Trial Outcomes & Findings for A Phase 2 Study to Evaluate the Safety and Efficacy of ABP-450 in the Treatment of Cervical Dystonia (NCT NCT04849988)

NCT ID: NCT04849988

Last Updated: 2024-02-14

Results Overview

The primary safety endpoint will be the number of participants with treatment-related serious adverse events since the start of treatment by Treatment Group when dosed with placebo, ABP-450 (low dose), ABP-450 (medium d dose), or ABP-450 (high dose).

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 61 participants
• Primary outcome timeframe: Baseline up to 20 Weeks
• Results posted on: 2024-02-14

Participant Flow

Participants were recruited at 25 sites in the U.S. between March 29, 2021 and July 11, 2021.

Of the 70 participants screened, 61 were randomized. There were 59 participants randomized and dosed, and 57 of them were included in the modified full analysis set (mFAS) for the efficacy analysis.

Participant milestones

Measure	Placebo Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Overall Study STARTED	15	15	16	15
Overall Study Randomized But Not Dosed	1	1	0	0
Overall Study Treated	14	14	16	15
Overall Study Safety Analysis Set	14	14	16	15
Overall Study Full Analysis Set	14	14	16	15
Overall Study Modified Full Analysis Set	14	14	14	15
Overall Study COMPLETED	11	9	13	10
Overall Study NOT COMPLETED	4	6	3	5

Reasons for withdrawal

Measure	Placebo Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Overall Study Lack of Efficacy	3	4	1	4
Overall Study Other reasons	1	2	2	1

Baseline Characteristics

A Phase 2 Study to Evaluate the Safety and Efficacy of ABP-450 in the Treatment of Cervical Dystonia

Baseline characteristics by cohort

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.	Total n=57 Participants Total of all reporting groups
Age, Continuous	59.3 years STANDARD_DEVIATION 11.06 • n=5 Participants	55.8 years STANDARD_DEVIATION 12.97 • n=7 Participants	54.6 years STANDARD_DEVIATION 13.61 • n=5 Participants	55.3 years STANDARD_DEVIATION 11.39 • n=4 Participants	56.2 years STANDARD_DEVIATION 12.09 • n=21 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants	12 Participants n=4 Participants	39 Participants n=21 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	7 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants	18 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants	14 Participants n=7 Participants	11 Participants n=5 Participants	14 Participants n=4 Participants	52 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	2 Participants n=21 Participants
Previous treatment with BOTOX®	10 Participants n=5 Participants	9 Participants n=7 Participants	11 Participants n=5 Participants	10 Participants n=4 Participants	40 Participants n=21 Participants
Duration of Cervical Dystonia	14.83 years STANDARD_DEVIATION 11.134 • n=5 Participants	14.58 years STANDARD_DEVIATION 10.799 • n=7 Participants	7.36 years STANDARD_DEVIATION 6.464 • n=5 Participants	11.53 years STANDARD_DEVIATION 7.175 • n=4 Participants	12.26 years STANDARD_DEVIATION 9.421 • n=21 Participants
Baseline TWSTRS-Total Score	39.68 units on a scale STANDARD_DEVIATION 7.525 • n=5 Participants	44.30 units on a scale STANDARD_DEVIATION 6.093 • n=7 Participants	42.20 units on a scale STANDARD_DEVIATION 11.585 • n=5 Participants	43.62 units on a scale STANDARD_DEVIATION 9.847 • n=4 Participants	42.47 units on a scale STANDARD_DEVIATION 8.962 • n=21 Participants

PRIMARY outcome

Timeframe: Baseline up to 20 Weeks

Population: All treated patients are accounted for in the Treatment-related Serious Adverse Events data table including the two patients with changes in background medications.

The primary safety endpoint will be the number of participants with treatment-related serious adverse events since the start of treatment by Treatment Group when dosed with placebo, ABP-450 (low dose), ABP-450 (medium d dose), or ABP-450 (high dose).

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=16 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Number of Participants With Treatment-Related Serious Adverse Events	0 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) scale is used to assess the severity of cervical Dystonia. The TWSTRS-total score has a minimum score of 0 and a maximum score of 85, where higher scores represent worse outcomes. It comprises 3 subscales, which are summated to get the total score: the Torticollis Severity Scale (minimum score of 0, maximum score of 35), the Disability Scale (minimum score of 0, maximum score of 30), and the Pain Scale (minimum score of 0, maximum score of 20).

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total Score From Baseline to Week 4	3.57 score on a scale Standard Error 2.67	14.01 score on a scale Standard Error 2.63	11.28 score on a scale Standard Error 2.70	9.92 score on a scale Standard Error 2.54

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The Disability subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of disability in cervical dystonia. The disability subscale has a minimum score of 0 and maximum score of 30, where higher scores represent worse outcomes.

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change in the Subscale Score of Disability of the Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	1.02 score on a scale Standard Error 1.11	4.53 score on a scale Standard Error 1.09	4.24 score on a scale Standard Error 1.14	2.62 score on a scale Standard Error 1.06

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The Pain subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of pain in cervical dystonia. The pain subscale has a minimum score of 0 and maximum score of 20, where higher scores represent worse outcomes.

