Trial · NCT01777542
Treatment of Rett Syndrome With Recombinant Human IGF-1
NCT01777542, Phase 2, Completed, Boston Children's Hospital; results first posted 2018-03-26
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ConductScience Library. Treatment of Rett Syndrome With Recombinant Human IGF-1. Trials, record NCT01777542. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01777542
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Rett Syndrome With Recombinant Human IGF-1
- NCT number
- NCT01777542
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Boston Children's Hospital
- Conditions
- Rett Syndrome
- Interventions
- Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo
- First posted
- 2013-01-29
- Last updated
- 2018-03-26
- Results first posted
- 2018-03-26
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The RSBQ is an informant/parent-completed measure of abnormal behaviors typically observed in individuals with RTT, which is completed by a parent/caregiver/LAR. Each item, grouped into eight domains/factors: General mood, Breathing problems, Body rocking and expressionless face, Hand behaviors, Repetitive face movements, Night-time behaviors, Fear/anxiety and Walking/standing), is scored on a Likert scale of 0-2, according to how well the item describes the individual's behavior. A score of "0" indicates the described item is "not true," a score of "1" indicates the described item is "somewhat or sometimes true," and a score of "2" indicates the described item is "very true or often true." The total sum of items in each subscale is reported. For the fear/anxiety subscale, the sum total could be between 0-8. The higher the sum total score, the greater the frequency of fear/anxiety behaviors. |
| Primary | Anxiety, Depression, and Mood Scale (ADAMS) - Social Avoidance Subscale | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The ADAMS is completed by the parent/caregiver/LAR and consists of 29 items which are scored on a 4-point rating scale that combines frequency and severity ratings. The instructions ask the rater to describe the individual's behavior over the last six months on the following scale: "0" if the behavior has not occurred, "1" if the behavior occurs occasionally or is a mild problem, "2" if the behavior occurs quite often or is moderate problem, or "3" if the behavior occurs a lot or is a severe problem. The Social Avoidance subscale of the ADAMS will be used as a primary outcome measure for this trial. The range for this subscale is 0-21. The higher the subscale score, the more problematic the behavior. |
| Primary | Clinical Global Impression - Severity (CGI-S) | Every 10 weeks during each of the two 20-week treatment periods | This scale is used to judge the severity of the subject's disease prior to entry into the study. The clinician will rate the severity of behavioral symptoms at baseline on a 7-point scale from not impaired to the most impaired. The scores that correspond to each possible grouping are as follows: 1=Normal, not at all impaired; 2=Borderline impaired; 3=Mildly impaired; 4=Moderately impaired; 5=Markedly impaired; 6=Severely impaired; 7=The most impaired. The possible range for reported scores is 1-7. |
| Primary | Clinical Global Impression - Improvement (CGI-I) | Every 10 weeks during each of the two 20-week treatment periods | Each time the patient was seen after the study intervention was initiated, the clinician compared the patient's overall clinical condition to the CGI-S score obtained at the baseline (visit 1) visit. Based on information collected, the clinician determined if any improvement occurred on the following 7-point scale: 1=Very much improved since the initiation of treatment; 2=Much improved; 3=Minimally improved; 4=No change from baseline (the initiation of treatment); 5=Minimally worse; 6=Much worse; 7=Very much worse since the initiation of treatment. The possible range for reported scores is 1-7. |
| Primary | Parental Global Impression - Severity (PGI-S) | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The PGI-S is the parent version of the CGI-S. Parents/caregivers/LAR are asked to rate the severity of their child's symptoms at baseline on a 7-point scale from not at all impaired to the most impaired. The parents/caregivers/LAR will complete the PGI-S at each study visit. The scores that correspond to each possible grouping are as follows: 1=Normal, not at all impaired; 2=Borderline impaired; 3=Mildly impaired; 4=Moderately impaired; 5=Markedly impaired; 6=Severely impaired; 7=The most impaired. The possible range for reported scores is 1-7. |
| Primary | Parental Global Impression - Improvement (PGI-I) | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | As part of each visit after the study intervention was initiated, the parent/caregiver was asked to compare the patient's overall clinical condition to the score obtained at the baseline (visit 1) visit. Based on information collected, the clinician determined if any improvement occurred on the following 7-point scale: 1=Very much improved since the initiation of treatment; 2=Much improved; 3=Minimally improved; 4=No change from baseline (the initiation of treatment); 5=Minimally worse; 6=Much worse; 7=Very much worse since the initiation of treatment. The possible range for reported scores is 1-7. |
| Primary | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 1 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The parent or caretaker identifies the three most troublesome, RTT-specific, "target" symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS). The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom. |
| Primary | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 2 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The parent or caretaker identifies the three most troublesome, RTT-specific, "target" symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS). The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom. |
| Primary | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 3 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | The parent or caretaker identifies the three most troublesome, RTT-specific, "target" symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS). The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom. |
