Trial Outcomes & Findings for Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid (NCT NCT00007020)
NCT ID: NCT00007020
Last Updated: 2023-10-03
Results Overview
Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
85 participants
Primary outcome timeframe
Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
Results posted on
2023-10-03
Participant Flow
A total of 85 patients were enrolled.
Of the patients diagnosed with a defect in bile acid synthesis during routine diagnostic procedures at the Cincinnati Children's Hospital Medical Center (CCHMC) Mass Spectrometry Laboratory, 85 patients were invited to participate in the study.
Participant milestones
| Measure |
Cholic Acid
Cholic acid capsules, each containing 250 mg of cholic acid, or a liquid preparation of 15 mg/mL cholic acid, to be administered orally in a dose of 15 mg/kg body weight/day.
|
|---|---|
|
Overall Study
STARTED
|
85
|
|
Overall Study
COMPLETED
|
41
|
|
Overall Study
NOT COMPLETED
|
44
|
Reasons for withdrawal
| Measure |
Cholic Acid
Cholic acid capsules, each containing 250 mg of cholic acid, or a liquid preparation of 15 mg/mL cholic acid, to be administered orally in a dose of 15 mg/kg body weight/day.
|
|---|---|
|
Overall Study
Death
|
16
|
|
Overall Study
Lost to Follow-up
|
13
|
|
Overall Study
Pt withdrawn (worsening cholestasis)
|
1
|
|
Overall Study
Pt withdrawn (liver transplant)
|
4
|
|
Overall Study
Pt cared for by outside physician
|
4
|
|
Overall Study
No evidence of treatment
|
6
|
Baseline Characteristics
Continuous age data were not available for 8 out of 85 patients.
Baseline characteristics by cohort
| Measure |
Cholic Acid
n=85 Participants
All patients entered into the study
|
|---|---|
|
Age, Continuous
|
3 years
STANDARD_DEVIATION 4 • n=77 Participants • Continuous age data were not available for 8 out of 85 patients.
|
|
Sex: Female, Male
Female
|
31 Participants
n=81 Participants • Information on gender was not recorded for 4 patients.
|
|
Sex: Female, Male
Male
|
50 Participants
n=81 Participants • Information on gender was not recorded for 4 patients.
|
|
Region of Enrollment
United States
|
85 Participants
n=85 Participants
|
PRIMARY outcome
Timeframe: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 yearsPopulation: Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment
Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)
Outcome measures
| Measure |
Cholic Acid
n=70 Participants
Patients treated
|
|---|---|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
On treatment, no atypical bile acid excretion
|
51 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
Baseline, no atypical bile acid excretion
|
10 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
Baseline, slight atypical bile acid excretion
|
11 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
On treatment, slight atypical bile acid excretion
|
9 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
Baseline, significant atypical bile acid excretion
|
16 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
OT, significant atypical bile acid excretion
|
4 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
Baseline, marked atypical bile acid excretion
|
33 Participants
|
|
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
On treatment, marked atypical bile acid excretion
|
6 Participants
|
SECONDARY outcome
Timeframe: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 yearsPopulation: Patients treated and with at least 1 pre-treatment and 1 post-Treatment assessment
Patients with elevations of liver function tests (alanine transaminase \[ALT\], aspartate transaminase \[AST\]) measured as multiples of the upper limit of normal (ULN) at baseline (worst value) and on treatment (best value)
Outcome measures
| Measure |
Cholic Acid
n=70 Participants
Patients treated
|
|---|---|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, ALT <ULN
|
14 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, ALT <ULN
|
49 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, ULN ≤ ALT <2 x ULN
|
16 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, ULN ≤ ALT <2 x ULN
|
13 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, 2 ULN ≤ ALT <3 x ULN
|
8 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, 2 ULN ≤ ALT <3 x ULN
|
2 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, ALT ≥3 x ULN
|
29 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, ALT ≥3 x ULN
|
3 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, AST <ULN
|
9 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, AST <ULN
|
38 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, ULN ≤ AST <2 x ULN
|
14 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, ULN ≤ AST <2 x ULN
|
15 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, 2 ULN ≤ AST <3 x ULN
|
7 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, 2 ULN ≤ AST <3 x ULN
|
4 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, AST ≥3 x ULN
|
35 Participants
|
|
Change in Liver Function Tests (LFTs) Measured in Serum
On treatment, AST ≥3 x ULN
|
9 Participants
|
SECONDARY outcome
Timeframe: At baseline (if no historical data were available) and between 1 and 6 months following treatment start.Population: All patients entered into the study
Patients (number, percentage) with pathological findings for qualitative (the presence of inflammation, fibrosis, necrosis, giant cells and cholestasis) and quantitative (the degrees of the aforementioned histologic features) liver histopathology at baseline (BL) and on treatment (OT).
