Trial Outcomes & Findings for A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome (NCT NCT05979831)
NCT ID: NCT05979831
Last Updated: 2026-05-14
Results Overview
An AE is any untoward medical occurrence in a patient administered a pharmaceutical product and that does not necessarily have to have a causal relationship with this treatment. AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) v 27.1.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Screening (Day 0) up to Week 45 (end of study)
Results posted on
2026-05-14
Participant Flow
A total of 9 participants were enrolled in the study and randomized to treatment at 1 clinic site in France.
Study eligibility will be assessed at the beginning of the Observational Part and randomization for treatment will be done at the beginning of the Interventional Part, when eligibility will be re-confirmed.
Participant milestones
| Measure |
DS-2325a
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
Placebo
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
|---|---|---|
|
Interventional Part Main (12 Wks)
STARTED
|
5
|
4
|
|
Interventional Part Main (12 Wks)
COMPLETED
|
5
|
3
|
|
Interventional Part Main (12 Wks)
NOT COMPLETED
|
0
|
1
|
|
Interventional Part Extension (24 Wks)
STARTED
|
5
|
3
|
|
Interventional Part Extension (24 Wks)
COMPLETED
|
0
|
1
|
|
Interventional Part Extension (24 Wks)
NOT COMPLETED
|
5
|
2
|
Reasons for withdrawal
| Measure |
DS-2325a
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
Placebo
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
|---|---|---|
|
Interventional Part Main (12 Wks)
Study terminated by Sponsor
|
0
|
1
|
|
Interventional Part Extension (24 Wks)
Withdrew (Extension), discontinued after safety FU
|
1
|
0
|
|
Interventional Part Extension (24 Wks)
Study terminated by Sponsor
|
4
|
2
|
Baseline Characteristics
A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome
Baseline characteristics by cohort
| Measure |
DS-2325a
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
Placebo
n=4 Participants
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase). Participants then received DS-2325a doses for a total of 24 weeks (Extension Phase).
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
28 years
n=1512 Participants
|
29.5 years
n=504 Participants
|
29 years
n=2016 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=1512 Participants
|
4 Participants
n=504 Participants
|
5 Participants
n=2016 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=1512 Participants
|
0 Participants
n=504 Participants
|
4 Participants
n=2016 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=1512 Participants
|
1 Participants
n=504 Participants
|
1 Participants
n=2016 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 Participants
n=1512 Participants
|
1 Participants
n=504 Participants
|
2 Participants
n=2016 Participants
|
|
Race/Ethnicity, Customized
White
|
3 Participants
n=1512 Participants
|
1 Participants
n=504 Participants
|
4 Participants
n=2016 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=1512 Participants
|
1 Participants
n=504 Participants
|
2 Participants
n=2016 Participants
|
PRIMARY outcome
Timeframe: Screening (Day 0) up to Week 45 (end of study)Population: Adverse events were assessed in the Safety Analysis Set.
An AE is any untoward medical occurrence in a patient administered a pharmaceutical product and that does not necessarily have to have a causal relationship with this treatment. AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) v 27.1.
Outcome measures
| Measure |
Placebo (Observational Part)
n=4 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
n=4 Participants
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
n=5 Participants
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
n=3 Participants
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Number of Participants With Treatment-emergent Adverse Events
|
2 Participants
|
5 Participants
|
4 Participants
|
4 Participants
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Main Phase: Week 1, 1 hour postdose, 2 hour postdose and Predose at Weeks 3, 5, 7, 9, and 11Population: Participants received different frequencies of doses (changing from weekly to biweekly from Week 5 after protocol amendment) and different numbers of doses due to early study termination. The Ctrough summaries excluded one participant who received weekly dosing instead of biweekly dosing through Week 11.
The Ctrough summaries include trough concentrations at the designed pre-dose time points that received a prior planned dose.
