Trial · NCT01197378
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
NCT01197378, Phase 3, Completed, Amgen; results first posted 2018-07-24
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103). Trials, record NCT01197378. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01197378
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
- NCT number
- NCT01197378
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 60 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Cystinosis
- Interventions
- Cysteamine Bitartrate Delayed-release Capsules
- First posted
- 2010-09-09
- Last updated
- 2024-12-27
- Other registries' IDs
- eudract:2010-018365-34
- Results first posted
- 2018-07-24
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events | From first dose of study drug to 7 days after the last dose; median duration of treatment was 1461 days. | Drug-related adverse events (AEs) are AEs the investigator assessed as having relation to drug of 'possibly', 'probably' or 'definitely'. The severity of AEs was categorized according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0 as follows: * Mild (Grade 1): experience is minor and does not cause significant discomfort to subject or change in activities of daily living (ADL); subject is aware of symptoms but symptoms are easily tolerated; * Moderate (Grade 2): experience is an inconvenience or concern to the subject and causes interference with ADL, but the subject is able to continue with ADL. * Severe (Grade 3): experience significantly interferes with ADL and the subject is incapacitated and/or unable to continue with ADL * Life-threatening (Grade 4): experience that, in the view of the Investigator, places the subject at immediate risk of death from the event as it occurred. |
| Secondary | Trough Plasma Cysteamine Concentration | Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose | Plasma cysteamine concentration was determined using methods employing Hydrophilic Interaction Liquid Chromatography (HILC) high pressure liquid chromatography (HPLC) tandem mass spectrometry (HPLC-MS/MS). |
| Secondary | White Blood Cell Cystine Concentration | Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose | White blood cell (WBC) cystine concentration was determined using high performance liquid chromatography-electrospray ionization tandem mass spectrometry (LC-ESI-MS/MS). |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events | From first dose of study drug to 7 days after the last dose; median duration of treatment was 1461 days. | All participants who received at least 1 dose of cysteamine bitartrate. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Trough Plasma Cysteamine Concentration | Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose | The Pharmacokinetic/Pharmacodynamic (PK/PD) Population includes all participants who had at least one PK/PD measurement. Day 1 results only include participants who did not complete Study RP103-03. | mg/L | MEAN |
| SECONDARY | White Blood Cell Cystine Concentration | Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose | The Pharmacokinetic/Pharmacodynamic (PK/PD) Population includes all participants who had at least one PK/PD measurement. Day 1 results only include participants who did not complete Study RP103-03. | nmol 1/2 Cystine/mg protein | MEAN |
Source ClinicalTrials.gov
Results by group21
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events | Cysteamine Bitartrate | Any adverse event | 58 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events | Cysteamine Bitartrate | Adverse events related to study drug | 37 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events | Cysteamine Bitartrate | Adverse events ≥ Grade 3 | 24 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events | Cysteamine Bitartrate | Serious adverse events | 32 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events | Cysteamine Bitartrate | Adverse events leading to discontinuation | 3 | Participants | |
| Trough Plasma Cysteamine Concentration | Cysteamine Bitartrate | Day 1 | 0.17 | Standard Deviation 0.093 | mg/L |
| Trough Plasma Cysteamine Concentration | Cysteamine Bitartrate | Month 6 | 0.29 | Standard Deviation 0.613 | mg/L |
| Trough Plasma Cysteamine Concentration | Cysteamine Bitartrate | Year 1 | 0.37 | Standard Deviation 0.513 | mg/L |
| Trough Plasma Cysteamine Concentration | Cysteamine Bitartrate | Year 1.5 | 0.48 | Standard Deviation 0.718 | mg/L |
| Trough Plasma Cysteamine Concentration | Cysteamine Bitartrate | Year 2 | 0.36 | Standard Deviation 0.412 | mg/L |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cysteamine Bitartrate | 60 | |
| Overall Study | Received Treatment | Cysteamine Bitartrate | 59 | |
| Overall Study | COMPLETED | Cysteamine Bitartrate | 53 | |
| Overall Study | NOT COMPLETED | Cysteamine Bitartrate | 7 |
Source ClinicalTrials.gov
Adverse events113
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 | Cysteamine Bitartrate | 40 | 59 | |
| other | Diarrhoea | Gastrointestinal disorders | Cysteamine Bitartrate | 17 | 59 | |
| other | Nausea | Gastrointestinal disorders | Cysteamine Bitartrate | 16 | 59 | |
| other | Abdominal pain | Gastrointestinal disorders | Cysteamine Bitartrate | 12 | 59 | |
| other | Breath odour | Gastrointestinal disorders | Cysteamine Bitartrate | 8 | 59 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Cysteamine Bitartrate | 7 | 59 | |
| other | Constipation | Gastrointestinal disorders | Cysteamine Bitartrate | 4 | 59 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Cysteamine Bitartrate | 3 | 59 | |
| other | Dyspepsia | Gastrointestinal disorders | Cysteamine Bitartrate | 3 | 59 | |
| other | Flatulence | Gastrointestinal disorders | Cysteamine Bitartrate | 3 | 59 |
1 to 10 of 113Source ClinicalTrials.gov
See all 113 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cysteamine Bitartrate | 0 | 59 |
| Total, other adverse events | Cysteamine Bitartrate | 58 | 59 |
| Total, serious adverse events | Cysteamine Bitartrate | 32 | 59 |
Source ClinicalTrials.gov
Papers4
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT01197378
GET https://conductscience.org/api/trials/v1/NCT01197378/results?page=2