Trial Outcomes & Findings for Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome) (NCT NCT01525901)
NCT ID: NCT01525901
Last Updated: 2022-05-12
Results Overview
16 items on ABC-SW subscale, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score from 0 to 48 with higher score indicating poorer health outcomes.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Baseline and Week 12
Results posted on
2022-05-12
Participant Flow
Participant milestones
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 1: Normal Saline Then IGF-1
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
Study 2: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 2: Normal Saline Then IGF-1
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
|---|---|---|---|---|
|
Period 1: 12 Weeks
STARTED
|
5
|
4
|
5
|
5
|
|
Period 1: 12 Weeks
COMPLETED
|
5
|
4
|
5
|
5
|
|
Period 1: 12 Weeks
NOT COMPLETED
|
0
|
0
|
0
|
0
|
|
Washout 4 Weeks
STARTED
|
5
|
4
|
5
|
5
|
|
Washout 4 Weeks
COMPLETED
|
5
|
4
|
5
|
5
|
|
Washout 4 Weeks
NOT COMPLETED
|
0
|
0
|
0
|
0
|
|
Period 2: 12 Weeks
STARTED
|
5
|
4
|
5
|
5
|
|
Period 2: 12 Weeks
COMPLETED
|
5
|
3
|
5
|
5
|
|
Period 2: 12 Weeks
NOT COMPLETED
|
0
|
1
|
0
|
0
|
Reasons for withdrawal
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 1: Normal Saline Then IGF-1
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
Study 2: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 2: Normal Saline Then IGF-1
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
|---|---|---|---|---|
|
Period 2: 12 Weeks
Adverse Event
|
0
|
1
|
0
|
0
|
Baseline Characteristics
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
Baseline characteristics by cohort
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
n=5 Participants
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 1: Normal Saline Then IGF-1
n=4 Participants
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
Study 2: Insulin-Like Growth Factor-1 (IGF-1) Then Normal Saline
n=5 Participants
Participants who have pathogenic deletions or sequence variants of the SHANK3 gene.
Insulin-Like Growth Factor-1 (IGF-1) and then placebo administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.
|
Study 2: Normal Saline Then IGF-1
n=5 Participants
12 weeks in each treatment arm (placebo and then IGF-1), separated by a 4-week wash-out phase
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
9.80 years
STANDARD_DEVIATION 4.64 • n=5 Participants
|
6.90 years
STANDARD_DEVIATION 1.51 • n=7 Participants
|
5.93 years
STANDARD_DEVIATION 1.06 • n=5 Participants
|
6.97 years
STANDARD_DEVIATION 1.71 • n=4 Participants
|
7.43 years
STANDARD_DEVIATION 2.89 • n=21 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
ABC-SW subscale score
|
20.4 units on a scale
STANDARD_DEVIATION 8.38 • n=5 Participants
|
12.5 units on a scale
STANDARD_DEVIATION 7.72 • n=7 Participants
|
19.2 units on a scale
STANDARD_DEVIATION 3.63 • n=5 Participants
|
21 units on a scale
STANDARD_DEVIATION 6.36 • n=4 Participants
|
18.58 units on a scale
STANDARD_DEVIATION 6.95 • n=21 Participants
|
|
Repetitive Behavior Scale (RBS) - Total
|
35.4 units on a scale
STANDARD_DEVIATION 29.63 • n=5 Participants
|
28 units on a scale
STANDARD_DEVIATION 16.10 • n=7 Participants
|
12.4 units on a scale
STANDARD_DEVIATION 15.88 • n=5 Participants
|
18 units on a scale
STANDARD_DEVIATION 9.92 • n=4 Participants
|
23.21 units on a scale
STANDARD_DEVIATION 20.07 • n=21 Participants
|
|
Vineland Adaptive Behavior Composite Standard Score
|
43.6 units on a scale
STANDARD_DEVIATION 13.67 • n=5 Participants
|
47.75 units on a scale
STANDARD_DEVIATION 3.59 • n=7 Participants
|
48.8 units on a scale
STANDARD_DEVIATION 5.81 • n=5 Participants
|
49.2 units on a scale
STANDARD_DEVIATION 13.33 • n=4 Participants
|
47.32 units on a scale
STANDARD_DEVIATION 9.80 • n=21 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 1216 items on ABC-SW subscale, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score from 0 to 48 with higher score indicating poorer health outcomes.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=9 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=9 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1
Baseline
|
15.78 score on a scale
Standard Error 9.16
|
11.22 score on a scale
Standard Error 7.29
|
—
|
—
|
|
Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1
Week 12
|
7.61 score on a scale
Standard Error 5.71
|
9.70 score on a scale
Standard Error 8.07
|
—
|
—
|
PRIMARY outcome
Timeframe: Baseline and Week 1216 items on ABC-SW subscale, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score from 0 to 48 with higher score indicating poorer health outcomes..
