Standard Deviation 15.316
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score Change at Month 30	8.91 T-score Standard Deviation 12.599

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Population: Adult participants with a baseline and postbaseline assessment at given time point.

SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=2 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score Change at Month 6	12.37 T-score Standard Deviation 5.367
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score Change at Month 12	5.09 T-score Standard Deviation 1.619
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score Change at Month 0	10.25 T-score Standard Deviation 5.077
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score Change at Month 18	-0.35 T-score Standard Deviation NA 1 participant assessed.

SECONDARY outcome

Timeframe: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Population: Adult participants with a baseline and postbaseline assessment at given time point.

Outcome measures

Outcome measures
Measure	UX007 (Triheptanoin) n=2 Participants UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score Change at Month 6	5.84 T-score Standard Deviation 3.316
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score Change at Month 12	6.90 T-score Standard Deviation 5.706
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score Change at Month 18	16.96 T-score Standard Deviation NA 1 participant assessed.
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score Change at Month 0	8.63 T-score Standard Deviation 2.249

Adverse Events

UX007 (Triheptanoin)

Serious events: 2 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	UX007 (Triheptanoin) n=15 participants at risk UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Gastrointestinal disorders Intestinal Obstruction	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Croup Infectious	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Ear Infection	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Otitis Media	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Otitis Media Acute	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Other adverse events

Other adverse events
Measure	UX007 (Triheptanoin) n=15 participants at risk UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Cardiac disorders Tachycardia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Abdominal Pain	20.0% 3/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Abdominal Pain Upper	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Breath Odour	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Constipation	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Diarrhoea	46.7% 7/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Dysphagia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Flatulence	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Haematochezia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Nausea	20.0% 3/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Gastrointestinal disorders Vomiting	33.3% 5/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
General disorders Pyrexia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
General disorders Thirst	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Immune system disorders Seasonal Allergy	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Ear Infection	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Influenza	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Otitis Media	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Otitis Media Acute	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Pharyngitis Streptococcal	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Upper Respiratory Tract Infection	20.0% 3/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Infections and infestations Viral Upper Respiratory Tract Infection	20.0% 3/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Injury, poisoning and procedural complications Contusion	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Injury, poisoning and procedural complications Head Injury	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Injury, poisoning and procedural complications Ligament Sprain	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Injury, poisoning and procedural complications Procedural Pain	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Investigations Alanine Aminotransferase Increased	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Investigations Blood Glucose Increased	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Investigations Weight Increased	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Metabolism and nutrition disorders Hypoglycaemia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Metabolism and nutrition disorders Hyponatraemia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Musculoskeletal and connective tissue disorders Back Pain	13.3% 2/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Musculoskeletal and connective tissue disorders Muscle Spasms	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Musculoskeletal and connective tissue disorders Muscular Weakness	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Musculoskeletal and connective tissue disorders Pain In Extremity	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Disturbance In Attention	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Dysarthria	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Head Titubation	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Headache	26.7% 4/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Lethargy	20.0% 3/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Petit Mal Epilepsy	13.3% 2/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Seizure	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Nervous system disorders Tremor	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Psychiatric disorders Insomnia	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Reproductive system and breast disorders Dysmenorrhoea	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Reproductive system and breast disorders Vulvovaginal Pruritus	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Respiratory, thoracic and mediastinal disorders Cough	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Skin and subcutaneous tissue disorders Hair Growth Abnormal	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Vascular disorders Hot Flush	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Vascular disorders Hypertension	6.7% 1/15 • From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Additional Information

Medical Information

Ultragenyx Pharmaceutical Inc

Phone: 1-888-756-8657

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Publication restrictions are in place

Restriction type: OTHER