Trial Outcomes & Findings for The Childhood and Adolescent Migraine Prevention Study (NCT NCT01581281)

Last Updated: 2017-08-10

Results Overview

The primary endpoint was a ≥ 50% reduction in headache frequency from the 28 days (4 weeks) baseline period prior to randomization to the last 28 days (4 weeks) of the trial. Headache frequency was defined as the number of days with headache for a given four week 28 day (4 week) period. A headache day was defined as any day during which any headache occurs within a 24 hour period, starting and ending at midnight. For each participant, the primary endpoint involved a determination of whether a 50% or greater reduction in headache frequency was observed during the last 4 weeks of active treatment as compared with the headache frequency during the 4-week baseline period. Results were compared across the three treatment groups.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

488 participants

Primary outcome timeframe

4 week baseline period and last 4 weeks of the 24-week trial

Results posted on

2017-08-10

Participant Flow

From July 16, 2012 through November 24, 2014, 488 patients were assessed for eligibility from 31 sites in the United States. Of those, 361 patients underwent randomization.

Of the patients assessed for eligibility, 21 (4%) did not undergo randomization owing to early trial closure and 106 (22%) were excluded. Eighty-one (81) were excluded due to not meeting inclusion criteria, 25 declined participation (13 were unwilling, but eligibility was otherwise confirmed and 12 were willing and eligibility was unknown).

Participant milestones

Participant milestones
Measure	Topiramate Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Placebo Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Overall Study STARTED	145	72	144
Overall Study COMPLETED	130	66	132
Overall Study NOT COMPLETED	15	6	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Topiramate Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Placebo Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Overall Study Early Study Closure	15	6	12

Baseline Characteristics

The Childhood and Adolescent Migraine Prevention Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Topiramate n=145 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Placebo n=72 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=144 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Total n=361 Participants Total of all reporting groups
Age, Categorical <=18 years	145 Participants n=5 Participants	72 Participants n=7 Participants	144 Participants n=5 Participants	361 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	14.2 years STANDARD_DEVIATION 2.5 • n=5 Participants	14.2 years STANDARD_DEVIATION 2.2 • n=7 Participants	14.2 years STANDARD_DEVIATION 2.4 • n=5 Participants	14.2 years STANDARD_DEVIATION 2.4 • n=4 Participants
Sex: Female, Male Female	101 Participants n=5 Participants	49 Participants n=7 Participants	97 Participants n=5 Participants	247 Participants n=4 Participants
Sex: Female, Male Male	44 Participants n=5 Participants	23 Participants n=7 Participants	47 Participants n=5 Participants	114 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	14 Participants n=5 Participants	6 Participants n=7 Participants	8 Participants n=5 Participants	28 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	123 Participants n=5 Participants	65 Participants n=7 Participants	128 Participants n=5 Participants	316 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	8 Participants n=5 Participants	1 Participants n=7 Participants	8 Participants n=5 Participants	17 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	14 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants	27 Participants n=4 Participants
Race (NIH/OMB) Asian	6 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	6 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Black or African American	24 Participants n=5 Participants	17 Participants n=7 Participants	26 Participants n=5 Participants	67 Participants n=4 Participants
Race (NIH/OMB) White	98 Participants n=5 Participants	48 Participants n=7 Participants	107 Participants n=5 Participants	253 Participants n=4 Participants
Race (NIH/OMB) More than one race	NA Participants n=5 Participants	NA Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants
Region of Enrollment United States	145 Participants n=5 Participants	72 Participants n=7 Participants	144 Participants n=5 Participants	361 Participants n=4 Participants
Headache Days	11.5 days STANDARD_DEVIATION 6.1 • n=5 Participants	11.0 days STANDARD_DEVIATION 6.3 • n=7 Participants	11.5 days STANDARD_DEVIATION 6.2 • n=5 Participants	11.4 days STANDARD_DEVIATION 6.1 • n=4 Participants
Pediatric Migriane Disability Assessment Score (PedMIDAS)	42.6 units on a scale STANDARD_DEVIATION 27.4 • n=5 Participants	42.9 units on a scale STANDARD_DEVIATION 26.7 • n=7 Participants	40.6 units on a scale STANDARD_DEVIATION 26.4 • n=5 Participants	41.9 units on a scale STANDARD_DEVIATION 26.8 • n=4 Participants

