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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-002773-64 | EudraCT Number |
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The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis patients are taking called Cystagon®.
In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of Cystagon® (cysteamine bitartrate) reduces cystine in the body. RP103 has the same active ingredient as Cystagon® and is designed to reduce cystine in a similar way that Cystagon® does. To decide if RP103 is better than Cystagon®, the study will look at two types of blood tests. One test is pharmacodynamics (PD), which measures the amount of white blood cell (WBC) cystine after taking study drug. WBC cystine is a laboratory test used to find out if cysteamine bitartrate is reducing cystine levels in the body. The second test is pharmacokinetics (PK), which measures the amount of cysteamine in the blood after taking the drug.
RP103 is different from Cystagon®: Instead of the cysteamine bitartrate being absorbed from the stomach, RP103 is designed to be absorbed from the small intestine. This may make the effects of the drug last longer, so that it can be taken twice a day instead of four times a day like Cystagon®.
Some cystinosis patients have bad breath (halitosis) when they take Cystagon®. Study participants who experience bad breath with Cystagon® will be asked if they would like to participate in an optional "halitosis substudy" to investigate this issue by collecting some extra PK blood samples.
Study with completed results acquired from Horizon in 2024.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | Experimental | Cystagon® Phase: From Screening and during Months 1, 2, 3 participants receive their usual dose of Cystagon® every 6 hours (Q6H). RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants receive RP103 every 12 hours (Q12H). Long Term Phase: On or after Month 7, for the remainder of study participants receive RP103 Q12H. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RP103 | Drug |
|
| |
| Measure | Description | Time Frame |
|---|---|---|
| Average Difference Between Morning and Non-Morning Log White Blood Cell (WBC) Cystine Values | The primary analysis of WBC cystine was performed using the natural log transformed WBC cystine level; the log transformation is a normalizing transformation. For each participant, the difference between the morning and corresponding non-morning log WBC cystine value (non-morning minus morning) at each monthly visit during the Cystagon® phase (Months 1, 2, and 3) was computed and these differences were averaged. The average difference between morning and non-morning log WBC cystine value was similarly computed for each participant during the RP103 phase (Months 5, 6, and 7). The primary analysis compared within-subject pairs (Cystagon® phase paired with RP103 phase) of non-morning minus morning average differences of log WBC cystine level. | While taking Cystagon® (Months 1, 2, 3): within 15 minutes pre-morning (AM) and pre-non AM dose. During 3 months of RP103 (Months 5, 6, 7): 30 minutes post-AM and post-evening (PM) dose. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | AE: any untoward medical occurrence that does not necessarily have a causal relationship with study drug. SAE: any untoward medical occurrence that at any dose: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; is a congenital anomaly or birth defect; or is medically significant, and though not included in the above list, is an important medical event, according to the Investigator. Treatment-emergent adverse events (TEAEs) occurred after first dose of study drug. Clinically significant abnormalities in laboratory values (hematology, blood chemistry, urinalysis), electrocardiograms (ECGs), vital signs, and physical examinations were to be reported as adverse events and so are included in this summary of TEAEs |
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INCLUSION CRITERIA:
EXCLUSION CRITERIA:
Younger than 12 years of age
Current history of the following conditions or any other health issues that make it, in the opinion of the investigator, unsafe for study participation:
Hemoglobin level of < 9 g/dL at Screening or, in the opinion of the investigator, a hemoglobin level that would make it unsafe for study participation
Known hypersensitivity to cysteamine and penicillamine
Female subjects who are nursing, planning a pregnancy, or are known or suspected to be pregnant
Subjects who, in the opinion of the investigator, are not able or willing to comply with study requirements.
