Trial · NCT01000961
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
NCT01000961, Phase 3, Completed, Amgen; results first posted 2014-11-19
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis. Trials, record NCT01000961. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01000961
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
- NCT number
- NCT01000961
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 43 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Cystinosis
- Interventions
- Cystagon® (Cysteamine Bitartrate), Cysteamine Bitartrate Delayed-release Capsules (RP103)
- First posted
- 2009-10-23
- Last updated
- 2024-12-19
- Results first posted
- 2014-11-19
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® | 4 weeks after the last subject has completed the study | |
| Secondary | Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®. | 4 weeks after the last subject has completed the study | |
| Secondary | Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®. | 4 weeks after the last subject has completed the study | |
| Secondary | Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®. | 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® | 4 weeks after the last subject has completed the study | nmol ½ Cystine / mg protein | LEAST_SQUARES_MEAN | |
| SECONDARY | Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®. | 4 weeks after the last subject has completed the study | Cmax (mg/L) | LEAST_SQUARES_MEAN | |
| SECONDARY | Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®. | 4 weeks after the last subject has completed the study | Tmax (minute) | LEAST_SQUARES_MEAN | |
| SECONDARY | Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®. | 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103. | AUC(0-t) (min*mg/L) | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® | RP103 | 0.5152 | Standard Error 0.05555 | nmol ½ Cystine / mg protein | |
| The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® | Cystagon® | 0.4367 | Standard Error 0.05555 | nmol ½ Cystine / mg protein | |
| Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®. | Cystagon® | 2.73 | Standard Deviation 1.36 | Cmax (mg/L) | |
| Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®. | RP103 | 3.70 | Standard Deviation 1.72 | Cmax (mg/L) | |
| Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®. | Cystagon® | 72 | Standard Deviation 31 | Tmax (minute) | |
| Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®. | RP103 | 187 | Standard Deviation 89 | Tmax (minute) | |
| Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®. | Cystagon® | 357 | Standard Deviation 150 | AUC(0-t) (min*mg/L) | |
| Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®. | RP103 | 739 | Standard Deviation 334 | AUC(0-t) (min*mg/L) |
Source ClinicalTrials.gov
Participant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Run-in Period of 2-3 Weeks on Cystagon | STARTED | Cystagon First, Then Cystagon, Then RP103 | 21 | |
| Run-in Period of 2-3 Weeks on Cystagon | STARTED | Cystagon First, Then RP103, Then Cystagon | 22 | |
| Run-in Period of 2-3 Weeks on Cystagon | COMPLETED | Cystagon First, Then Cystagon, Then RP103 | 21 | |
| Run-in Period of 2-3 Weeks on Cystagon | COMPLETED | Cystagon First, Then RP103, Then Cystagon | 22 | |
| Run-in Period of 2-3 Weeks on Cystagon | NOT COMPLETED | Cystagon First, Then Cystagon, Then RP103 | 0 | |
| Run-in Period of 2-3 Weeks on Cystagon | NOT COMPLETED | Cystagon First, Then RP103, Then Cystagon | 0 | |
| First Intervention (3 Weeks) | STARTED | Cystagon First, Then Cystagon, Then RP103 | 21 | |
| First Intervention (3 Weeks) | STARTED | Cystagon First, Then RP103, Then Cystagon | 22 | |
| First Intervention (3 Weeks) | COMPLETED | Cystagon First, Then Cystagon, Then RP103 | 21 | |
| First Intervention (3 Weeks) | COMPLETED | Cystagon First, Then RP103, Then Cystagon | 20 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events22
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Vomiting | Gastrointestinal disorders | RP103 | 5 | 43 | 8 |
| other | Vomiting | Gastrointestinal disorders | Cystagon® | 3 | 41 | 5 |
| other | Nausea | Gastrointestinal disorders | RP103 | 5 | 43 | 7 |
| other | Nausea | Gastrointestinal disorders | Cystagon® | 2 | 41 | 3 |
| other | Abdominal Pain | Gastrointestinal disorders | RP103 | 3 | 43 | 4 |
| other | Abdominal Pain | Gastrointestinal disorders | Cystagon® | 0 | 41 | 0 |
| other | Hypokalaemia | Metabolism and nutrition disorders | RP103 | 3 | 43 | 3 |
| other | Hypokalaemia | Metabolism and nutrition disorders | Cystagon® | 0 | 41 | 0 |
| serious | Knee deformity | Musculoskeletal and connective tissue disorders | RP103 | 1 | 43 | 1 |
| serious | Knee deformity | Musculoskeletal and connective tissue disorders | Cystagon® | 0 | 41 | 0 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RP103 | ||
| Total, all-cause mortality | Cystagon® | ||
| Total, other adverse events | RP103 | 16 | 43 |
| Total, other adverse events | Cystagon® | 5 | 41 |
| Total, serious adverse events | RP103 | 6 | 43 |
| Total, serious adverse events | Cystagon® | 1 | 41 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| RP103 vs Cystagon®: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01000961
GET https://conductscience.org/api/trials/v1/NCT01000961/events?page=2