Outcome measures
| Measure |
Placebo (Observational Part)
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=4 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 1 (1 hour [hr] postdose)
|
—
|
—
|
—
|
—
|
—
|
311000 ng/mL
Standard Deviation 49132
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 1 (2 hr postdose)
|
—
|
—
|
—
|
—
|
—
|
327500 ng/mL
Standard Deviation 38492
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 3 (predose)
|
—
|
—
|
—
|
—
|
—
|
89375 ng/mL
Standard Deviation 11312
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 5 (predose)
|
—
|
—
|
—
|
—
|
—
|
66850 ng/mL
Standard Deviation 14591
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 7 (predose)
|
—
|
—
|
—
|
—
|
—
|
54575 ng/mL
Standard Deviation 19102
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 9 (predose)
|
—
|
—
|
—
|
—
|
—
|
52450 ng/mL
Standard Deviation 17702
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Main Phase
Week 11 (predose)
|
—
|
—
|
—
|
—
|
—
|
45000 ng/mL
Standard Deviation 13279
|
SECONDARY outcome
Timeframe: Extension Phase: Predose at Weeks 13, 15, 17, 19, 21, and 23Population: Participants received different frequencies of doses (changing from weekly to biweekly from Week 5 after protocol amendment) and different numbers of doses due to early study termination. The Ctrough summaries excluded one participant who received weekly dosing instead of biweekly dosing through Week 11.
The Ctrough summaries include trough concentrations at the designed pre-dose time points that received a prior planned dose.
Outcome measures
| Measure |
Placebo (Observational Part)
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=3 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 23 (predose)
|
—
|
—
|
—
|
—
|
—
|
26100 ng/mL
Standard Deviation NA
Standard deviation cannot be calculated for 1 person.
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 21 (predose)
|
—
|
—
|
—
|
—
|
—
|
28000 ng/mL
Standard Deviation NA
Standard deviation cannot be calculated for 1 person.
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 13 (predose)
|
—
|
—
|
—
|
—
|
—
|
48466.7 ng/mL
Standard Deviation 18006
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 15 (predose)
|
—
|
—
|
—
|
—
|
—
|
82900 ng/mL
Standard Deviation 24183
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 17 (predose)
|
—
|
—
|
—
|
—
|
—
|
66400 ng/mL
Standard Deviation 36345
|
|
Pharmacokinetic Parameter Trough Concentration (Ctrough) - Extension Phase
Week 19 (predose)
|
—
|
—
|
—
|
—
|
—
|
49600 ng/mL
Standard Deviation 23335
|
SECONDARY outcome
Timeframe: Baseline up to Week 13Population: The IASI score was assessed in participants with available data in the Modified Intent-to-Treat Analysis Set.
The IASI assesses the intensity of the participant's erythema (IASI-Erythema) and scaling (IASI-Scaling) using a 4-point Likert scale ranging from 0 (none) to 4 (very severe). The total IASI (range 0 to 48) is determined by adding IASI-Erythema and IASI-Scaling scores. Higher scores indicate worse clinical outcome. A change from baseline is being reported and the greater the change the worse clinical outcome.
Outcome measures
| Measure |
Placebo (Observational Part)
n=3 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Median Change From Baseline in Ichthyosis Area Severity Index (IASI) Scores
|
7.450 score on a scale
Interval 0.93 to 8.48
|
—
|
—
|
—
|
—
|
0.900 score on a scale
Interval -5.38 to 9.8
|
SECONDARY outcome
Timeframe: Baseline up to Week 13Population: The IGA scores were accessed in participants with available data in the Modified Intent-to-Treat Analysis Set.
The IGA assesses a participant's erythema, scaling, inflammatory papules or plaques, oozing, and lichenification using a 5-point scale (0, clear; 1, almost clear; 2, mild; 3, moderate; 4, severe). Higher scores indicate worse clinical outcome. The change from baseline is being reported where negative values indicate an improvement in clinical outcome.