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| California Pacific Medical Center (CPMC) Research Institute | San Francisco | California | 94115 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 19158356 | Background | Schwartz GJ, Munoz A, Schneider MF, Mak RH, Kaskel F, Warady BA, Furth SL. New equations to estimate GFR in children with CKD. J Am Soc Nephrol. 2009 Mar;20(3):629-37. doi: 10.1681/ASN.2008030287. Epub 2009 Jan 21. |
| Label | URL |
|---|---|
| RP103 (marketed as PROCYSBI) is now approved by the US FDA for management of nephropathic cystinosis in patients 6 years and older. | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | All Participants | Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® every 6 hours (Q6H). RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 every 12 hours (Q12H). Long Term Phase: On or after Month 7, for the remainder of study participants received RP103 Q12H. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Cystagon® Phase |
|
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| Cystagon® |
| Drug |
|
|
| From first dose of study drug to 7 days after last dose. Median duration of exposure was 91 days (range 82-108) for Cystagon® phase, 119 days (range 98-137) for the RP103 phase, and 861 days (range 30 - 1350) during the long-term RP-103 phase. |
| Halitosis Substudy: Maximum Plasma Concentration (Cmax) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of dimethylsulfide (DMS) in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state pharmacokinetic (PK) samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
| Halitosis Substudy: Time to Cmax (Tmax) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
| Area Under the Plasma Concentration Time Curve From Time Point 0 Through the Last Measurable Point (AUC0-t) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
| Halitosis Substudy: Expired Air DMS Concentrations | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose 30 min post-dose, 2, 3, 4 and 6 hours post-dose. While taking RP103 (Month 4, 5, or 7): Within 15 min. prior to morning dose. 1, 2, 3, 4, 5, 6, 8, 10, 12 hours post dose |
| Stanford University Medical School |
| Stanford |
| California |
| 94305 |
| United States |
| Emory Children's Center | Atlanta | Georgia | 30322 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60614 | United States |
| Baylor College of Medicine / Texas Childrens Hospital | Houston | Texas | 77030 | United States |
| University Hospital of Leuven | Leuven | Belgium |
| Hospices Civils de Lyon | Lyon | France |
| Hôpital Necker-Enfants Malades | Paris | France |
| Hôpital Robert Debré | Paris | France |
| Ospedale Pediatrico Bambino Gesù | Rome | Italy |
| Radboud University Nijmegen Medical Center | Nijmegen | Netherlands |
| Queen Elizabeth Hospital Birmingham | Birmingham | United Kingdom |
| Great Ormond Street | London | United Kingdom |
| Guy's Hospital | London | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
|
| RP103 Phase |
|
|
| Long Term Extension Phase |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | All Participants | Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H. RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H. Long Term Phase: On or after Month 7, for the remainder of study participants received RP103 Q12H. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Average Difference Between Morning and Non-Morning Log White Blood Cell (WBC) Cystine Values | The primary analysis of WBC cystine was performed using the natural log transformed WBC cystine level; the log transformation is a normalizing transformation. For each participant, the difference between the morning and corresponding non-morning log WBC cystine value (non-morning minus morning) at each monthly visit during the Cystagon® phase (Months 1, 2, and 3) was computed and these differences were averaged. The average difference between morning and non-morning log WBC cystine value was similarly computed for each participant during the RP103 phase (Months 5, 6, and 7). The primary analysis compared within-subject pairs (Cystagon® phase paired with RP103 phase) of non-morning minus morning average differences of log WBC cystine level. | Pharmacodynamic (PD) Analysis Set: All participants who received at least one treatment of Cystagon® and RP103 and who had at least one WBC cystine level recorded after each of Cystagon® treatment and RP103 treatment. Only participants with an average difference during both the Cystagon® and RP103 phases were included. | Posted | Mean | Standard Deviation | log [nmol ½ cystine/mg protein] | While taking Cystagon® (Months 1, 2, 3): within 15 minutes pre-morning (AM) and pre-non AM dose. During 3 months of RP103 (Months 5, 6, 7): 30 minutes post-AM and post-evening (PM) dose. |
|
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | AE: any untoward medical occurrence that does not necessarily have a causal relationship with study drug. SAE: any untoward medical occurrence that at any dose: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; is a congenital anomaly or birth defect; or is medically significant, and though not included in the above list, is an important medical event, according to the Investigator. Treatment-emergent adverse events (TEAEs) occurred after first dose of study drug. Clinically significant abnormalities in laboratory values (hematology, blood chemistry, urinalysis), electrocardiograms (ECGs), vital signs, and physical examinations were to be reported as adverse events and so are included in this summary of TEAEs | Safety Analysis Set: all participants who received at least 1 dose of study drug (RP103). The row "At least 1 TEAE" includes both serious and non-serious AEs. | Posted | Count of Participants | Participants | From first dose of study drug to 7 days after last dose. Median duration of exposure was 91 days (range 82-108) for Cystagon® phase, 119 days (range 98-137) for the RP103 phase, and 861 days (range 30 - 1350) during the long-term RP-103 phase. |
