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Open-Label Study of N-Acetylcysteine in Children and Adolescents 5-17 With Bipolar Spectrum Disorders

Primary Purpose

Bipolar Disorder

Status

Completed

Phase

Phase 4

Locations

United States

Study Type

Interventional

Intervention

Open-label Treatment with N-Acetylcysteine

About this trial

This is an interventional treatment trial for Bipolar Disorder focused on measuring mania, temper tantrum, irritability, mood, bipolar

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female subjects, 5-17 years of age
Subjects must have a DSM-IV diagnosis of a bipolar spectrum disorder (type I, II, or Not Otherwise Specified) and currently display mixed, manic, or hypomanic symptoms (without psychotic features) according to clinical assessment based on the DSM-IV and confirmed with structured diagnostic interview
Subjects and their legal representative must have a level of understanding sufficient to communicate intelligently with the investigator and study coordinator and to cooperate with all tests and examinations required by the protocol
Subjects and their legal representatives must be considered reliable.
Each subject and his/her authorized legal representative must understand the nature of the study. The subject's authorized legal representative must provide written consent and the subject must provide written assent.
Subjects must have an initial score on the YMRS of at least 15.

Exclusion Criteria:

Investigator and his/her immediate family (defined as the investigator's spouse, parent, child, grandparent, or grandchild)
Serious or unstable illness including hepatic, rental, gastroenterological, respiratory, cardiovascular (including ischemic heart disease), endocrinologic, neurologic, immunologic, or hematologic disease
Uncorrected hypothyroidism or hyperthyroidism
History of sensitivity to N-acetylcysteine, a history of intolerance to N-acetylcysteine, or a non-responder after 2 months of treatment at adequate doses as determined by the clinician
Severe allergies or multiple adverse drug reactions
Current or past history of seizures
Active substance abusers, per clinician judgment
Judged clinically to be at serious suicidal risk
Current diagnosis of schizophrenia
Pregnancy
C-SSRS score ≥ 4
IQ < 70

Sites / Locations

Massachusetts General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-label Treatment with NAC

Arm Description

12-week, open-label treatment with NAC. Subjects will be treated with the following dose: Subjects ages 5-12: Week 1: 900mg po daily Weeks 2+: 900mg po QAM, 900mg po QPM Subjects ages 13-17: Week 1: 900mg po daily Weeks 2-3: 900mg po QAM, 900mg po QPM Weeks 4+: 1800mg po QAM, 900mg po QPM In Weeks 3-12 for subjects ages 13-17, we will encourage twice per day dosing, but we will permit daily dosing if needed for adherence.

Outcomes

Primary Outcome Measures

Mean Change in the Young Mania Rating Scale (YMRS) Score

The Young Mania Rating Scale (YMRS) consists of 7 items rated on a scale from 0 (symptoms not present) to 4 (symptoms extremely severe) and 4 items rated on a scale from 0 (symptom not present) to 8 (symptom extremely severe).The YMRS score ranges from 0-60. Questions are asked about the last week. A higher score signifies more severe manic symptoms.

Secondary Outcome Measures

Mean Change in the Children's Depression Rating Scale (CDRS) Score

The Children's Depression Rating Scale (CDRS) is a clinician-rated instrument with 17 items scored on a 1 to 5 or 1 to 7 scale. A rating of 1 indicates normal, thus the minimum score is 17. The maximum score is 113. Scores of 20-30 suggest borderline depression. Scores of 40-60 indicate moderate depression.

Full Information

NCT ID

NCT02357290

First Posted

February 2, 2015

Last Updated

October 15, 2019

Sponsor

Massachusetts General Hospital

1. Study Identification

Unique Protocol Identification Number

NCT02357290

Brief Title

Open-Label Study of N-Acetylcysteine in Children and Adolescents 5-17 With Bipolar Spectrum Disorders

Official Title

Open-Label Study of N-Acetylcysteine in Children and Adolescents Ages 5-17 With Bipolar I, Bipolar II, and Bipolar Spectrum Disorder

Study Type

Interventional

2. Study Status

Record Verification Date

October 2019

Overall Recruitment Status

Completed

Study Start Date

January 2014 (Actual)

Primary Completion Date

September 24, 2018 (Actual)

Study Completion Date

September 24, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Massachusetts General Hospital

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Children between the ages of 5-17 years old who have or display symptoms of emotional dysregulation (explosiveness, mood swings, irritability, and/or violent behavior) are invited to participate in a 12-week research study to determine the effectiveness and safety of the natural treatment N-acetylcysteine (NAC) for children with emotional and behavioral problems. After undergoing a comprehensive evaluation by medical doctors with a specialty in this area, children who are found eligible to participate in this research study will be treated with NAC. Following the evaluation period, this research study requires 12 weekly visits, either in our office or over the phone, in an effort to closely monitor each child's response to the medication. Eligible participants will receive study-related evaluations and weekly study visits with our study doctors at no cost.

