Trial Outcomes & Findings for Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency (NCT NCT03288103)
NCT ID: NCT03288103
Last Updated: 2024-07-30
Results Overview
Height Standard Deviation Score is the standard deviation above or below the mean the height is for age and gender. Values were obtained by plotting heights on Centers for Disease Control and Prevention growth charts. An increase in Height Standard Deviation Score correlates with increase in height. Results are reported for 10 patients treated with recombinant human growth hormone.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
22 participants
Primary outcome timeframe
Annually through three years of treatment
Results posted on
2024-07-30
Participant Flow
Total number of participants enrolled was 22, including 12 pediatric participants, and 10 adult participants. Only 10 of the pediatric patients were eligible to participate in the treatment arm of the study.
Participant milestones
| Measure |
Treatment Group
Patients with Aggrecan (ACAN) deficiency treated with human growth hormone (rHGH) for 3 years.
|
Pediatric Phenotype Group
First degree pediatric relatives of patients in the treatment group who also have Aggrecan (ACAN) deficiency but did not participate in the treatment trial.
|
Adult Phenotype Group
First degree adult relatives of patients in the treatment group who also have Aggrecan (ACAN) deficiency but did not participate in the treatment trial.
|
|---|---|---|---|
|
Overall Study
STARTED
|
10
|
2
|
10
|
|
Overall Study
COMPLETED
|
10
|
2
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
Baseline characteristics by cohort
| Measure |
Treatment Group
n=10 Participants
Patients with Aggrecan (ACAN) deficiency who have been treatment with human growth hormone (rHGH) for 3 years. All patients who participated in the treatment group also participated in the phenotype arm of the study.
|
Phenotype Only Group
n=12 Participants
First degree relatives of patients in the treatment group who also have Aggrecan (ACAN) deficiency but did not participate in the treatment trial. Includes child and adult relatives. In total there are 22 participants in the phenotype arm, and 10 of those are also in the treatment arm.
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
12 participants
n=7 Participants
|
22 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Annually through three years of treatmentPopulation: 10 pre-pubertal children with Aggrecan deficiency
Height Standard Deviation Score is the standard deviation above or below the mean the height is for age and gender. Values were obtained by plotting heights on Centers for Disease Control and Prevention growth charts. An increase in Height Standard Deviation Score correlates with increase in height. Results are reported for 10 patients treated with recombinant human growth hormone.
Outcome measures
| Measure |
Growth Hormone Treatment for Participants With ACAN Deficiency
n=10 Participants
Pre-pubertal children with ACAN deficiency on daily growth hormone (Norditropin) regimen for a 3 year period.
Norditropin: Single dose of daily growth hormone regimen. Dose will be 50 micrograms/kg/day, adjusted as needed per Insulin-like growth factor 1 (IGF-I) safety monitoring.
|
Adult Phenotype Group
This include affected first degree adult relatives of the participants in the treatment arm.
|
|---|---|---|
|
Height Standard Deviation Score
Baseline
|
-2.52 standard deviation score
Interval -4.27 to -1.07
|
—
|
|
Height Standard Deviation Score
Year 1
|
-1.57 standard deviation score
Interval -3.89 to -0.46
|
—
|
|
Height Standard Deviation Score
Year 2
|
-1.19 standard deviation score
Interval -3.55 to -0.08
|
—
|
|
Height Standard Deviation Score
Year 3
|
-1.09 standard deviation score
Interval -3.45 to -0.02
|
—
|
PRIMARY outcome
Timeframe: Annually through three years of treatmentA participants calculated height velocity derived from height measurements taken over a period of 36 months (baseline visit to 36 month visit). Only those in the treatment arm were treated with growth hormone and observed for response.
Outcome measures
| Measure |
Growth Hormone Treatment for Participants With ACAN Deficiency
n=10 Participants
Pre-pubertal children with ACAN deficiency on daily growth hormone (Norditropin) regimen for a 3 year period.
Norditropin: Single dose of daily growth hormone regimen. Dose will be 50 micrograms/kg/day, adjusted as needed per Insulin-like growth factor 1 (IGF-I) safety monitoring.
|
Adult Phenotype Group
This include affected first degree adult relatives of the participants in the treatment arm.
|
|---|---|---|
|
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)
Baseline
|
5.2 cm/year
Interval 3.8 to 7.1
|
—
|
|
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)
Year 1
|
8.3 cm/year
Interval 7.3 to 11.2
|
—
|
|
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)
Year 2
|
7.7 cm/year
Interval 5.9 to 8.8
|
—
|
|
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)
Year 3
|
6.8 cm/year
Interval 4.9 to 7.2
|
—
|
SECONDARY outcome
Timeframe: BaselineEvidence of osteochondritis dissecans on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees.