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| Secondary | Halitosis Substudy: Maximum Plasma Concentration (Cmax) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of dimethylsulfide (DMS) in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state pharmacokinetic (PK) samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint. | Posted | Mean | Standard Deviation | mg/L | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
|
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| Secondary | Halitosis Substudy: Time to Cmax (Tmax) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint. | Posted | Mean | Standard Deviation | hour | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
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| Secondary | Area Under the Plasma Concentration Time Curve From Time Point 0 Through the Last Measurable Point (AUC0-t) for Plasma Cysteamine | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint. | Posted | Mean | Standard Deviation | hr*mg/L | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose |
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| Secondary | Halitosis Substudy: Expired Air DMS Concentrations | Participants who reported halitosis ("bad breath") as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered. | PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint. | Posted | Mean | Standard Deviation | nmol/L | While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose 30 min post-dose, 2, 3, 4 and 6 hours post-dose. While taking RP103 (Month 4, 5, or 7): Within 15 min. prior to morning dose. 1, 2, 3, 4, 5, 6, 8, 10, 12 hours post dose |
|
|
From first dose of study drug to 7 days after last dose. Median duration of exposure was 91 days (range 82-108) for Cystagon® phase, 119 days (range 98-137) for the RP103 phase, and 861 days (range 30-1350) during the long term RP-103 phase.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cystagon® Phase | From Screening and during Months 1, 2, 3: participants received their usual dose of Cystagon® Q6H. | 0 | 41 | 5 | 41 | 28 | 41 |
| EG001 | RP103 Phase | During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H. | 0 | 41 | 6 | 41 | 33 | 41 |
| EG002 | Long-Term Phase | From Month 7 and for the remainder of study: participants received RP103 Q12H. | 1 | 38 | 13 | 38 | 32 | 38 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Death | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Arteriovenous fistula operation | Surgical and medical procedures | MedDRA 15.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Psychotic disorder | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Arteriovenous fistula thrombosis | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Procedural hypotension | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hyperkalemia | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Migraine with aura | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Testicular pain | Reproductive system and breast disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Peripheral artery thrombosis | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Arteriovenous fistula aneurysm | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Ossiculoplasty | Surgical and medical procedures | MedDRA 15.1 | Systematic Assessment |
| |
| Renal transplant | Surgical and medical procedures | MedDRA 15.1 | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Arteriovenous fistula site complication | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Arteriovenous fistula site haemorrhage | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis norovirus | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Graft infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
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| Conjunctivitis | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Candidiasis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 15.1 | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Knee deformity | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Evelyn Olson, Director | Horizon Pharma USA, Inc. | 224-383-3000 | clinicaltrials@horizonpharma.com |
| ID | Term |
|---|---|
| D003554 | Cystinosis |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D016464 | Lysosomal Storage Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D003543 | Cysteamine |
| ID | Term |
|---|---|
| D008624 | Mercaptoethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
| D013438 | Sulfhydryl Compounds |
| D013457 | Sulfur Compounds |
Not provided
Not provided
| OG001 | RP103 Phase | During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H. Observations on or after the first dose of RP103 up to Month 7 or study termination visit (whichever occurred first) were attributed to the RP103 Phase. |
| OG002 | Long-Term Phase | From Month 7 and for the remainder of study: participants received RP103 Q12H. Observations on or after the Month 7 visit through study termination were attributed to the Long-Term RP103 Phase. |
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