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=10 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale Study 2
|
-5.5 score on a scale
Standard Deviation 7.46
|
-6.3 score on a scale
Standard Deviation 5.38
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Participants in Study 2
Repetitive Behavior Scale (RBS) - Total Score 43 items, each item scored on 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. with total score from 0 (mild) to 129 (severe). The subscales are stereotyped behaviors 6 items (subscale 0-18), self-injurious behaviors 8 items (subscale 0-24), Compulsive behaviors- 8 items (subscale 0-24), Ritualistic Behaviors 6 items (subscale from 0-18), Sameness 11 items (subscale 0-33), restricted behaviors 4 items (subscale 0-12). Total score is the sum of all items in the subscale with total score range from 0 to 129. Higher scores indicate greater symptom severity.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=10 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Stereotyped Behavior
|
-0.3 score on a scale
Standard Deviation 2.63
|
-0.8 score on a scale
Standard Deviation 2.19
|
—
|
—
|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Self-Injurious Behavior
|
-0.3 score on a scale
Standard Deviation 1.64
|
0.2 score on a scale
Standard Deviation 1.14
|
—
|
—
|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Compulsive Behavior
|
-0.8 score on a scale
Standard Deviation 1.4
|
0.1 score on a scale
Standard Deviation 0.88
|
—
|
—
|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Sameness Behavior
|
-1.4 score on a scale
Standard Deviation 2.27
|
-0.37 score on a scale
Standard Deviation 1.74
|
—
|
—
|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Restricted Behavior
|
-1.1 score on a scale
Standard Deviation 1.66
|
-0.1 score on a scale
Standard Deviation 1.29
|
—
|
—
|
|
Change in Repetitive Behavior Scale - Study 2
RBS-Total
|
-3.5 score on a scale
Standard Deviation 6.2
|
-0.77 score on a scale
Standard Deviation 5.73
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 12The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=10 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in CGI-Improvement and Severity Scales; - Study 2
CGI - Severity
|
-0.2 score on a scale
Standard Deviation 0.42
|
-0.1 score on a scale
Standard Deviation 0.32
|
—
|
—
|
|
Change in CGI-Improvement and Severity Scales; - Study 2
CGI - Improvement
|
2.6 score on a scale
Standard Deviation 0.84
|
3.1 score on a scale
Standard Deviation 1.28
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: 4 participants missing data from at least one data point
21 question tool, about caregiver strain, where each question uses a Likert scale where 1 is "Not at all" and 5 is "Very much." The full scale ranges from 21-105, where higher scores indicate more severe strain.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=9 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=9 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
n=6 Participants
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Caregiver Strain Questionnaire
Baseline
|
64.11 score on a scale
Standard Deviation 17.32
|
63.22 score on a scale
Standard Deviation 20.48
|
58 score on a scale
Standard Deviation 18.89
|
55.00 score on a scale
Standard Deviation 19.54
|
|
Change in Caregiver Strain Questionnaire
Week 12
|
61.11 score on a scale
Standard Deviation 17.60
|
64.11 score on a scale
Standard Deviation 17.08
|
55.67 score on a scale
Standard Deviation 18.99
|
53.00 score on a scale
Standard Deviation 19.68
|
SECONDARY outcome
Timeframe: Baseline and Week 12The SP is a standardized parent-completed questionnaire that assesses sensory processing and its impact on the functioning of children ages 3-10 yr. The 125 items represent behaviors that can be interpreted as responses to sensory experiences. The parent rates the observed frequency of these behaviors on a 5-point Likert scale (ranging from 1 always to 5 never). The tool consists of 14 sections, listed below, that refer to sensory processing, modulation, and behavioral and emotional responses. Subscale scores are listed below. Each section's raw score is compared with a threshold value to determine a category of performance: typical performance, probable difference (1 standard deviation below the mean), and definite difference (2 standard deviations below the mean). A lower raw score means a greater difference.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=10 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Sensory Profile (SP) - Study 2
SP-Vestibular Processing (subscale range 11-55)
|
0.9 units on a scale
Standard Deviation 5.2
|
-0.53 units on a scale
Standard Deviation 4.06
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Touch Processing (subscale range 18-90)
|
3.9 units on a scale
Standard Deviation 4.89
|
0.3 units on a scale
Standard Deviation 5.36
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Multisensory Processing (subscale range 7-35)
|
0.9 units on a scale
Standard Deviation 4.23
|
-0.7 units on a scale
Standard Deviation 3.68
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Oral Sensory Processing (subscale range 12-60)
|
1 units on a scale
Standard Deviation 5.08
|
0.5 units on a scale
Standard Deviation 5.32