Outcome measures
| Measure |
Growth Hormone Treatment for Participants With ACAN Deficiency
n=12 Participants
Pre-pubertal children with ACAN deficiency on daily growth hormone (Norditropin) regimen for a 3 year period.
Norditropin: Single dose of daily growth hormone regimen. Dose will be 50 micrograms/kg/day, adjusted as needed per Insulin-like growth factor 1 (IGF-I) safety monitoring.
|
Adult Phenotype Group
n=10 Participants
This include affected first degree adult relatives of the participants in the treatment arm.
|
|---|---|---|
|
Number of Participants With Clinical Features of ACAN Deficiency - Osteochondritis Dissecans
|
3 participants
|
1 participants
|
SECONDARY outcome
Timeframe: BaselineEvidence of early joint pathology evident (osteoarthritis) on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees, performed in those who were age appropriate and would cooperate.
Outcome measures
| Measure |
Growth Hormone Treatment for Participants With ACAN Deficiency
n=12 Participants
Pre-pubertal children with ACAN deficiency on daily growth hormone (Norditropin) regimen for a 3 year period.
Norditropin: Single dose of daily growth hormone regimen. Dose will be 50 micrograms/kg/day, adjusted as needed per Insulin-like growth factor 1 (IGF-I) safety monitoring.
|
Adult Phenotype Group
n=10 Participants
This include affected first degree adult relatives of the participants in the treatment arm.
|
|---|---|---|
|
Number of Participants With Clinical Features of ACAN Deficiency - Osteoarthritis
|
0 participants
|
8 participants
|
Adverse Events
Treatment Group
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Phenotype Group
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment Group
n=10 participants at risk
Patients with Aggrecan (ACAN) deficiency who have been treatment with human growth hormone (rHGH) for 3 years.
|
Phenotype Group
n=12 participants at risk
First degree relatives of patients in the treatment group who also have Aggrecan (ACAN) deficiency but did not participate in the treatment trial.
|
|---|---|---|
|
General disorders
Significant behavioral changes
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Eye disorders
Conjunctivitis
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Infections and infestations
Fever
|
30.0%
3/10 • Number of events 3 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Hip pain
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Knee Pain
|
40.0%
4/10 • Number of events 5 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Ear and labyrinth disorders
Ear Infection
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Skin and subcutaneous tissue disorders
Injection site reaction (hives)
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
General disorders
Headache
|
40.0%
4/10 • Number of events 5 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Vascular disorders
Face tingling
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Pes planus
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
General disorders
Injection site reaction (bruising)
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Ankle Pain
|
30.0%
3/10 • Number of events 4 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
General disorders
Vomiting
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Infections and infestations
COVID-19
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Injury, poisoning and procedural complications
Dog bite on right ear
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Surgical and medical procedures
Root Canal
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Surgical and medical procedures
Tooth Extraction
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Infections and infestations
Bronchitis
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Skin and subcutaneous tissue disorders
Lipohypertrophy of upper arms (bilateral)
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Ear and labyrinth disorders
Sore Throat
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Leg Pain
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Gastrointestinal disorders
Stomach pain/discomfort
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Injury, poisoning and procedural complications
Contusion right leg
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Injury, poisoning and procedural complications
Laceration above left eye
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Musculoskeletal and connective tissue disorders
Foot pain
|
20.0%
2/10 • Number of events 2 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Product Issues
Medication for injection was cloudy
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Skin and subcutaneous tissue disorders
Paronychia (big toe)
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Infections and infestations
Vaginal Yeast Infection
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Eye disorders
Eyelid Erythema
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Skin and subcutaneous tissue disorders
Idiopathic Uticaria
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Skin and subcutaneous tissue disorders
Molluscum
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
|
Injury, poisoning and procedural complications
Right Knee Injury (Abnormal ACL)
|
10.0%
1/10 • Number of events 1 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
0.00%
0/12 • Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit.
The definition of adverse event and/or serious adverse events does not differ from that of www.clinicialtrials.gov.
|
Additional Information
Dr. Philippe Backeljauw, MD
Cincinnati Childrens Hospital Medical Center
Phone: 15136368444
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place