| Primary | Kerr Clinical Severity Scale | At the start and end of each 20-week treatment period | The Kerr clinical severity scale (Kerr scale) is a quantitative measure of global disease severity. The Kerr scale is a summation of individual items related to Rett syndrome phenotypic characteristics. The items are based on the severity or degree of abnormality of each characteristic on a discrete scale (0, 1, 2) with the highest level corresponding to the most severe or most abnormal presentations. The possible range of scores is 0-48. The higher the score, the more severe the symptoms. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Anxiety, Depression, and Mood Scale (ADAMS) - Social Avoidance Subscale | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Clinical Global Impression - Severity (CGI-S) | Every 10 weeks during each of the two 20-week treatment periods | One participant from the rhIGF-1 First, Then Placebo group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point. | units on a scale | MEDIAN |
| PRIMARY | Clinical Global Impression - Improvement (CGI-I) | Every 10 weeks during each of the two 20-week treatment periods | Data was not collected at Visit 1 because participants had not yet been exposed to either intervention.~One participant from the rhIGF-1 First, Then Placebo group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point. | units on a scale | MEDIAN |
| PRIMARY | Parental Global Impression - Severity (PGI-S) | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Parental Global Impression - Improvement (PGI-I) | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | Data was not collected at visit 1 because participants had not yet been exposed to either intervention.~In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 1 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 2 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Parent Targeted Visual Analog Scale (PTSVAS) - Scale 3 | Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms. | units on a scale | MEDIAN |
| PRIMARY | Kerr Clinical Severity Scale | At the start and end of each 20-week treatment period | In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.~One participant from the rhIGF-1 First, Then Placebo group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point. | units on a scale | MEDIAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group678
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Placebo First, Then rhIGF-1 | Visit 1 - First Intervention | 4.00 | Inter-Quartile Range 3.00 to 6.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | rhIGF-1 First, Then Placebo | Visit 1 - First Intervention | 5.00 | Inter-Quartile Range 2.00 to 6.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Placebo First, Then rhIGF-1 | Visit 2 - First Intervention | 5.00 | Inter-Quartile Range 3.00 to 6.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | rhIGF-1 First, Then Placebo | Visit 2 - First Intervention | 3.00 | Inter-Quartile Range 2.00 to 5.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Placebo First, Then rhIGF-1 | Visit 3 - First Intervention | 4.00 | Inter-Quartile Range 3.00 to 5.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | rhIGF-1 First, Then Placebo | Visit 3 - First Intervention | 3.00 | Inter-Quartile Range 2.00 to 5.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Placebo First, Then rhIGF-1 | Visit 4 - First Intervention | 4.00 | Inter-Quartile Range 2.00 to 5.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | rhIGF-1 First, Then Placebo | Visit 4 - First Intervention | 3.00 | Inter-Quartile Range 1.00 to 6.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | Placebo First, Then rhIGF-1 | Visit 5 - First Intervention | 3.00 | Inter-Quartile Range 2.00 to 5.00 | units on a scale |
| Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale | rhIGF-1 First, Then Placebo | Visit 5 - First Intervention | 3.00 | Inter-Quartile Range 1.00 to 5.00 | units on a scale |
1 to 10 of 678Source ClinicalTrials.gov
See all 678 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| First Intervention (20 Weeks) | STARTED | Placebo First, Then rhIGF-1 | 15 | |
| First Intervention (20 Weeks) | STARTED | rhIGF-1 First, Then Placebo | 15 | |
| First Intervention (20 Weeks) | COMPLETED | Placebo First, Then rhIGF-1 | 15 | |
| First Intervention (20 Weeks) | COMPLETED | rhIGF-1 First, Then Placebo | 15 | |
| First Intervention (20 Weeks) | NOT COMPLETED | Placebo First, Then rhIGF-1 | 0 | |
| First Intervention (20 Weeks) | NOT COMPLETED | rhIGF-1 First, Then Placebo | 0 | |
| Washout Period (28 +/- 2 Weeks) | STARTED | Placebo First, Then rhIGF-1 | 15 | |
| Washout Period (28 +/- 2 Weeks) | STARTED | rhIGF-1 First, Then Placebo | 15 | |
| Washout Period (28 +/- 2 Weeks) | COMPLETED | Placebo First, Then rhIGF-1 | 15 | |
| Washout Period (28 +/- 2 Weeks) | COMPLETED | rhIGF-1 First, Then Placebo | 15 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events153
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abnormal ECG - Prolonged QT | Cardiac disorders | Placebo | 0 | 30 | 0 |
| other | Abnormal ECG - Prolonged QT | Cardiac disorders | rhIGF-1 | 0 | 30 | 0 |
| other | Abnormal ECG - Prolonged QT | Cardiac disorders | Off Treatment | 2 | 30 | 2 |
| other | Adenoidectomy | Surgical and medical procedures | Placebo | 0 | 30 | 0 |
| other | Adenoidectomy | Surgical and medical procedures | rhIGF-1 | 1 | 30 | 1 |
| other | Adenoidectomy | Surgical and medical procedures | Off Treatment | 0 | 30 | 0 |
| other | Advanced Bone Age | Endocrine disorders | Placebo | 0 | 30 | 0 |
| other | Advanced Bone Age | Endocrine disorders | rhIGF-1 | 1 | 30 | 1 |
| other | Advanced Bone Age | Endocrine disorders | Off Treatment | 0 | 30 | 0 |
| other | Anxiety | Psychiatric disorders | Placebo | 1 | 30 | 1 |
1 to 10 of 153Source ClinicalTrials.gov
See all 153 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 30 |
| Total, all-cause mortality | rhIGF-1 | 0 | 30 |
| Total, all-cause mortality | Off Treatment | 0 | 30 |
| Total, other adverse events | Placebo | 16 | 30 |
| Total, other adverse events | rhIGF-1 | 20 | 30 |
| Total, other adverse events | Off Treatment | 15 | 30 |
| Total, serious adverse events | Placebo | 4 | 30 |
| Total, serious adverse events | rhIGF-1 | 4 | 30 |
| Total, serious adverse events | Off Treatment | 2 | 30 |
Source ClinicalTrials.gov
Papers57
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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