Trial Outcomes & Findings for Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis (NCT NCT01438411)
NCT ID: NCT01438411
Last Updated: 2023-10-03
Results Overview
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
53 participants
Primary outcome timeframe
At baseline, then every 12 months for an average of 3.5 years
Results posted on
2023-10-03
Participant Flow
This study included subjects with inborn errors of bile acid metabolism who had previously participated in studies CAC-91-10-10 or CAC-001-01 as well as newly diagnosed subjects. Data were collected from 1 Jan 2010 through study completion on 31 Jul 2016. Note that treatment with cholic acid continues throughout a subject's lifetime.
Of 53 subjects, 31 subjects rolled over from studies CAC-91-10-10 and/or CAC-001-01, while 22 patients were treatment-naive, i.e. received their first dose of cholic acid during study CAC-002-01.
Participant milestones
| Measure |
Cholic Acid
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Overall Study
STARTED
|
53
|
|
Overall Study
COMPLETED
|
40
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Cholic Acid
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Lack of Efficacy
|
1
|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
Baseline characteristics by cohort
| Measure |
Cholic Acid
n=53 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Age, Continuous
|
9 years
STANDARD_DEVIATION 9 • n=99 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
24 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
22 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
45 participants
n=99 Participants
|
|
Region of Enrollment
Argentina
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Chile
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Israel
|
2 participants
n=99 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Mexico
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Australia
|
1 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
Outcome measures
| Measure |
Cholic Acid
n=51 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Baseline score: normal
|
25 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Baseline score: slight
|
6 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Baseline score: significant
|
10 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Baseline score: marked
|
10 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Worst-post-BL score: normal
|
25 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Worst-post-BL score: slight
|
11 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Worst-post-BL score: significant
|
6 Participants
|
|
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
Worst-post-BL score: marked
|
9 Participants
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes in ALT were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: \<ULN; ≥1 ULN but \<2 ULN; ≥2 ULN but \<3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.
Outcome measures
| Measure |
Cholic Acid
n=48 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Evaluation of Serum Transaminases: ALT
Baseline: ALT<ULN
|
27 Participants
|
|
Evaluation of Serum Transaminases: ALT
Baseline: ULN<=ALT<2 ULN
|
9 Participants
|
|
Evaluation of Serum Transaminases: ALT
Baseline: 2 ULN<=ALT<3 ULN
|
7 Participants
|
|
Evaluation of Serum Transaminases: ALT
Baseline: ALT>=3 ULN
|
5 Participants
|
|
Evaluation of Serum Transaminases: ALT
Worst-post-BL value: ALT<ULN
|
20 Participants
|
|
Evaluation of Serum Transaminases: ALT
Worst-post-BL value: ULN<=ALT<2 ULN
|
15 Participants
|
|
Evaluation of Serum Transaminases: ALT
Worst-post-BL value: 2 ULN<=ALT<3 ULN
|
9 Participants
|
|
Evaluation of Serum Transaminases: ALT
Worst-post-BL value: ALT>=3 ULN
|
4 Participants
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes in AST were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: \<ULN; ≥1 ULN but \<2 ULN; ≥2 ULN but \<3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.
Outcome measures
| Measure |
Cholic Acid
n=47 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Evaluation of Serum Transaminases: AST
Baseline: AST<ULN
|
17 Participants
|
|
Evaluation of Serum Transaminases: AST
Baseline: ULN<=AST<2 ULN
|
17 Participants
|
|
Evaluation of Serum Transaminases: AST
Baseline: 2 ULN<=AST<3 ULN
|
5 Participants
|
|
Evaluation of Serum Transaminases: AST
Baseline: AST<=3 ULN
|
8 Participants
|
|
Evaluation of Serum Transaminases: AST
Worst-post-BL value: AST<ULN
|
15 Participants
|
|
Evaluation of Serum Transaminases: AST
Worst-post-BL value: ULN<=AST<2 ULN
|
17 Participants
|
|
Evaluation of Serum Transaminases: AST
Worst-post-BL value: 2 ULN<=AST<3 ULN
|
6 Participants
|
|
Evaluation of Serum Transaminases: AST
Worst-post-BL value: AST<=3 ULN
|
9 Participants
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for bilirubin. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Outcome measures
| Measure |
Cholic Acid
n=48 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Clinical Laboratory Results: Bilirubin
Baseline
|
3.678 mg/dL
Standard Error 1.1427
|
|
Clinical Laboratory Results: Bilirubin
Worst-post-BL value
|
3.785 mg/dL
Standard Error 1.1464
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for GGT. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Outcome measures
| Measure |
Cholic Acid
n=42 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)
Baseline
|
38.8 U/L
Standard Error 7.13
|
|
Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)
Worst-post-BL value
|
16.4 U/L
Standard Error 8.28
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for alkaline phosphatase. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Outcome measures
| Measure |
Cholic Acid
n=47 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Clinical Laboratory Results: Alkaline Phosphatase
Baseline
|
293.7 U/L
Standard Error 30.30
|
|
Clinical Laboratory Results: Alkaline Phosphatase
Worst-post-BL value
|
39.6 U/L
Standard Error 17.71
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for prothrombin time. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Outcome measures
| Measure |
Cholic Acid
n=46 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Clinical Laboratory Results: Prothrombin Time
Baseline
|
17.977 sec
Standard Error 2.1338
|
|
Clinical Laboratory Results: Prothrombin Time
Worst-post-BL value
|
1.045 sec
Standard Error 2.1442
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes in height percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.
