Trial Outcomes & Findings for A Double-Blind, Placebo Controlled Study of Intravenous Immunoglobulin for HIV-Associated Myelopathy (NCT NCT01561755)

Results Overview

Strength score as measured by pounds of force sustained in lower extremity strength.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

12 participants

Primary outcome timeframe

at 2 months

Results posted on

2018-04-13

Participant Flow

Participants were recruited from three main sources: the clinical NeuroAIDS practices of the investigators; pre-existing observational research studies (Manhattan HIV Brain Bank and the CNS HIV Antiretroviral Effects Research (CHARTER)); and via a referral network of regional HIV care providers. for a total of four years 2012-2016.

Participant milestones

Participant milestones
Measure	Intravenous Immunoglobulin Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo Saline 2gr/kg over 2 days of saline
Overall Study STARTED	6	6
Overall Study COMPLETED	6	5
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Intravenous Immunoglobulin Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo Saline 2gr/kg over 2 days of saline
Overall Study Lost to Follow-up	0	1

Baseline Characteristics

A Double-Blind, Placebo Controlled Study of Intravenous Immunoglobulin for HIV-Associated Myelopathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=6 Participants Saline 2gr/kg over 2 days of saline	Total n=12 Participants Total of all reporting groups
Age, Continuous	48 years n=5 Participants	58 years n=7 Participants	48 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Number of participant with undetectable Viral Load	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
mean CD4+	620 cells/mm^3 STANDARD_DEVIATION 223 • n=5 Participants	564 cells/mm^3 STANDARD_DEVIATION 270 • n=7 Participants	592 cells/mm^3 STANDARD_DEVIATION 212 • n=5 Participants

PRIMARY outcome

Timeframe: at 2 months

Strength score as measured by pounds of force sustained in lower extremity strength.

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Strength Scores Hip Flexion	14.0 pounds of force Standard Deviation 32.7	1.9 pounds of force Standard Deviation 13.4
Strength Scores Knee Flexion	12.4 pounds of force Standard Deviation 14.4	10.3 pounds of force Standard Deviation 14.5
Strength Scores Ankle Dorsiflexion	1.7 pounds of force Standard Deviation 8.2	-6.0 pounds of force Standard Deviation 5.2
Strength Scores Lower extremity - sum of all three	28.2 pounds of force Standard Deviation 49.2	6.2 pounds of force Standard Deviation 28.9

SECONDARY outcome

Timeframe: at 2 months

Population: One participant in placebo group in wheelchair and unable to complete the walking test

2 minute walking test

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=4 Participants Saline 2gr/kg over 2 days of saline
Walking Test	93.49 meters Standard Deviation 42.57	112.08 meters Standard Deviation 102.00

SECONDARY outcome

Timeframe: 2 months

0 = Normal bladder function, 1 = Asymptomatic on current treatment, 2 = Urinary frequency, hesitancy, urgency, with no incontinence, 3 = Occasional urinary incontinence (once or more during the last month but not every week) or intermittent catheterization, 4 = Frequent urinary incontinence, or occasional incontinence despite regular catheterization, 5 = Daily urinary incontinence or permanent catheter

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Urinary Function	2.6 units on a scale Interval 0.0 to 5.0	2.5 units on a scale Interval 0.0 to 5.0

SECONDARY outcome

Timeframe: 2 months

0 = No bowel problems, 1 = Asymptomatic on current drug tx or constipation not requiring any tx, 2 = Constipation requiring laxative or suppositories or fecal urgency, 3 = Constipation requiring the use of an enema, 4 = Constipation requiring manual evacuation of stools or occasional fecal incontinence (once or more during the last month but not every week, 5 = Weekly fecal incontinence

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Bowel Function	1.3 units on a scale Interval 0.0 to 5.0	2.07 units on a scale Interval 0.0 to 5.0

SECONDARY outcome

Timeframe: at 2 months

0 = Normal strength; 1 = minor symptoms but capable of running; 2 = The subject is able to walk 30 ft. but unable to run; 3 = The subject is able to walk 30 ft. with the assistance of one person; 4 = A walker or cane; 5 = The subject is unable to walk.

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Hughes Function Score	3 units on a scale Interval 2.0 to 4.0	3.2 units on a scale Interval 1.0 to 5.0

SECONDARY outcome

Timeframe: at 2 months

The self-report measure Patient Global Impression of Change (PGIC) reflects a patient's belief about the efficacy of treatment 1. Marked improvement; 2 Moderate Improvement; 3 Minimal Improvement; 4 No Change; 5 Minimal Worsening; 6 Moderate Worsening; 7 Marked worsening

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Impression of Change Score - Participant	3.58 units on a scale Interval 2.0 to 5.0	3.7 units on a scale Interval 2.0 to 4.0

SECONDARY outcome

Timeframe: at 2 months

The clinician measure of Global Impression of Change reflects the clinician's view about the efficacy of treatment as measured by 1. Marked improvement; 2 Moderate Improvement; 3 Minimal Improvement; 4 No Change; 5 Minimal Worsening; 6 Moderate Worsening; 7 Marked worsening

Outcome measures

Outcome measures
Measure	Intravenous Immunoglobulin n=6 Participants Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=5 Participants Saline 2gr/kg over 2 days of saline
Impression of Change Score - Clinician	3.58 units on a scale Interval 2.0 to 5.0	4 units on a scale Interval 3.0 to 5.0

Adverse Events

Intravenous Immunoglobulin

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Intravenous Immunoglobulin n=6 participants at risk Intravenous Immunoglobulin - 2gr/kg over 2 days of Privigen®	Placebo n=6 participants at risk Saline 2gr/kg over 2 days of saline
General disorders Mild headache	16.7% 1/6 • Number of events 1	0.00% 0/6

Additional Information

Dr. David Simpson

Icahn School of Medicine at Mount Sinai

Phone: 212-241-8748

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place