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Modulation of Visual Input Affecting Emotional Responses & Activity Level (subscale range 4-20)
|
0.4 units on a scale
Standard Deviation 1.65
|
0.9 units on a scale
Standard Deviation 1.52
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Emotional/Social Responses (subscale range 17-85)
|
3 units on a scale
Standard Deviation 7.85
|
0.1 units on a scale
Standard Deviation 5.3
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Behavioral Outcomes of Sensory Processing (subscale range 6-30)
|
2.83 units on a scale
Standard Deviation 6.59
|
0.11 units on a scale
Standard Deviation 3.55
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Items Indicating Thresholds for Response (subscale range 3-15)
|
1 units on a scale
Standard Deviation 1.55
|
0 units on a scale
Standard Deviation 2
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Auditory Processing (subscale range 8-40)
|
1.6 units on a scale
Standard Deviation 2.46
|
0.2 units on a scale
Standard Deviation 2.39
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Visual Processing (subscale range 9-45)
|
1.3 units on a scale
Standard Deviation 4.92
|
1.5 units on a scale
Standard Deviation 3.69
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Sensory Processing Related to Endurance/Tone (subscale range 9-45)
|
0.5 units on a scale
Standard Deviation 5.93
|
2 units on a scale
Standard Deviation 3.33
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Modulation Related to Body Position and Movement (subscale range 10-50)
|
5 units on a scale
Standard Deviation 4.08
|
-0.4 units on a scale
Standard Deviation 3.24
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Modulation of Movement Affecting Activity Level (subscale range 7-35)
|
0.2 units on a scale
Standard Deviation 3.16
|
1.7 units on a scale
Standard Deviation 5.58
|
—
|
—
|
|
Change in Sensory Profile (SP) - Study 2
SP-Modulation of Sensory Input Affecting Emotional Responses (subscale range 4-20)
|
-0.4 units on a scale
Standard Deviation 1.17
|
0.6 units on a scale
Standard Deviation 1.84
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 12 weeksThe Short Sensory Profile is a caregiver report measure consisting of 38 items, each scored on a 1-point (always) to 5-point (never) Likert scale. SSP Subscales Tactile Sensitivity (7 to 35) Taste/Smell Sensitivity (4 to 20), Movement Sensitivity (3 to 15), Under-Responsive/Seek Sensation (7 to 35) , Auditory Filtering (6 to 30), Low Energy/Weak (6 to 30), Visual/Auditory Sensitivity (5 to 25), with total scale from (38 to 190) Lower scores indicate more sensory alterations.
Outcome measures
| Measure |
Study 1: Insulin-Like Growth Factor-1 (IGF-1)
n=10 Participants
IGF-1 (Increlex; Ipsen Biopharmaceuticals, Inc) is an aqueous solution for injection containing human insulin-like growth factor-1 (rhIGF-1) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily.
|
Study 1: Normal Saline
n=10 Participants
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
Study 2: Insulin-Like Growth
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Study 2: Normal Saline
Placebo consisted of saline prepared in identical bottles by the research pharmacy at Mount Sinai.
|
|---|---|---|---|---|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Tactile Sensitivity
|
0.9 score on a scale
Standard Deviation 2.73
|
0.7 score on a scale
Standard Deviation 2.45
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Taste/Smell Sensitivity
|
-0.2 score on a scale
Standard Deviation 2.57
|
-0.5 score on a scale
Standard Deviation 1.08
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Movement Sensitivity
|
0.4 score on a scale
Standard Deviation 1.26
|
-0.3 score on a scale
Standard Deviation 1.57
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Underresponsive/Seeks Sensation
|
2.87 score on a scale
Standard Deviation 4.47
|
-1.23 score on a scale
Standard Deviation 3.61
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Auditory Filtering
|
0.9 score on a scale
Standard Deviation 2.23
|
0.3 score on a scale
Standard Deviation 2.67
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Low Energy/Weak
|
-0.6 score on a scale
Standard Deviation 3.89
|
1.36 score on a scale
Standard Deviation 1.99
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Visual/Auditory Sensitivity
|
0.8 score on a scale
Standard Deviation 1.23
|
0.8 score on a scale
Standard Deviation 1.55
|
—
|
—
|
|
Change in Short Sensory Profile (SSP) - Study 2
SSP-Total
|
5.07 score on a scale
Standard Deviation 11.43
|
1.13 score on a scale
Standard Deviation 10.32
|
—
|
—
|
Adverse Events
Insulin-Like Growth Factor-1 (IGF-1)
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
Normal Saline
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Insulin-Like Growth Factor-1 (IGF-1)
n=19 participants at risk
IGF-1 is an aqueous solution for injection containing human insulin-like growth factor-1 (Increlex; Ipsen Biopharmaceuticals, Inc)) produced by recombinant DNA technology. Dose titration was initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Medication was administered subcutaneously twice daily with meals and glucose monitoring was performed by parents prior to each injection and at bedtime.