Outcome measures
| Measure |
Cholic Acid
n=41 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Physical Examinations: Height
Baseline
|
26.767 Height percentiles
Standard Error 4.4448
|
|
Physical Examinations: Height
Worst-post-BL value
|
25.540 Height percentiles
Standard Error 4.1064
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsPopulation: Patients with values
Changes in body weight percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.
Outcome measures
| Measure |
Cholic Acid
n=48 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Physical Examinations: Body Weight
Baseline
|
38.030 Body weight percentiles
Standard Error 5.2853
|
|
Physical Examinations: Body Weight
Worst-post-BL value
|
33.227 Body weight percentiles
Standard Error 4.9842
|
SECONDARY outcome
Timeframe: At baseline, then every 12 months for an average of 3.5 yearsNumber (%) of patients with any AE
Outcome measures
| Measure |
Cholic Acid
n=53 Participants
Cholic acid capsules, each containing 50 mg or 250 mg of cholic acid to be administered orally at a daily dose of 10-15 mg/kg body weight
|
|---|---|
|
Incidence of Adverse Events
|
44 Participants
|
Adverse Events
Cholic Acid
Serious events: 19 serious events
Other events: 21 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
Cholic Acid
n=53 participants at risk
Active drug
Cholic Acid: 10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules
|
|---|---|
|
Blood and lymphatic system disorders
Coagulopathy
|
5.7%
3/53 • Number of events 3 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Cardiac disorders
Bradykardia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Cardiac disorders
Cardiac arrest
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Gastrointestinal disorders
Abdominal distension
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Gastrointestinal disorders
Gastric ulcer
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
General disorders
Disease progression
|
9.4%
5/53 • Number of events 6 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
General disorders
Pyrexia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Hepatobiliary disorders
Hepatic artery thrombosis
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Hepatobiliary disorders
Hepatic failure
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
1.9%
1/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Bacteraemia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Ear infection
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Gastroenteritis viral
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Influenza
|
3.8%
2/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Pneumonia
|
5.7%
3/53 • Number of events 3 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Sepsis
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Upper respiratory tract infection
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Injury, poisoning and procedural complications
Fracture
|
3.8%
2/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Metabolism and nutrition disorders
Dehydration
|
1.9%
1/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Oesophageal carcinoma
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Nervous system disorders
Encephalopathy
|
3.8%
2/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Nervous system disorders
Mental impairment
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Nervous system disorders
Somnolence
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Psychiatric disorders
Completed suicide
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
3.8%
2/53 • Number of events 2 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Surgical and medical procedures
Adenoidectomy
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Surgical and medical procedures
Orchidopexy
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Vascular disorders
Thrombosis
|
1.9%
1/53 • Number of events 1 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
Other adverse events
| Measure |
Cholic Acid
n=53 participants at risk
Active drug
Cholic Acid: 10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
11.3%
6/53 • Number of events 8 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.3%
6/53 • Number of events 7 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Nasopharyngitis
|
7.5%
4/53 • Number of events 4 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Infections and infestations
Upper respiratory tract infection
|
15.1%
8/53 • Number of events 8 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Investigations
Hepatic enzyme increased
|
7.5%
4/53 • Number of events 6 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Investigations
Vitamin D decreased
|
11.3%
6/53 • Number of events 6 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.7%
3/53 • Number of events 3 • Subjects underwent assessments at baseline and every 12 months or when clinically indicated. Treatment with cholic acid will continue throughout a subject's lifetime. CAC-002-01 study reports data collected from 1 January 2010 until study completion on 31 July 2016, approximately 6.5 years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place