Trial Outcomes & Findings for Long-Term Study of IGIV, 10% in Alzheimer´s Disease (NCT NCT01736579)

NCT ID: NCT01736579

Last Updated: 2021-05-19

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

6 participants

Primary outcome timeframe

6 months

Results posted on

2021-05-19

Participant Flow

Enrollment was conducted at one clinical site in the US.

Six participants were enrolled (i.e. signed informed consent form), of which 2 failed screening and 4 were treated with study product.

Participant milestones

Participant milestones
Measure
IGIV, 10% at 0.2 g/kg Body Weight
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
IGIV, 10% at 0.4 g/kg Body Weight
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
Overall Study
STARTED
2
2
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
2
2

Reasons for withdrawal

Reasons for withdrawal
Measure
IGIV, 10% at 0.2 g/kg Body Weight
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
IGIV, 10% at 0.4 g/kg Body Weight
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
Overall Study
Termination of Study by Sponsor
2
2

Baseline Characteristics

Long-Term Study of IGIV, 10% in Alzheimer´s Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
IGIV, 10% at 0.2 g/kg Body Weight
n=2 Participants
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
IGIV, 10% at 0.4 g/kg Body Weight
n=2 Participants
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
Total
n=4 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Categorical
>=65 years
2 Participants
n=99 Participants
2 Participants
n=107 Participants
4 Participants
n=206 Participants
Sex: Female, Male
Female
1 Participants
n=99 Participants
1 Participants
n=107 Participants
2 Participants
n=206 Participants
Sex: Female, Male
Male
1 Participants
n=99 Participants
1 Participants
n=107 Participants
2 Participants
n=206 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure
IGIV, 10% at 0.4 g/kg Body Weight
n=2 Participants
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
IGIV, 10% at 0.2 g/kg Body Weight
n=2 Participants
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
Number and Severity of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Non-serious AEs-Mild
0 adverse events
2 adverse events
Number and Severity of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Non-serious AEs-Moderate
1 adverse events
1 adverse events
Number and Severity of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAEs
0 adverse events
0 adverse events

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure
IGIV, 10% at 0.4 g/kg Body Weight
n=7 Infusions
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
IGIV, 10% at 0.2 g/kg Body Weight
n=10 Infusions
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
Number of Infusions Temporally Associated With Adverse Events (AEs) and Serious Adverse Events (SAEs)
0 infusions
1 infusions

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure
IGIV, 10% at 0.4 g/kg Body Weight
n=7 Infusions
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
IGIV, 10% at 0.2 g/kg Body Weight
n=10 Infusions
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
Number of Infusions Causally Associated With Adverse Events (AEs) and Serious Adverse Events (SAEs)
1 infusions
1 infusions

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure
IGIV, 10% at 0.4 g/kg Body Weight
n=7 Infusions
IGIV, 10% at 0.4 g/kg body weight every 2 weeks
IGIV, 10% at 0.2 g/kg Body Weight
n=10 Infusions
IGIV, 10% at 0.2 g/kg body weight every 2 weeks
Number of Infusions Discontinued, Slowed or Interrupted Due to an Adverse Event (AE) or Serious Adverse Event (SAE)
0 infusions
0 infusions

