Trial Outcomes & Findings for Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis (NCT NCT01000961)
NCT ID: NCT01000961
Last Updated: 2024-12-19
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
43 participants
Primary outcome timeframe
4 weeks after the last subject has completed the study
Results posted on
2024-12-19
Participant Flow
Participants randomized to each per sequence Arm are expected to remain in the same Arm throughout all intervention periods.
Participant milestones
| Measure |
Cystagon First, Then Cystagon, Then RP103
Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in first intervention (after Run-in period) and RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in second intervention period.
|
Cystagon First, Then RP103, Then Cystagon
RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in first intervention (after Run-in period) and Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in second intervention period.
|
|---|---|---|
|
Run-in Period of 2-3 Weeks on Cystagon
STARTED
|
21
|
22
|
|
Run-in Period of 2-3 Weeks on Cystagon
COMPLETED
|
21
|
22
|
|
Run-in Period of 2-3 Weeks on Cystagon
NOT COMPLETED
|
0
|
0
|
|
First Intervention (3 Weeks)
STARTED
|
21
|
22
|
|
First Intervention (3 Weeks)
COMPLETED
|
21
|
20
|
|
First Intervention (3 Weeks)
NOT COMPLETED
|
0
|
2
|
|
Second Intervention (3 Weeks)
STARTED
|
21
|
20
|
|
Second Intervention (3 Weeks)
COMPLETED
|
21
|
20
|
|
Second Intervention (3 Weeks)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cystagon First, Then Cystagon, Then RP103
Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in first intervention (after Run-in period) and RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in second intervention period.
|
Cystagon First, Then RP103, Then Cystagon
RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in first intervention (after Run-in period) and Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in second intervention period.
|
|---|---|---|
|
First Intervention (3 Weeks)
Withdrawal by Subject
|
0
|
1
|
|
First Intervention (3 Weeks)
Infection after pre-planned surgery
|
0
|
1
|
Baseline Characteristics
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Baseline characteristics by cohort
| Measure |
RP103 and Cystagon® Crossover
n=39 Participants
Per Protocol Population
|
|---|---|
|
Age, Categorical
<=18 years
|
36 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
|
11.9 years
STANDARD_DEVIATION 4.33 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Region of Enrollment
France
|
13 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
22 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 weeks after the last subject has completed the studyOutcome measures
| Measure |
RP103
n=39 Participants
Per Protocol Population
|
Cystagon®
n=39 Participants
Per Protocol Population
|
|---|---|---|
|
The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®
|
0.5152 nmol ½ Cystine / mg protein
Standard Error 0.05555
|
0.4367 nmol ½ Cystine / mg protein
Standard Error 0.05555
|
SECONDARY outcome
Timeframe: 4 weeks after the last subject has completed the studyOutcome measures
| Measure |
RP103
n=39 Participants
Per Protocol Population
|
Cystagon®
n=37 Participants
Per Protocol Population
|
|---|---|---|
|
Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®.
|
2.73 Cmax (mg/L)
Standard Deviation 1.36
|
3.70 Cmax (mg/L)
Standard Deviation 1.72
|
SECONDARY outcome
Timeframe: 4 weeks after the last subject has completed the studyOutcome measures
| Measure |
RP103
n=39 Participants
Per Protocol Population
|
Cystagon®
n=37 Participants
Per Protocol Population
|
|---|---|---|
|
Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®.
|
72 Tmax (minute)
Standard Deviation 31
|
187 Tmax (minute)
Standard Deviation 89
|
SECONDARY outcome
Timeframe: 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103.Outcome measures
| Measure |
RP103
n=39 Participants
Per Protocol Population
|
Cystagon®
n=37 Participants
Per Protocol Population
|
|---|---|---|
|
Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®.
|
357 AUC(0-t) (min*mg/L)
Standard Deviation 150
|
739 AUC(0-t) (min*mg/L)
Standard Deviation 334
|
Adverse Events
RP103
Serious events: 6 serious events
Other events: 16 other events
Deaths: 0 deaths
Cystagon®
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
RP103
n=43 participants at risk
Safety Population during treatment periods
|
Cystagon®
n=41 participants at risk
Safety population during treatment periods
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Metabolism and nutrition disorders
Hypovolaemia
|
0.00%
0/43 • Treatment periods
Safety population AE reporting
|
2.4%
1/41 • Number of events 1 • Treatment periods
Safety population AE reporting
|
|
Infections and infestations
Gastroenteritis
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Gastrointestinal disorders
Abdominal Discomfort
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Gastrointestinal disorders
Vomiting
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Injury, poisoning and procedural complications
Femur Fracture
|
2.3%
1/43 • Number of events 1 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
Other adverse events
| Measure |
RP103
n=43 participants at risk
Safety Population during treatment periods
|
Cystagon®
n=41 participants at risk
Safety population during treatment periods
|
|---|---|---|
|
Gastrointestinal disorders
Vomiting
|
11.6%
5/43 • Number of events 8 • Treatment periods
Safety population AE reporting
|
7.3%
3/41 • Number of events 5 • Treatment periods
Safety population AE reporting
|
|
Gastrointestinal disorders
Nausea
|
11.6%
5/43 • Number of events 7 • Treatment periods
Safety population AE reporting
|
4.9%
2/41 • Number of events 3 • Treatment periods
Safety population AE reporting
|
|
Gastrointestinal disorders
Abdominal Pain
|
7.0%
3/43 • Number of events 4 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
7.0%
3/43 • Number of events 3 • Treatment periods
Safety population AE reporting
|
0.00%
0/41 • Treatment periods
Safety population AE reporting
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Investigators may publish or disclose study data, with the restriction that sponsor may embargo such communications for a period up to 60 days from the time submitted to sponsor. As a multi-center study, first publication of results will take place in conjunction with all investigators. If such a multi-center publication is not forthcoming within 18 months of study completion, individual investigators may publish as restricted according to limitations expressed above (60 day embargo).
- Publication restrictions are in place
Restriction type: OTHER