Outcome measures
| Measure |
Placebo (Observational Part)
n=3 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Median Change From Baseline in Investigator Global Assessment (IGA) Scores
Erythema
|
0.75 score on a scale
Interval 0.0 to 1.0
|
—
|
—
|
—
|
—
|
0 score on a scale
Interval -2.3 to 0.3
|
|
Median Change From Baseline in Investigator Global Assessment (IGA) Scores
Scaling
|
0.75 score on a scale
Interval 0.3 to 1.5
|
—
|
—
|
—
|
—
|
0 score on a scale
Interval -0.8 to 1.0
|
|
Median Change From Baseline in Investigator Global Assessment (IGA) Scores
Inflammatory papules or plaques
|
2.00 score on a scale
Interval 0.0 to 3.5
|
—
|
—
|
—
|
—
|
-0.50 score on a scale
Interval -1.8 to 1.0
|
|
Median Change From Baseline in Investigator Global Assessment (IGA) Scores
Oozing
|
1.00 score on a scale
Interval 0.5 to 2.0
|
—
|
—
|
—
|
—
|
0.50 score on a scale
Interval -1.8 to 2.8
|
|
Median Change From Baseline in Investigator Global Assessment (IGA) Scores
Lichenification
|
0.75 score on a scale
Interval -0.8 to 1.0
|
—
|
—
|
—
|
—
|
0 score on a scale
Interval -2.0 to 0.8
|
SECONDARY outcome
Timeframe: Baseline up to Week 13Population: The Itch NRS scores were accessed in participants with available data in the Modified Intent-to-Treat Analysis Set.
The Itch NRS is a self-rated single item scale designed for assessing worst pruritus in the past 7 days. The scale utilizes an 11-point NRS, scored from 0 (no itch) to 10 (worst imaginable itch). Higher scores indicate worse clinical outcome. The change from baseline is being reported with negative values indicating an improvement in clinical outcome.
Outcome measures
| Measure |
Placebo (Observational Part)
n=3 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Median Change From Baseline in Itch Numerical Rating Scale (NRS) Scores
|
-1.00 score on a scale
Interval -1.3 to -0.8
|
—
|
—
|
—
|
—
|
1.00 score on a scale
Interval -1.0 to 2.8
|
SECONDARY outcome
Timeframe: Baseline up to Week 13Population: Skindex-29 responses were assessed in participants with available data in the Modified Intent-to-Treat Analysis Set.
The Skindex-29 is a 29-item questionnaire that assesses the burden of the participant's skin condition on 3 scales - symptoms, social functioning and emotional well-being. The score for each scale ranges from 0 to 100. Higher scores reflect a worse quality of life.
Outcome measures
| Measure |
Placebo (Observational Part)
n=3 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Median Change From Baseline in Skindex-29 Responses
Symptoms Score
|
-13.393 score on a scale
Interval -18.75 to 0.0
|
—
|
—
|
—
|
—
|
4.464 score on a scale
Interval -22.32 to 29.46
|
|
Median Change From Baseline in Skindex-29 Responses
Emotions Score
|
5.00 score on a scale
Interval 1.25 to 19.38
|
—
|
—
|
—
|
—
|
1.875 score on a scale
Interval -8.13 to 39.38
|
|
Median Change From Baseline in Skindex-29 Responses
Functioning Score
|
-5.208 score on a scale
Interval -12.5 to 18.23
|
—
|
—
|
—
|
—
|
0 score on a scale
Interval -6.77 to 29.17
|
SECONDARY outcome
Timeframe: Baseline up to Week 13Population: Dermatology Life Quality Index Questionnaire was assessed in participants with available data in the Modified Intent-to-Treat Analysis Set.
The Dermatology Life Quality Index (DLQI) is a 10-item validated questionnaire used to assess participants' perception of the impact of their skin disease on different aspects of their quality of life over the prior week. The DLQI score is the sum of the 10 item scores and ranges from 0 to 30. A high score is indicative of a poor quality of life.
