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Trial record 3 of 32 for:    CYSTEAMINE

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

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ClinicalTrials.gov Identifier: NCT01744782
Recruitment Status : Completed
First Posted : December 7, 2012
Results First Posted : January 16, 2018
Last Update Posted : February 14, 2018
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystinosis
Intervention Drug: RP103
Enrollment 17
Recruitment Details  
Pre-assignment Details Safety Population: All participants who received at least 1 dose of RP103.
Arm/Group Title RP103
Hide Arm/Group Description From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Period Title: Overall Study
Started 17
Completed 16
Not Completed 1
Reason Not Completed
Death             1
Arm/Group Title RP103
Hide Arm/Group Description From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Baseline Participants 17
Hide Baseline Analysis Population Description
Safety Population: All participants who received at least 1 dose of RP103.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants
3.80  (5.116)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
7
  41.2%
Male
10
  58.8%
1.Primary Outcome
Title Mean White Blood Cell (WBC) Cystine Concentration at Each Visit
Time Frame 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants <6 years of age who received at least 1 dose of RP103 and who had at least 1 WBC cystine level recorded.
Arm/Group Title RP103
Hide Arm/Group Description:
From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: nmol 1/2 Cystine/mg protein
Day 1 Number Analyzed 15 participants
3.1709  (2.95209)
Week 2 Number Analyzed 15 participants
2.2899  (3.13707)
Week 4 Number Analyzed 13 participants
1.8474  (1.62820)
Week 6 Number Analyzed 13 participants
2.3403  (4.31136)
Week 8 Number Analyzed 13 participants
0.9589  (1.05851)
Week 10 Number Analyzed 12 participants
1.1219  (1.27413)
Week 12 Number Analyzed 13 participants
1.1828  (1.31272)
Month 6 Number Analyzed 2 participants
2.7250  (1.08187)
Month 9 Number Analyzed 6 participants
2.0196  (1.90931)
Month 12 Number Analyzed 13 participants
0.8012  (0.59706)
Month 15 Number Analyzed 9 participants
1.2443  (1.47616)
Month 18 Number Analyzed 9 participants
0.7356  (0.64027)
Study Exit Number Analyzed 13 participants
0.8197  (0.76413)
2.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description 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.
Time Frame Day 1 through study exit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population: All participants who received at least 1 dose of RP103.
Arm/Group Title RP103
Hide Arm/Group Description:
From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Participants Analyzed 17
Measure Type: Count of Participants
Unit of Measure: Participants
17
 100.0%
3.Secondary Outcome
Title Maximum Observed Plasma Concentration (Cmax) of Cysteamine
Hide Description 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.
Time Frame 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants age <6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit.
Arm/Group Title RP103
Hide Arm/Group Description:
From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mg/L
1.26  (0.86)
4.Secondary Outcome
Title Time of the Maximum Observed Plasma Concentration (Tmax) of Cysteamine
Hide Description 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.
Time Frame 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants age <6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit.
Arm/Group Title RP103
Hide Arm/Group Description:
From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: minutes
199  (138)
5.Secondary Outcome
Title Area Under the Plasma Concentration Versus Time Curve (AUC) of Cysteamine
Hide Description 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.
Time Frame 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants age <6 years who received at least 1 dose of RP103 and participated in frequent sampling at the Month 6 visit.
Arm/Group Title RP103
Hide Arm/Group Description:
From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: min*mg/L
AUClast Number Analyzed 11 participants
206  (113)
AUCinf Number Analyzed 10 participants
231  (123)
Time Frame From Day 1 through study exit
Adverse Event Reporting Description Adverse events (AEs) were defined as any AE with onset following initial drug administration (on Day 1).
 