PRIMARY outcome

Timeframe: 4 week baseline period and last 4 weeks of the 24-week trial

Population: The primary endpoint involved a determination of whether a 50% or greater reduction in headache frequency was observed during the last 4 weeks of active treatment as compared with the headache frequency during the 4-week baseline period between the participants receiving amitriptyline and participants receiving placebo.

Outcome measures

Outcome measures
Measure	Topiramate n=130 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved	Placebo n=66 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=132 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Number (Percentage) of Participants Reporting a ≥ 50% Reduction in Headache Days	72 Participants	40 Participants	69 Participants

SECONDARY outcome

Timeframe: baseline and 24 week endpoint

The PedMIDAS scale which evaluated the impact of headaches in school, home, play, and social activities, is comprised of six items that pertain to days missed in various activities over the past 90 days. Questions were answered by the youth in consultation with their parents and reviewed by study staff. The PedMIDAS scale was administered at baseline (covering the three months prior to enrollment) and at the 24-week endpoint visit (the end of the maintenance period, covering three months of enrollment). A total PedMIDAS score (sum of items 1-6) was used in this trial. Scores range from 0-240; with a score of 0-10 indicating no disability, 11-30 mild disability, 31-50 moderate disability, and more than 50 severe disability in daily activities. The main outcome measure for this comparison will be the difference in the baseline and endpoint (24 week) PedMIDAS total scores for: 1. Amitriptyline vs. Placebo 2. Topiramate vs. Placebo 3. Amitriptyline vs Topiramate

Outcome measures

Outcome measures
Measure	Topiramate n=130 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved	Placebo n=66 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=132 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Change in Absolute Headache Disability Score on PedMIDAS	-26.8 units on a scale Standard Deviation 27.5	-22.6 units on a scale Standard Deviation 29.42	-22.5 units on a scale Standard Deviation 26.37

SECONDARY outcome

Timeframe: 4 week baseline period and last 4 weeks of the 24-week trial

Population: The analysis population included all participants who had observed end-point data: 101 in the topiramate group, 59 in the placebo group, and104 in the amitriptyline group.

This outcomes measure examines whether the rate of absolute number of headache days, per 28 day period, differs between treatment groups over time. This was assessed longitudinally based on the actual number of headache days from the 28 days prior to randomization to the last 28 days of this 24 week trial. The change in absolute headache days was compared between: 1. Amitriptyline vs. placebo 2. Topiramate vs. placebo 3. Amitriptyline vs. Topiramate

Outcome measures

Outcome measures
Measure	Topiramate n=101 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved	Placebo n=59 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=104 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Change in Number of Headache Days	-6.7 days Standard Deviation 4.99	-5.9 days Standard Deviation 6.96	-6.7 days Standard Deviation 6.20

SECONDARY outcome

Timeframe: 24 weeks

Population: Tolerability was assessed for all participants included in the primary analysis. Of those randomized, 33 were not included in the primary analysis due to early trial closure.

To assess tolerability, the percentage of subjects who complete the entire 24-week treatment period will be estimated in each of the three groups.

Outcome measures

Outcome measures
Measure	Topiramate n=130 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved	Placebo n=66 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=132 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Tolerability, as Indicated by the Number (Percentage) of Participants That Completed the 24-week Treatment Phase	102 Participants	59 Participants	106 Participants

SECONDARY outcome

Timeframe: 24 weeks of the trial

To determine if amitriptyline or topiramate differ from placebo on the occurrence of treatment emergent serious adverse events.

