Trial Outcomes & Findings for Cysteamine Therapy for Major Depressive Disorder (NCT NCT00715559)

NCT ID: NCT00715559

Last Updated: 2017-04-07

Results Overview

This scale measures depression severity. It ranges from a score of 0 to 60, with higher score indicating higher level of depression severity.

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 3 participants
• Primary outcome timeframe: 8 weeks
• Results posted on: 2017-04-07

Participant Flow

A total of 3 participants were recruited between June 2007 and May 2009.

Participants with major depression were enrolled if they had previously failed to respond to at least one FDA-approved antidepressant. There are no prospective treatment or lead-in and the study was conducted open-label.

Participant milestones

Measure	Cysteamine Bitartrate Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Overall Study STARTED	3
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Cysteamine Bitartrate Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Overall Study medication not tolerated - nausea	1

Baseline Characteristics

Cysteamine Therapy for Major Depressive Disorder

Baseline characteristics by cohort

Measure	Cysteamine Bitartrate n=3 Participants Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	47 years STANDARD_DEVIATION 15.6 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 weeks

Population: Mean MADRS score at end of treatment (LOCF) in 3 participants treated with cysteamine bitartrate.

This scale measures depression severity. It ranges from a score of 0 to 60, with higher score indicating higher level of depression severity.

Outcome measures

Measure	Cysteamine Bitartrate n=3 Participants Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Montgomery-Åsberg Depression Rating Scale (MADRS)	27 scale score Standard Deviation 3

SECONDARY outcome

Timeframe: 8 weeks

This set of scales measures "global" improvement in a patient's level of symptoms, without reference to a particular condition (ie depression). GCI-S is a measure of severity, which ranges from 0 (not ill) to 7 (severely ill). CGI-I is a measure of change, with a score of 4 indicating no change, 1 indicating very much improved and 7 indicating very much worse.

Outcome measures

Measure	Cysteamine Bitartrate n=3 Participants Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Clinical Global Impression Scales for Severity (CGI-S) and Improvement (CGI-I)	4 scale score Standard Deviation 0

SECONDARY outcome

Timeframe: 8 weeks

This is a self-report which measures the level of depression severity. I ranges from 0 (no illness) to 27 (severe illness).

Outcome measures

Measure	Cysteamine Bitartrate n=3 Participants Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Quick Inventory of Depressive Symptomatology-Self-Report (QIDS-SR16)	13.3 scale score Standard Deviation 2.9

SECONDARY outcome

Timeframe: weekly, for 8 weeks

Population: Study participants were assessed for side effects or adverse events with the SAFTEE at each study visit over the 8 week trial.

The SAFTEE is used to measure somatic and other symptoms which may arise during the course of a clinical trial. This is a non-quantitative instrument that does not yield a numeric score. Instead, it provides study subjects the opportunity to check off symptoms listed on a checklist and indicate if the severity of the symptoms is "mild" "moderate" or "severe." The reported values represent symptoms that were indicated at any point during the 8 week trial at a level of "moderate" or "severe" that also represented a change from a baseline-line pre-intervention SAFTEE assessment.

Outcome measures

Measure	Cysteamine Bitartrate n=3 Participants Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Systematic Assessment for Treatment Emergent Effects (SAFTEE)	24.3 symptoms Standard Deviation 17.5

Adverse Events

Cysteamine Bitartrate

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Cysteamine Bitartrate n=3 participants at risk Study participants received cysteamine bitartrate by mouth up to 300 mg three times daily
Nervous system disorders Numbness or tingling	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Nervous system disorders Dizziness	66.7% 2/3 • Number of events 2 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Nervous system disorders Headache	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
General disorders Dry mouth	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Gastrointestinal disorders Drooling	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Gastrointestinal disorders Nausea	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Gastrointestinal disorders Stomach discomfort	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Reproductive system and breast disorders loss of sexual interest	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Gastrointestinal disorders appetite decrease	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Nervous system disorders word finding difficulty	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Vascular disorders bruising	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Endocrine disorders hot flashes	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.
Psychiatric disorders apathy	33.3% 1/3 • Number of events 1 • 8 weeks Adverse events are recorded with the SAFTEE at each weekly visit during the 8 week study. All items reported here are endorsed with a severity greater than mild (e.g. moderate or severe) and represent a change from baseline.

Additional Information

Dr. James Murrough

Mount Sinai School of Medicine

Phone: 212-241-7574

Email: james.murrough@mssm.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place