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change in the Subscale Score of Pain of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	1.27 score on a scale Standard Error 1.10	3.56 score on a scale Standard Error 1.05	3.58 score on a scale Standard Error 1.08	3.69 score on a scale Standard Error 1.02

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The Severity subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of torticollis in cervical dystonia. The severity subscale has a minimum score of 0 and maximum score of 35, where higher scores represent worse outcomes.

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change in the Subscale Score of Severity of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	1.91 score on a scale Standard Error 0.91	5.89 score on a scale Standard Error 0.91	2.94 score on a scale Standard Error 0.97	3.45 score on a scale Standard Error 0.87

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The PGI-C enables the patient to rate changes in their perception of their general health status over the duration of the assessment via a 7-point scale ranging from "much improved" to "much worse". The 7-point scale range is much improved (+3) to much worse (-3) with no change at "0".

The mean change in the subject's assessment of the change in clinical status since the start of treatment measured by the Patients' Global Impression of Change (PGI-C) Scale was assessed by Treatment Group at Week 4.

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change in Patient Global Impression of Change (PGI-C) From Baseline to Week 4	4.0 units on a scale Standard Error 1.11	2.6 units on a scale Standard Error 0.31	2.2 units on a scale Standard Error 0.32	2.7 units on a scale Standard Error 0.30

SECONDARY outcome

Timeframe: Baseline to Week 4

Population: Two patients were excluded from the analysis due to a change in their background medication.

The CGI-C enables the patient to rate changes in their perception of their general health status over the duration of the assessment via a 7-point scale ranging from "much improved" to "much worse". The 7-point scale range is much improved (+3) to much worse (-3) with no change at "0".

The mean change from Baseline in the Clinical Global Impression of Change (CGI-C) Score was assessed by Treatment Group at Week 4.

Outcome measures

Measure	Placebo n=14 Participants Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 Participants ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=14 Participants ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 Participants ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Mean Change in Clinical Global Impression of Change (CGI-C) From Baseline to Week 4	3.9 units on a scale Standard Error 0.33	2.3 units on a scale Standard Error 0.32	2.0 units on a scale Standard Error 0.33	2.7 units on a scale Standard Error 0.31

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

ABP-450 - 150U

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

ABP-450 - 250U

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

ABP-450 - 350U

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Placebo n=14 participants at risk Placebo (0.9% unpreserved saline) - Intramuscular injections into affected neck muscles.	ABP-450 - 150U n=14 participants at risk ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 250U n=16 participants at risk ABP-450 Medium Dose - Intramuscular injections into affected neck muscles.	ABP-450 - 350U n=15 participants at risk ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Gastrointestinal disorders Dysphagia	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	12.5% 2/16 • Up to Week 20	20.0% 3/15 • Up to Week 20
Gastrointestinal disorders Muscular Weakness	0.00% 0/14 • Up to Week 20	14.3% 2/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	6.7% 1/15 • Up to Week 20
Nervous system disorders Headache	7.1% 1/14 • Up to Week 20	14.3% 2/14 • Up to Week 20	12.5% 2/16 • Up to Week 20	6.7% 1/15 • Up to Week 20
Musculoskeletal and connective tissue disorders Joint Swelling	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Musculoskeletal and connective tissue disorders Neck pain	21.4% 3/14 • Up to Week 20	7.1% 1/14 • Up to Week 20	12.5% 2/16 • Up to Week 20	6.7% 1/15 • Up to Week 20
Musculoskeletal and connective tissue disorders Torticollis	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	13.3% 2/15 • Up to Week 20
Gastrointestinal disorders Nausea	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	12.5% 2/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Nervous system disorders Dizziness	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Nervous system disorders Head discomfort	0.00% 0/14 • Up to Week 20	7.1% 1/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Nervous system disorders Presyncope	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	6.7% 1/15 • Up to Week 20
General disorders Feeling abnormal	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
General disorders Injection site pain	7.1% 1/14 • Up to Week 20	7.1% 1/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
General disorders Injection site pruritus	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
General disorders Malaise	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Eye disorders Diplopia	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Skin and subcutaneous tissue disorders Rash macular	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	6.7% 1/15 • Up to Week 20
Infections and infestations COVID-19	0.00% 0/14 • Up to Week 20	7.1% 1/14 • Up to Week 20	6.2% 1/16 • Up to Week 20	13.3% 2/15 • Up to Week 20
Infections and infestations Upper respiratory tract infection	0.00% 0/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	12.5% 2/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Musculoskeletal and connective tissue disorders Dupuytren's contracture	7.1% 1/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Musculoskeletal and connective tissue disorders Limb discomfort	7.1% 1/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
Nervous system disorders Tension headache	7.1% 1/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20
General disorders Influenza like illness	7.1% 1/14 • Up to Week 20	0.00% 0/14 • Up to Week 20	0.00% 0/16 • Up to Week 20	0.00% 0/15 • Up to Week 20

Additional Information

Dana Testa, PharmD

AEON Biopharma, Inc.

Phone: 949-354-6499

Email: dt@aeonbiopharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee Data are the property of the sponsor and cannot be published without prior authorization from the sponsor.
• Publication restrictions are in place

Restriction type: OTHER