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The ADAS-Cog is a validated psychometric instrument that evaluates memory (word recall, word recognition), attention, reasoning (following commands), language (naming, comprehension), orientation, ideational praxis (placing letter in envelope) and constructional praxis (copying geometric designs). This test was administered by experienced raters certified by Alzheimer's Disease Cooperative Study (ADCS) at the site. Scores on the ADAS-Cog range from 0-70 with higher scores indicating greater impairment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The SIB is a 40-item psychometric assessment that is composed of simple one-step commands combined with gestures. The scoring range is from 0 to 100 with a lower score indicating greater cognitive impairment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The ADCS-ADL scale is a validated tool to assess instrumental and basic activities of daily living based on a 23 item structured interview of the caregiver or qualified study partner. Scores on the ADCS-ADL range from 0-78 with lower scores indicating greater impairment; hence decreases from baseline reflect potential functional deterioration.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The MMSE is a test for cognitive dysfunction. The test provides a 30-point composite rating for spatial and temporal orientation, verbal recall, simple attention, working memory, naming, repetition, comprehension, writing and constructional abilities. The total score can range from 0 to 30 with a higher score indicating better function.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The NPI is a validated instrument used to assess behavioral psychopathology in Alzheimer's Disease; it evaluates the frequency and severity of 12 neuropsychiatric features including delusions, hallucinations, dysphoria, anxiety, agitation/aggression, euphoria, disinhibition, irritability/lability, apathy, aberrant motor activity, sleep and night-time behavior change, and appetite and eating change. The NPI total score ranged 0-144, with higher scores indicating greater impairment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The QOL-AD is a validated, 13-item instrument developed specifically for individuals with dementia. The assessment rates the participant's quality of life for physical, emotional, interpersonal, and environmental domains. The QOL-AD total score ranged 13-52. Lower scores on the QOL-AD are associated with a lower quality of life.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. The EQ-5D also includes a standard vertical 20 cm visual analogue scale (VAS) ranging from best imaginable health state \[100\] to worst imaginable health state \[0\]. Caregivers are asked to describe how they believe a participant would rate his/her health state that day.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The HRUQ determines if there is a difference in healthcare utilization and health-related expenditures, most notably nursing home (ie, skilled nursing facility) admissions, when subjects are treated with study product. This assessment is performed with the primary caregiver. This assessment is descriptive and does not contain specific scores.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

The Caregiver burden questionnaires is a self-administered questionnaire that has been developed to measure the emotional, physical, and social impact of caregiving on Alzheimer's Disease caregivers. A Total score is calculated from this measure by summing the responses across the items. The Total score may range from 9-45, with higher scores indicating greater burden.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

Time to skilled nursing facility placement is defined as permanent admission to a skilled nursing facility. Time will be defined as the number of months between enrollment into this clinical study and placement in a skilled nursing facility placement.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Population: This study was terminated early. Summary tables were not provided for this outcome measure due to the small number of participants (up to 2 participants per arm) because of concerns about patient confidentiality.

Volumetric MRI measurements were obtained to assess rate of whole brain atrophy and ventricular enlargement. Additional volumetric measurements may be analyzed when specific hypotheses and methods are defined. Additional volumetric MRI analysis may include (but may not be limited to) one or more of the following: rate of hippocampal atrophy, entorhinal cortical thickness, and/or regional cortical thinning.

Outcome measures

Outcome data not reported

Adverse Events

IGIV, 10% at 0.2 g/kg Body Weight

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

IGIV, 10% at 0.4 g/kg Body Weight

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
IGIV, 10% at 0.2 g/kg Body Weight
n=2 participants at risk
IGIV, 10% at 0.2 g/kg body weight every 2 weeks.
IGIV, 10% at 0.4 g/kg Body Weight
n=2 participants at risk
IGIV, 10% at 0.4 g/kg body weight every 2 weeks.
General disorders
Infusion Site Pain
50.0%
1/2 • Throughout the study period (6 months)
0.00%
0/2 • Throughout the study period (6 months)
Infections and infestations
Tooth Infection
50.0%
1/2 • Throughout the study period (6 months)
0.00%
0/2 • Throughout the study period (6 months)
Investigations
Blood Pressure Increased
50.0%
1/2 • Throughout the study period (6 months)
0.00%
0/2 • Throughout the study period (6 months)
Skin and subcutaneous tissue disorders
Urticaria
0.00%
0/2 • Throughout the study period (6 months)
50.0%
1/2 • Throughout the study period (6 months)

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee Baxalta's agreements with PIs may vary per requirements of individual PI, but contain common elements. For this study, PIs are restricted from independently publishing results until the earlier of the primary multicenter publication or 12 months after study completion. Baxalta requires a review of results communications (eg, for confidential information) ≥30 days prior to submission or communication. Baxalta may request an additional delay of ≤60 days eg, for intellectual property protection.
  • Publication restrictions are in place

Restriction type: OTHER