Trial · NCT01744782
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
NCT01744782, Phase 3, Completed, Amgen; results first posted 2018-01-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis. Trials, record NCT01744782. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01744782
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
- NCT number
- NCT01744782
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 17 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Cystinosis
- Interventions
- RP103
- First posted
- 2012-12-07
- Last updated
- 2024-12-27
- Results first posted
- 2018-01-16
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | Day 1, Week 2, Week 4, Week 6, Week 8, Week 10, Week 12, Month 6, Month 9, Month 12, Month 15, Month 18, Study Exit | Blood samples were taken 30 minutes after the morning RP103 dose at each study visit to determine White Blood Cell (WBC) cystine concentration. WBC cystine concentrations were determined using liquid chromatography. |
| Secondary | Number of Participants With Adverse Events | Day 1 through study exit | Safety was assessed by the incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs). An AE/adverse experience was any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. For additional information regarding adverse events, please see the safety section of the record. |
| Secondary | Maximum Observed Plasma Concentration (Cmax) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Blood samples were collected and plasma cysteamine concentration was determined using liquid chromatography. The maximum observed plasma concentration (Cmax) of cysteamine was determined directly from the data. |
| Secondary | Time of the Maximum Observed Plasma Concentration (Tmax) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Blood samples were collected and plasma cysteamine concentration was determined using liquid chromatography. The time of the maximum observed plasma concentration (Tmax) of cysteamine was determined directly from the data. |
| Secondary | Area Under the Plasma Concentration Versus Time Curve (AUC) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Blood samples were collected and plasma cysteamine concentration was determined using liquid chromatography. AUC values were estimated using non-compartmental analysis methods. AUClast was defined as the area under the plasma concentration versus time curve, from time 0 to the time of the last measurable concentration (720 minutes). AUCinf was defined as the area under the plasma concentration-versus-time curve from time 0 to infinity. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | Day 1, Week 2, Week 4, Week 6, Week 8, Week 10, Week 12, Month 6, Month 9, Month 12, Month 15, Month 18, Study Exit | Participants \<6 years of age who received at least 1 dose of RP103 and who had at least 1 WBC cystine level recorded. | nmol 1/2 Cystine/mg protein | MEAN |
| SECONDARY | Number of Participants With Adverse Events | Day 1 through study exit | Safety population: All participants who received at least 1 dose of RP103. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Observed Plasma Concentration (Cmax) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Participants age \<6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit. | mg/L | MEAN |
| SECONDARY | Time of the Maximum Observed Plasma Concentration (Tmax) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Participants age \<6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit. | minutes | MEAN |
| SECONDARY | Area Under the Plasma Concentration Versus Time Curve (AUC) of Cysteamine | 30 minutes after the morning RP103 dose at Month 6 (prior to Protocol Amendment 1) or 0 (pre-dose), 30 minutes, 2, 3, 4, 6, 8, 10, and 12 hours after the morning RP103 dose at Month 6 for those enrolled under Protocol Amendment 1 or later | Participants age \<6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit. | min*mg/L | MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Day 1 | 3.1709 | Standard Deviation 2.95209 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 2 | 2.2899 | Standard Deviation 3.13707 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 4 | 1.8474 | Standard Deviation 1.62820 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 6 | 2.3403 | Standard Deviation 4.31136 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 8 | 0.9589 | Standard Deviation 1.05851 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 10 | 1.1219 | Standard Deviation 1.27413 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Week 12 | 1.1828 | Standard Deviation 1.31272 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Month 6 | 2.7250 | Standard Deviation 1.08187 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Month 9 | 2.0196 | Standard Deviation 1.90931 | nmol 1/2 Cystine/mg protein |
| Mean White Blood Cell (WBC) Cystine Concentration at Each Visit | RP103 | Month 12 | 0.8012 | Standard Deviation 0.59706 | nmol 1/2 Cystine/mg protein |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RP103 | 17 | |
| Overall Study | COMPLETED | RP103 | 16 | |
| Overall Study | NOT COMPLETED | RP103 | 1 |
Source ClinicalTrials.gov
Adverse events51
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 | 12 | 17 | |
| other | Diarrhoea | Gastrointestinal disorders | RP103 | 6 | 17 | |
| other | Breath Odour | Gastrointestinal disorders | RP103 | 4 | 17 | |
| other | Nausea | Gastrointestinal disorders | RP103 | 3 | 17 | |
| other | Abdominal Pain | Gastrointestinal disorders | RP103 | 1 | 17 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | RP103 | 8 | 17 | |
| other | Gastroenteritis | Infections and infestations | RP103 | 3 | 17 | |
| other | Rhinitis | Infections and infestations | RP103 | 3 | 17 | |
| other | Catheter Site Infection | Infections and infestations | RP103 | 1 | 17 | |
| other | Otitis Media | Infections and infestations | RP103 | 1 | 17 |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RP103 | 1 | 17 |
| Total, other adverse events | RP103 | 16 | 17 |
| Total, serious adverse events | RP103 | 12 | 17 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 19158356 | BACKGROUND |
Source PubMed
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/NCT01744782
GET https://conductscience.org/api/trials/v1/NCT01744782/events?page=2