Outcome measures

Outcome measures
Measure	Topiramate n=145 Participants Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved	Placebo n=72 Participants Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=144 Participants Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Occurrence of Treatment Emergent Serious Adverse Events	4 serious adverse events	2 serious adverse events	6 serious adverse events

Adverse Events

Topiramate

Serious events: 4 serious events

Other events: 111 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 51 other events

Deaths: 0 deaths

Amitriptyline

Serious events: 6 serious events

Other events: 106 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Topiramate n=145 participants at risk Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Placebo n=72 participants at risk Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=144 participants at risk Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Gastrointestinal disorders Intussusception	0.69% 1/145 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Injury, poisoning and procedural complications Contusion	0.69% 1/145 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Injury, poisoning and procedural complications Traumatic Liver Injury	0.69% 1/145 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Psychiatric disorders Suicide attempt	0.69% 1/145 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Infections and infestations Appendicitis	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	1.4% 1/72 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Infections and infestations Pharyngitis streptococcal	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	1.4% 1/72 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Immune system disorders Anaphylactic reaction	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.69% 1/144 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Nervous system disorders Syncope	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.69% 1/144 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Psychiatric disorders Mood altered	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	2.1% 3/144 • Number of events 3 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Respiratory, thoracic and mediastinal disorders Bronchospasm	0.00% 0/145 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.69% 1/144 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.

Other adverse events

Other adverse events
Measure	Topiramate n=145 participants at risk Topiramate enclosed in capsules to maintain the blind was administered orally in a divided dose of 1 capsule twice daily. A target dose was 2 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.	Placebo n=72 participants at risk Placebo enclosed in capsules to maintain the blind was administered orally twice daily during an 8 week titration period followed by a 16 week maintenance phase (mirroring the other two treatment arms).	Amitriptyline n=144 participants at risk Amitriptyline enclosed in capsules to maintain the blind was administered orally in dose of 1 capsule twice daily (AM capsule did not contain medication. A target dose was 1 mg per kilogram per day. Dose escalation occurred every two weeks over a period of 8 weeks, with dose modification based on side effects. A 16 week constant-dose (maintenance) phase followed at the highest dosage achieved.
Nervous system disorders Asphasia	15.9% 23/145 • Number of events 25 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	9.7% 7/72 • Number of events 9 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	9.0% 13/144 • Number of events 16 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Nervous system disorders Cognitive Disorder	15.9% 23/145 • Number of events 25 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	11.1% 8/72 • Number of events 8 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	9.7% 14/144 • Number of events 18 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Nervous system disorders Dizziness	6.2% 9/145 • Number of events 9 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	1.4% 1/72 • Number of events 2 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	2.1% 3/144 • Number of events 3 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Nervous system disorders Memory Impairment	16.6% 24/145 • Number of events 29 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	9.7% 7/72 • Number of events 8 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	7.6% 11/144 • Number of events 11 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Nervous system disorders Paresthesia	31.0% 45/145 • Number of events 67 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	8.3% 6/72 • Number of events 6 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	6.9% 10/144 • Number of events 10 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
General disorders Fatigue	24.8% 36/145 • Number of events 40 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	13.9% 10/72 • Number of events 12 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	29.9% 43/144 • Number of events 48 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Gastrointestinal disorders Dry Mouth	17.9% 26/145 • Number of events 30 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	12.5% 9/72 • Number of events 9 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	25.0% 36/144 • Number of events 45 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Infections and infestations Streptococcal Pharyngitis	0.69% 1/145 • Number of events 1 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	4.2% 3/72 • Number of events 3 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	4.9% 7/144 • Number of events 7 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Infections and infestations Upper Respiratory Tract Infection	12.4% 18/145 • Number of events 19 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	13.9% 10/72 • Number of events 10 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	9.7% 14/144 • Number of events 16 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Psychiatric disorders Mood Altered	9.7% 14/145 • Number of events 16 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	5.6% 4/72 • Number of events 4 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	5.6% 8/144 • Number of events 8 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.
Investigations Decreased Weight	7.6% 11/145 • Number of events 11 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/72 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.	0.00% 0/144 • Adverse events were collected after the ingestion of at least one dose of study drug through study completion, an average of 24 weeks.

Additional Information

Scott Powers, PhD

Cincinnati Children's Hospital Medical Center

Phone: 513-636-8106

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place