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

NCT02357290 | Completed Phase 4 Results

• 1 other identifier: 2014P002011
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Bipolar Disorder

Keywords

mania; temper tantrum; irritability; mood; bipolar

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (1)

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

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

Study Arms (1)

Open-label Treatment with NAC

EXPERIMENTAL

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.

Drug: Open-label Treatment with N-Acetylcysteine

Interventions

Open-label Treatment with N-Acetylcysteine DRUG

Open-label Treatment with NAC

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Related Publications (1)

• 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.

  PMID: 35505312 DERIVED

MeSH Terms

Conditions

Bipolar Disorder; Mania

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Bipolar and Related Disorders; Mood Disorders; Mental Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Dr. Janet Wozniak
• Organization: Massachusetts General Hospital

jwozniak@partners.org

617-724-5600

Study Officials

• Janet Wozniak, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Psychiatry, Harvard Medical School; Medical Director, Child and Adolescent Outpatient Psychiatry Services; Director, Pediatric Bipolar Clinical and Research Program in Pediatric Psychopharmacology, Massachusetts General Hospital

Study Record Dates

• First Submitted: February 2, 2015
• First Posted: February 6, 2015
• Study Start: January 1, 2014
• Primary Completion: September 24, 2018
• Study Completion: September 24, 2018
• Last Updated: November 4, 2019
• Results First Posted: November 4, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)