Trial Outcomes & Findings for Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD) (NCT NCT00947297)

NCT ID: NCT00947297

Last Updated: 2024-07-10

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 60 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2024-07-10

Participant Flow

Participant milestones

Measure	HPN-100 Patients who were treated with HPN-100
Overall Study STARTED	60
Overall Study COMPLETED	53
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Baseline characteristics by cohort

Measure	HPN-100 n=60 Participants Patients who were treated with HPN-100
Age, Categorical <=18 years	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	51 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	28.83 years STANDARD_DEVIATION 13.933 • n=5 Participants
Sex: Female, Male Female	41 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Region of Enrollment United States	54 participants n=5 Participants
Region of Enrollment Canada	6 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Measure	HPN-100 n=60 Participants Patients who were treated with HPN-100
Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug)	33 participants

SECONDARY outcome

Timeframe: 1 year

Number of hyperammonemic crises per patient

Outcome measures

Measure	HPN-100 n=60 Participants Patients who were treated with HPN-100
Number and Causes of Hyperammonemic Events	0.20 hyperammonemic events Standard Deviation 0.514

SECONDARY outcome

Timeframe: 1 Year

Venous Ammonia levels over time

Outcome measures

Measure	HPN-100 n=60 Participants Patients who were treated with HPN-100
Blood Ammonia Levels Baseline	27.623 Umol/L Standard Deviation 15.8875
Blood Ammonia Levels Month 12	24.202 Umol/L Standard Deviation 20.6124

SECONDARY outcome

Timeframe: Month 1 post dose

Population: all available questionnaires

Drug preference will be noted at week 3

Outcome measures

Measure	HPN-100 n=50 Participants Patients who were treated with HPN-100
Patient Satisfaction With HPN-100	90 % preferred HPN-100

Adverse Events

HPN-100

Serious events: 12 serious events

Other events: 59 other events

Deaths: 0 deaths

Serious adverse events

Measure	HPN-100 n=60 participants at risk Patients who were treated with HPN-100
Gastrointestinal disorders Abdominal pain	1.7% 1/60 • 1 year
Infections and infestations Gastroenteritis	1.7% 1/60 • 1 year
Infections and infestations Lobar pneumonia	1.7% 1/60 • 1 year
Metabolism and nutrition disorders Hyperammonaemia	15.0% 9/60 • 1 year
Nervous system disorders Dizziness	1.7% 1/60 • 1 year
Nervous system disorders Neuropathy peripheral	1.7% 1/60 • 1 year
Psychiatric disorders Psychotic disorder	1.7% 1/60 • 1 year
Reproductive system and breast disorders Pelvic pain	1.7% 1/60 • 1 year
Respiratory, thoracic and mediastinal disorders Lung infiltration	1.7% 1/60 • 1 year

Other adverse events

Measure	HPN-100 n=60 participants at risk Patients who were treated with HPN-100
Gastrointestinal disorders Abdominal discomfort	6.7% 4/60 • 1 year
Gastrointestinal disorders Abdominal distension	5.0% 3/60 • 1 year
General disorders Abdominal pain	10.0% 6/60 • 1 year
General disorders Constipation	6.7% 4/60 • 1 year
Gastrointestinal disorders Diarrhoea	18.3% 11/60 • 1 year
Gastrointestinal disorders Dyspepsia	6.7% 4/60 • 1 year
Gastrointestinal disorders Nausea	21.7% 13/60 • 1 year
Gastrointestinal disorders Vomiting	31.7% 19/60 • 1 year
General disorders Fatigue	11.7% 7/60 • 1 year
General disorders Pyrexia	6.7% 4/60 • 1 year
Infections and infestations Bronchitis	6.7% 4/60 • 1 year
Infections and infestations Gastroenteritis	6.7% 4/60 • 1 year
Infections and infestations Gastroenteritis viral	10.0% 6/60 • 1 year
Infections and infestations Nasopharyngitis	18.3% 11/60 • 1 year
Infections and infestations Upper respiratory tract infection	31.7% 19/60 • 1 year
Investigations Aspartate aminotransferase increased	5.0% 3/60 • 1 year
Investigations Vitamin D decreased	6.7% 4/60 • 1 year
Metabolism and nutrition disorders Decreased appetite	13.3% 8/60 • 1 year
Metabolism and nutrition disorders Hyperammonaemia	15.0% 9/60 • 1 year
Metabolism and nutrition disorders Increased appetite	5.0% 3/60 • 1 year
Musculoskeletal and connective tissue disorders Back pain	6.7% 4/60 • 1 year
Musculoskeletal and connective tissue disorders Pain in extremity	6.7% 4/60 • 1 year
Nervous system disorders Convulsion	5.0% 3/60 • 1 year
Nervous system disorders Dizziness	15.0% 9/60 • 1 year
Nervous system disorders Headache	16.7% 10/60 • 1 year
Nervous system disorders Tremor	5.0% 3/60 • 1 year
Reproductive system and breast disorders Metrorrhagia	5.0% 3/60 • 1 year
Respiratory, thoracic and mediastinal disorders Cough	13.3% 8/60 • 1 year
Respiratory, thoracic and mediastinal disorders Nasal congestion	6.7% 4/60 • 1 year
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	11.7% 7/60 • 1 year
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	6.7% 4/60 • 1 year
Skin and subcutaneous tissue disorders Acne	5.0% 3/60 • 1 year
Skin and subcutaneous tissue disorders Rash	10.0% 6/60 • 1 year
Skin and subcutaneous tissue disorders Skin odour abnormal	6.7% 4/60 • 1 year

Additional Information

Craig James

Hyperion Therapeutics

Phone: 650-745 7840

Email: craig.james@hyperiontx.com

Results disclosure agreements

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

Restriction type: GT60