|
Normal Saline
n=19 participants at risk
Placebo
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
36.8%
7/19 • 28 weeks
|
21.1%
4/19 • 28 weeks
|
|
Nervous system disorders
Sedation
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
21.1%
4/19 • 28 weeks
|
26.3%
5/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Periobital/facial swelling
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
10.5%
2/19 • 28 weeks
|
26.3%
5/19 • 28 weeks
|
|
Infections and infestations
URTI
|
47.4%
9/19 • 28 weeks
|
52.6%
10/19 • 28 weeks
|
|
Nervous system disorders
Sleep Disturbance
|
57.9%
11/19 • 28 weeks
|
36.8%
7/19 • 28 weeks
|
|
Metabolism and nutrition disorders
Increased appetite
|
47.4%
9/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Nervous system disorders
Mood changes/irritability
|
47.4%
9/19 • 28 weeks
|
42.1%
8/19 • 28 weeks
|
|
Metabolism and nutrition disorders
Increased thirst
|
5.3%
1/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Infections and infestations
Increased phlegm
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Teeth grinding
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.8%
3/19 • 28 weeks
|
21.1%
4/19 • 28 weeks
|
|
Nervous system disorders
Hand flapping
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Gastrointestinal disorders
Increased bowel movements
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Increased chewing/biting
|
10.5%
2/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Eye disorders
Decreased visual acuity
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Lethargy/decreased energy
|
31.6%
6/19 • 28 weeks
|
21.1%
4/19 • 28 weeks
|
|
Nervous system disorders
Cooler body temperature/sweating
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Infections and infestations
Runny nose/congestion
|
36.8%
7/19 • 28 weeks
|
42.1%
8/19 • 28 weeks
|
|
Nervous system disorders
Gait changes/fell
|
15.8%
3/19 • 28 weeks
|
26.3%
5/19 • 28 weeks
|
|
Infections and infestations
Stomach virus
|
15.8%
3/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Nervous system disorders
Anxiety
|
0.00%
0/19 • 28 weeks
|
10.5%
2/19 • 28 weeks
|
|
Renal and urinary disorders
Increased urine frequency
|
10.5%
2/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Infections and infestations
Fever
|
36.8%
7/19 • 28 weeks
|
26.3%
5/19 • 28 weeks
|
|
Nervous system disorders
Increased energy
|
10.5%
2/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Gastrointestinal disorders
Gagging
|
10.5%
2/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Infections and infestations
Conjunctivitis
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Eye disorders
Erythema/swollen eyes
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Gastrointestinal disorders
Vomiting
|
21.1%
4/19 • 28 weeks
|
15.8%
3/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.8%
3/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Facial swelling
|
10.5%
2/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Endocrine disorders
Warmer body temperature
|
10.5%
2/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Hair loss
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Increased aggression
|
5.3%
1/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Endocrine disorders
Hypoglycemia
|
57.9%
11/19 • 28 weeks
|
63.2%
12/19 • 28 weeks
|
|
Gastrointestinal disorders
Stomach aches
|
0.00%
0/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Bruising at injection site
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Reproductive system and breast disorders
Breast buds developed
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Blood and lymphatic system disorders
Bloody nose
|
5.3%
1/19 • 28 weeks
|
10.5%
2/19 • 28 weeks
|
|
Cardiac disorders
Elevated heart rate
|
0.00%
0/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Ear and labyrinth disorders
Increased ear wax
|
0.00%
0/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Gastrointestinal disorders
Increased reflux
|
5.3%
1/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Infections and infestations
Ear Infections
|
5.3%
1/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Injury, poisoning and procedural complications
Broken bone
|
0.00%
0/19 • 28 weeks
|
5.3%
1/19 • 28 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Increased drooling
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Increased eye rolling
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Nervous system disorders
Seizure
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
|
Skin and subcutaneous tissue disorders
Redness around perineum
|
5.3%
1/19 • 28 weeks
|
0.00%
0/19 • 28 weeks
|
Additional Information
Alexander Kolevzon, MD
Icahn School of Medicine at Mount Sinai
Phone: (212) 659-9134
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place