Arm/Group Title RP103
Hide Arm/Group Description From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules.
All-Cause Mortality
RP103
Affected / at Risk (%)
Total   1/17 (5.88%) 
Show Serious Adverse Events Hide Serious Adverse Events
RP103
Affected / at Risk (%)
Total   12/17 (70.59%) 
Blood and lymphatic system disorders   
Anaemia  1  1/17 (5.88%) 
Cardiac disorders   
Cardiopulmonary Failure  1  1/17 (5.88%) 
Congenital, familial and genetic disorders   
Fanconi Syndrome  1  1/17 (5.88%) 
Gastrointestinal disorders   
Vomiting  1  4/17 (23.53%) 
Abdominal Distension  1  1/17 (5.88%) 
Infections and infestations   
Gastroenteritis  1  5/17 (29.41%) 
Catheter Site Infection  1  1/17 (5.88%) 
Clostridium difficile Colitis  1  1/17 (5.88%) 
Gastroenteritis Viral  1  1/17 (5.88%) 
Metabolism and nutrition disorders   
Dehydration  1  4/17 (23.53%) 
Electrolyte Imbalance  1  2/17 (11.76%) 
Failure to Thrive  1  1/17 (5.88%) 
Hypernatraemia  1  1/17 (5.88%) 
Hypocalcaemia  1  1/17 (5.88%) 
Malnutrition  1  1/17 (5.88%) 
Metabolic Acidosis  1  1/17 (5.88%) 
Surgical and medical procedures   
Gastrostomy  1  2/17 (11.76%) 
Vascular disorders   
Hypovolaemic Shock  1  1/17 (5.88%) 
1
Term from vocabulary, MedDRA (16.1)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
RP103
Affected / at Risk (%)
Total   16/17 (94.12%) 
Blood and lymphatic system disorders   
Anaemia  1  1/17 (5.88%) 
Eye disorders   
Conjunctivitis  1  1/17 (5.88%) 
Strabismus  1  1/17 (5.88%) 
Gastrointestinal disorders   
Vomiting  1  12/17 (70.59%) 
Diarrhoea  1  6/17 (35.29%) 
Breath Odour  1  4/17 (23.53%) 
Nausea  1  3/17 (17.65%) 
Abdominal Pain  1  1/17 (5.88%) 
General disorders   
Pyrexia  1  4/17 (23.53%) 
Granuloma  1  1/17 (5.88%) 
Medical Device Site Reaction  1  1/17 (5.88%) 
Infections and infestations   
Upper Respiratory Tract Infection  1  8/17 (47.06%) 
Gastroenteritis  1  3/17 (17.65%) 
Rhinitis  1  3/17 (17.65%) 
Catheter Site Infection  1  1/17 (5.88%) 
Otitis Media  1  1/17 (5.88%) 
Pharyngitis  1  1/17 (5.88%) 
Sinusitis  1  1/17 (5.88%) 
Varicella  1  1/17 (5.88%) 
Metabolism and nutrition disorders   
Decreased Appetite  1  1/17 (5.88%) 
Hypernatraemia  1  1/17 (5.88%) 
Hypokalaemia  1  1/17 (5.88%) 
Malnutrition  1  1/17 (5.88%) 
Musculoskeletal and connective tissue disorders   
Foot Deformity  1  1/17 (5.88%) 
Nervous system disorders   
Headache  1  2/17 (11.76%) 
Reproductive system and breast disorders   
Perineal Erythema  1  1/17 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  4/17 (23.53%) 
Rhinorrhoea  1  2/17 (11.76%) 
Epistaxis  1  1/17 (5.88%) 
Skin and subcutaneous tissue disorders   
Dermatitis Diaper  1  2/17 (11.76%) 
Surgical and medical procedures   
Gastrostomy Closure  1  1/17 (5.88%) 
Gastrostomy Tube Removal  1  1/17 (5.88%) 
Vascular disorders   
Hypertension  1  1/17 (5.88%) 
1
Term from vocabulary, MedDRA (16.1)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Horizon requests that any Investigator/institution that plans on presenting or publishing results provide written notification of their request a minimum of 60 days prior to presentation or publication. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsors’ Intellectual Property rights .
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Evelyn Olson, Director
Organization: Horizon Pharma USA, Inc.
Phone: 224-383-3000
EMail: clinicaltrials@horizonpharma.com
Layout table for additonal information
Responsible Party: Horizon Pharma USA, Inc.
ClinicalTrials.gov Identifier: NCT01744782     History of Changes
Other Study ID Numbers: RP103-08
First Submitted: December 5, 2012
First Posted: December 7, 2012
Results First Submitted: December 13, 2017
Results First Posted: January 16, 2018
Last Update Posted: February 14, 2018