Outcome measures
| Measure |
Placebo (Observational Part)
n=3 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Median Change From Baseline in Dermatology Life Quality Index (DLQI) Questionnaire Score
|
-1.25 score on a scale
Interval -1.3 to 8.5
|
—
|
—
|
—
|
—
|
1.75 score on a scale
Interval -5.0 to 9.8
|
SECONDARY outcome
Timeframe: Baseline up to Week 45 (end of study)Population: ADA's were assessed in participants with available data in the Pharmacokinetic Analysis Set.
The anti-drug antibodies (ADAs) against DS-2325a was assessed as the immunogenicity endpoint.
Outcome measures
| Measure |
Placebo (Observational Part)
n=4 Participants
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
Participants were randomized to a single initial ("loading") dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS2325a (Interventional Part Main Phase)
n=5 Participants
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks (Main Phase).
|
|---|---|---|---|---|---|---|
|
Number of Participants With Anti-Drug Antibodies Against DS-2325a (Interventional Part Main and Extension Phases)
|
0 Participants
|
—
|
—
|
—
|
—
|
0 Participants
|
Adverse Events
DS-2325a (Observational Part)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo (Observational Part)
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
DS-2325a (Interventional Part Main Phase)
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo (Interventional Part Main Phase)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
DS-2325a (Interventional Part Extension Phase)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Placebo (Interventional Part Extension Phase)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
DS-2325a (Observational Part)
n=5 participants at risk
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Observational Part)
n=4 participants at risk
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
n=5 participants at risk
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
n=4 participants at risk
Participants were randomized to a single ("initial") loading dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
n=5 participants at risk
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
n=3 participants at risk
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
|---|---|---|---|---|---|---|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Nosocomial infection
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
Other adverse events
| Measure |
DS-2325a (Observational Part)
n=5 participants at risk
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Observational Part)
n=4 participants at risk
Participants were assessed for 12 weeks preceding study treatment administration to establish a baseline for safety and efficacy endpoints. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
DS-2325a (Interventional Part Main Phase)
n=5 participants at risk
Participants were randomized to a single initial ("loading") dose of DS-2325a (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
Placebo (Interventional Part Main Phase)
n=4 participants at risk
Participants were randomized to a single ("initial") loading dose of Placebo (Week 1) followed by ("maintenance") doses for a total of 12 weeks.
|
DS-2325a (Interventional Part Extension Phase)
n=5 participants at risk
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with DS-2325a at the start of the Interventional Part Main Phase.
|
Placebo (Interventional Part Extension Phase)
n=3 participants at risk
Participants received DS-2325a doses for a total of 24 weeks. This group was randomized to treatment with Placebo at the start of the Interventional Part Main Phase.
|
|---|---|---|---|---|---|---|
|
General disorders
Influenza-like illness
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
General disorders
Injection site erythema
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
General disorders
Injection site pruritus
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Eye disorders
Blepharitis
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Eye disorders
Conjunctivitis allergic
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
COVID-19
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Rhinitis
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Influenza
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Hordeolum
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Viral rhinitis
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
33.3%
1/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Cardiac disorders
Palpitations
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
33.3%
1/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Gastrointestinal disorders
Hemorrhoids
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Immune system disorders
Food allergy
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Gastrointestinal disorders
Constipation
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
33.3%
1/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Nervous system disorders
Sciatica
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
General disorders
Injection site pain
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
60.0%
3/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
50.0%
2/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
40.0%
2/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
33.3%
1/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
General disorders
Asthenia
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Congenital, familial and genetic disorders
Netherton's syndrome
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
75.0%
3/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
33.3%
1/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Bronchitis
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
20.0%
1/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Nosocomial infection
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Infections and infestations
Proteus infection
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
|
Cardiac disorders
Extrasystoles
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
25.0%
1/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/4 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/5 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
0.00%
0/3 • Adverse events were collected from screening (Day 0) up to Week 45 (end of study).
|
Additional Information
Daiichi Sankyo Contact for Clinical Trial Information
Daiichi Sankyo
Phone: 908-992-6400
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place