Outcome measures
| Measure |
Cholic Acid
n=85 Participants
Patients treated
|
|---|---|
|
Liver Histology
Periportal inflammation at BL, qualitative
|
19 Participants
|
|
Liver Histology
Periportal inflammation OT, qualitative
|
16 Participants
|
|
Liver Histology
Lobular inflammation at BL, qualitative
|
5 Participants
|
|
Liver Histology
Lobular inflammation OT, qualitative
|
4 Participants
|
|
Liver Histology
Inflammation (not spec.) at BL, qualitative
|
3 Participants
|
|
Liver Histology
Inflammation (not spec.) OT, qualitative
|
0 Participants
|
|
Liver Histology
Bridging fibrosis at BL, qualitative
|
25 Participants
|
|
Liver Histology
Bridging fibrosis OT, qualitative
|
31 Participants
|
|
Liver Histology
Fibrosis (not specified) at BL, qualitative
|
6 Participants
|
|
Liver Histology
Fibrosis (not specified) OT, qualitative
|
6 Participants
|
|
Liver Histology
Cholestasis at BL, qualitative
|
20 Participants
|
|
Liver Histology
Cholestasis OT, qualitative
|
10 Participants
|
|
Liver Histology
Giant cells at BL, qualitative
|
18 Participants
|
|
Liver Histology
Giant cells OT, qualitative
|
12 Participants
|
|
Liver Histology
Necrosis at BL, qualitative
|
14 Participants
|
|
Liver Histology
Necrosis OT, qualitative
|
2 Participants
|
|
Liver Histology
Periportal inflammation at BL, quantitative
|
18 Participants
|
|
Liver Histology
Periportal inflammation OT, quantitative
|
15 Participants
|
|
Liver Histology
Lobular inflammation at BL, quantitative
|
5 Participants
|
|
Liver Histology
Lobular inflammation OT, quantitative
|
4 Participants
|
|
Liver Histology
Inflammation (not specified) at BL, quantitative
|
3 Participants
|
|
Liver Histology
Inflammation (not specified) OT, quantitative
|
0 Participants
|
|
Liver Histology
Bridging fibrosis at baseline, quantitative
|
22 Participants
|
|
Liver Histology
Bridging fibrosis OT, quantitative
|
27 Participants
|
|
Liver Histology
Fibrosis (not specified) at BL, quantitative
|
3 Participants
|
|
Liver Histology
Fibrosis (not specified) OT, quantitative
|
5 Participants
|
|
Liver Histology
Cholestasis at BL, quantitative
|
15 Participants
|
|
Liver Histology
Cholestasis OT, quantitative
|
7 Participants
|
|
Liver Histology
Giant cells at BL, quantitative
|
13 Participants
|
|
Liver Histology
Giant cells OT, quantitative
|
9 Participants
|
|
Liver Histology
Necrosis at BL, quantitative
|
13 Participants
|
|
Liver Histology
Necrosis OT, quantitative
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 yearsPopulation: Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment
Change in height/weight percentiles from baseline (worst value) to the best on-treatment value, based on CDC (Centres for Disease Control and Prevention, US) growth chart percentiles
Outcome measures
| Measure |
Cholic Acid
n=70 Participants
Patients treated
|
|---|---|
|
Height and Weight
Height percentile, baseline
|
30.1 Percentile
Standard Error 5.5
|
|
Height and Weight
Height percentile, on treatment
|
44.0 Percentile
Standard Error 6.1
|
|
Height and Weight
Weight percentile, baseline
|
21.3 Percentile
Standard Error 3.7
|
|
Height and Weight
Weight percentile, on treatment
|
42.3 Percentile
Standard Error 4.8
|
SECONDARY outcome
Timeframe: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 yearsPopulation: Patients entered into the study and treated
Number of patients with any adverse event
Outcome measures
| Measure |
Cholic Acid
n=79 Participants
Patients treated
|
|---|---|
|
Adverse Events
Patients at risk
|
79 Participants
|
|
Adverse Events
Patients with any AE
|
38 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline and on treatment (every 1, 3, or 6 months, depending on protocol version, for an average of 2.8 years)Population: All available bilirubin laboratory data are collected then grouped by bilirubin test type and collection time points (baseline vs. on treatment). Not all participant had available data, and each participant may have more than one type of bilirubin collected at a time point, and may have multiple on-treatment collections.