Detailed Description

This will be a 12-week, open-label study of NAC in the treatment of bipolar disorder in children and adolescents. Subjects will include youth ages 5-17 years with a bipolar spectrum disorder (type I, II, or NOS), mixed, manic, or hypomanic state, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition DSM-5 (American Psychiatric Association, 2013). The primary outcome measures will be improvement in manic symptoms as measured by the Young Mania Rating Scale (YMRS) and improvement in depressive symptoms as measured by the Child Depression Rating Scale (CDRS). Bipolar diagnoses will be made according to the DSM-5 in a clinical evaluation by a Child Psychiatrist and confirmed using the Schedule for Affective Disorders and Schizophrenia for School-Age Children - Epidemiological Version (K-SADS-E)(Orvaschel, 1994). All subjects must have a YMRS score of at least 15. Only patients who are not responding to their current treatment regimen will be tapered from their medications; youth on concomitant psychiatric medications will be permitted to continue those medications as listed in the concomitant medication section.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Bipolar Disorder

Keywords

mania, temper tantrum, irritability, mood, bipolar

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

40 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Open-label Treatment with NAC

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Open-label Treatment with N-Acetylcysteine

Primary Outcome Measure Information:

Title

Mean Change in the Young Mania Rating Scale (YMRS) Score

Description

Time Frame

Baseline and Endpoint (12 weeks or last observation carried forward if dropped prior to week 12)

Secondary Outcome Measure Information:

Title

Mean Change in the Children's Depression Rating Scale (CDRS) Score

Description

Time Frame

Baseline and Endpoint (12 weeks or last observation carried forward if dropped prior to week 12)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female subjects, 5-17 years of age Subjects must have a DSM-IV diagnosis of a bipolar spectrum disorder (type I, II, or Not Otherwise Specified) and currently display mixed, manic, or hypomanic symptoms (without psychotic features) according to clinical assessment based on the DSM-IV and confirmed with structured diagnostic interview Subjects and their legal representative must have a level of understanding sufficient to communicate intelligently with the investigator and study coordinator and to cooperate with all tests and examinations required by the protocol Subjects and their legal representatives must be considered reliable. Each subject and his/her authorized legal representative must understand the nature of the study. The subject's authorized legal representative must provide written consent and the subject must provide written assent. Subjects must have an initial score on the YMRS of at least 15. Exclusion Criteria: Investigator and his/her immediate family (defined as the investigator's spouse, parent, child, grandparent, or grandchild) Serious or unstable illness including hepatic, rental, gastroenterological, respiratory, cardiovascular (including ischemic heart disease), endocrinologic, neurologic, immunologic, or hematologic disease Uncorrected hypothyroidism or hyperthyroidism History of sensitivity to N-acetylcysteine, a history of intolerance to N-acetylcysteine, or a non-responder after 2 months of treatment at adequate doses as determined by the clinician Severe allergies or multiple adverse drug reactions Current or past history of seizures Active substance abusers, per clinician judgment Judged clinically to be at serious suicidal risk Current diagnosis of schizophrenia Pregnancy C-SSRS score ≥ 4 IQ < 70

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Janet Wozniak, MD

Organizational Affiliation

Massachusetts General Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

35505312

Citation

Wozniak J, DiSalvo M, Farrell A, Vaudreuil C, Uchida M, Ceranoglu TA, Joshi G, Cook E, Faraone SV, Biederman J. Findings from a pilot open-label trial of N-acetylcysteine for the treatment of pediatric mania and hypomania. BMC Psychiatry. 2022 May 3;22(1):314. doi: 10.1186/s12888-022-03943-x.

Results Reference

derived

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Open-Label Study of N-Acetylcysteine in Children and Adolescents 5-17 With Bipolar Spectrum Disorders

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