Trial · NCT00872729
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
NCT00872729, Phase 1, Phase 2, Completed, Amgen; results first posted 2014-10-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis. Trials, record NCT00872729. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00872729
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
- NCT number
- NCT00872729
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Cystinosis
- Interventions
- Cystagon®, RP103
- First posted
- 2009-03-31
- Last updated
- 2024-12-19
- Results first posted
- 2014-10-28
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Plasma Pharmacokinetic Parameter: Cmax of Cysteamine | 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing | |
| Primary | Plasma Pharmacokinetic Parameter: Tmax of Cysteamine | 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing | |
| Primary | Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine | 12 hours post RP103 dosing and 6 hours post 1st Cystagon® dosing | t = 6 for Cystagon and t = 12 for RP103. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours. |
| Primary | Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | up to 12 hours post Cystagon® dosing and RP103 dosing | The pharmacodynamic (PD) parameter measures the changes of WBC cystine level from the baseline. Cystine is a disulfide amino acid formed through oxidation of two molecules of cysteine; hence, cystine's concentration is commonly given in half-cystine equivalents to avoid confusion. The level of cystine in WBC/leukocytes is expressed in units of nmol half-cystine/mg protein (nmol ½ cystine/mg protein). Half-cystine is quantified by a reduction of cystine followed by an assay for cysteine, which is then normalized by the total cellular protein content within the sample using methods of such as Lowry assay, bicinchoninic acid assay, or Bradford. |
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 | Plasma Pharmacokinetic Parameter: Cmax of Cysteamine | 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing | umol/L | GEOMETRIC_MEAN | |
| PRIMARY | Plasma Pharmacokinetic Parameter: Tmax of Cysteamine | 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing | hour | MEDIAN | |
| PRIMARY | Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine | 12 hours post RP103 dosing and 6 hours post 1st Cystagon® dosing | Subjects were enrolled sequentially according to the study design. A mixed-effects linear model was used to assess differences between the RP103 and Cystagon treatment groups. Sample size is based on feasibility rather than statistical considerations. | umol•h/L | GEOMETRIC_MEAN |
| PRIMARY | Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | up to 12 hours post Cystagon® dosing and RP103 dosing | Sample size is based on feasibility rather than statistical considerations. Analysis time differences (0-6 hours) is due to different absorption characteristics of Cysteamine between RP103 and Cystagon. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours. | 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 |
|---|---|---|---|---|---|
| Plasma Pharmacokinetic Parameter: Cmax of Cysteamine | Cystagon® | 42.02 | Geometric Coefficient of Variation 51 | umol/L | |
| Plasma Pharmacokinetic Parameter: Cmax of Cysteamine | RP103 | 33.06 | Geometric Coefficient of Variation 55 | umol/L | |
| Plasma Pharmacokinetic Parameter: Tmax of Cysteamine | Cystagon® | 1.00 | Full Range 0.50 to 2.00 | hour | |
| Plasma Pharmacokinetic Parameter: Tmax of Cysteamine | RP103 | 3.00 | Full Range 0.00 to 5.13 | hour | |
| Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine | Cystagon® | 107.89 | Geometric Coefficient of Variation 58 | umol•h/L | |
| Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine | RP103 | 119.17 | Geometric Coefficient of Variation 46 | umol•h/L | |
| Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | Cystagon® (0.5 Hour) | 0.11 | Standard Deviation 0.24 | nmol 1/2 cystine/mg protein | |
| Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | Cystagon® (1 Hour) | -0.10 | Standard Deviation 0.02 | nmol 1/2 cystine/mg protein | |
| Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | RP103 (1 Hour) | 0.87 | Standard Deviation 0.33 | nmol 1/2 cystine/mg protein | |
| Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline | Cystagon® (2 Hour) | -0.07 | Standard Deviation 0.28 | nmol 1/2 cystine/mg protein |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cystagon® and RP103 | 9 | |
| Overall Study | COMPLETED | Cystagon® and RP103 | 9 | |
| Overall Study | NOT COMPLETED | Cystagon® and RP103 | 0 |
Source ClinicalTrials.gov
Adverse events24
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 | Cystagon® | 4 | 9 | 4 |
| other | Vomiting | Gastrointestinal disorders | RP103 | 0 | 9 | 0 |
| other | Nausea | Gastrointestinal disorders | Cystagon® | 3 | 9 | 3 |
| other | Nausea | Gastrointestinal disorders | RP103 | 0 | 9 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Cystagon® | 1 | 9 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | RP103 | 0 | 9 | 0 |
| other | Breath odor | Gastrointestinal disorders | Cystagon® | 1 | 9 | 1 |
| other | Breath odor | Gastrointestinal disorders | RP103 | 0 | 9 | 0 |
| other | Constipation | Gastrointestinal disorders | Cystagon® | 1 | 9 | 1 |
| other | Constipation | Gastrointestinal disorders | RP103 | 0 | 9 | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cystagon® | ||
| Total, all-cause mortality | RP103 | ||
| Total, other adverse events | Cystagon® | 8 | 9 |
| Total, other adverse events | RP103 | 2 | 9 |
| Total, serious adverse events | Cystagon® | 0 | 9 |
| Total, serious adverse events | RP103 | 0 | 9 |
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 |
|---|
| Cystagon® vs RP103: 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/NCT00872729
GET https://conductscience.org/api/trials/v1/NCT00872729/results?page=2