Bilirubin concentration in serum at baseline and on treatment
Outcome measures
| Measure |
Cholic Acid
n=689 Number of Test Results
Patients treated
|
|---|---|
|
Change in Bilirubin Measured in Serum
Baseline, bilirubin
|
0.3 mg/dL
|
|
Change in Bilirubin Measured in Serum
On treatment, bilirubin
|
0.6 mg/dL
Standard Deviation 0.5
|
|
Change in Bilirubin Measured in Serum
Baseline, direct bilirubin
|
2.2 mg/dL
Standard Deviation 5.6
|
|
Change in Bilirubin Measured in Serum
On treatment, direct bilirubin
|
0.5 mg/dL
Standard Deviation 1.8
|
|
Change in Bilirubin Measured in Serum
Baseline, indirect bilirubin
|
0.5 mg/dL
Standard Deviation 0.9
|
|
Change in Bilirubin Measured in Serum
On treatment, indirect bilirubin
|
0.2 mg/dL
Standard Deviation 0.2
|
|
Change in Bilirubin Measured in Serum
Baseline, total bilirubin
|
2.8 mg/dL
Standard Deviation 10.6
|
|
Change in Bilirubin Measured in Serum
On treatment, total bilirubin
|
1.5 mg/dL
Standard Deviation 5.2
|
Adverse Events
Cholic Acid
Serious events: 20 serious events
Other events: 35 other events
Deaths: 13 deaths
Serious adverse events
| Measure |
Cholic Acid
n=79 participants at risk
All patients entered into the study and treated with Cholic Acid. 85 patients enrolled in the study and 79 received treatment (safety set).
|
|---|---|
|
Investigations
Nutritional condition abnormal
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
1.3%
1/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Blood and lymphatic system disorders
Coagulopathy
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Nervous system disorders
Convulsion
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
General disorders
Disease progression
|
8.9%
7/79 • Number of events 7 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Diarrhoea
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Gastric ulcer
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.3%
1/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Pneumoperitoneum
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Hepatobiliary disorders
Jaundice
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Dehydration
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Gastroenteritis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Gastroenteritis viral
|
1.3%
1/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Infection
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Nasopharyngitis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Rotavirus infection
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Urinary tract infection
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
Other adverse events
| Measure |
Cholic Acid
n=79 participants at risk
All patients entered into the study and treated with Cholic Acid. 85 patients enrolled in the study and 79 received treatment (safety set).
|
|---|---|
|
General disorders
Disease progression
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
General disorders
Injection site inflammation
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
General disorders
Malaise
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
General disorders
Pyrexia
|
8.9%
7/79 • Number of events 7 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Psychiatric disorders
Hallucination
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Reproductive system and breast disorders
Ovarian cyst
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Reproductive system and breast disorders
Ovarian failure
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Injury, poisoning and procedural complications
Contusion
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Injury, poisoning and procedural complications
Fracture
|
5.1%
4/79 • Number of events 6 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Investigations
Amino acid level increased
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Investigations
Occult blood
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Investigations
Weight decreased
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary congestion
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Nervous system disorders
Convulsion
|
5.1%
4/79 • Number of events 6 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Nervous system disorders
Hypotonia
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Nervous system disorders
Lethargy
|
3.8%
3/79 • Number of events 3 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Abdominal discomfort
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Abdominal pain
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Anal fistula
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Ascites
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Constipation
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Diarrhoea
|
5.1%
4/79 • Number of events 4 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Enteritis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Inguinal hernia
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Gastrointestinal disorders
Vomiting
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Hepatobiliary disorders
Cholelithiasis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Hepatobiliary disorders
Cholestasis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Hepatobiliary disorders
Jaundice
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Hepatobiliary disorders
Portal hypertension
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Decreased appetite
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Dehydration
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Iron deficiency
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Malnutrition
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Metabolism and nutrition disorders
Vitamin E deficiency
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Bronchopneumonia
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Ear infection
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Gastroenteritis viral
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Otitis media
|
2.5%
2/79 • Number of events 2 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Pneumonia
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Respiratory tract infection
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Upper respiratory tract infection
|
6.3%
5/79 • Number of events 5 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
|
Infections and infestations
Varicella
|
1.3%
1/79 • Number of events 1 • Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place