Time Frame |
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.
|
Adverse Event Reporting Description |
[Not Specified]
|
|
Arm/Group Title
|
Cystagon® Phase
|
RP103 Phase
|
Long-Term Phase
|
Arm/Group Description |
From Screening and during Months 1,...
|
During Months 3.5, 4, 5, 6, 7: part...
|
From Month 7 and for the remainder ...
|
Arm/Group Description |
From Screening and during Months 1, 2, 3: participants received their usual dose of Cystagon® Q6H.
|
During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H.
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From Month 7 and for the remainder of study: participants received RP103 Q12H.
|
|
|
Cystagon® Phase
|
RP103 Phase
|
Long-Term Phase
|
|
Affected / at Risk (%) |
Affected / at Risk (%) |
Affected / at Risk (%) |
Total |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
|
|
Cystagon® Phase
|
RP103 Phase
|
Long-Term Phase
|
|
Affected / at Risk (%) |
Affected / at Risk (%) |
Affected / at Risk (%) |
Total |
5/41 (12.20%) |
6/41 (14.63%) |
13/38 (34.21%) |
Blood and lymphatic system disorders |
|
|
|
Anaemia |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Gastrointestinal disorders |
|
|
|
Abdominal pain |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Diarrhoea |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Nausea |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Vomiting |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
General disorders |
|
|
|
Chest pain |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Death |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Malaise |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Fatigue |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Pyrexia |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Infections and infestations |
|
|
|
Bacteraemia |
0/41 (0.00%) |
1/41 (2.44%) |
0/38 (0.00%) |
Cellulitis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Diverticulitis |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Gastroenteritis |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Lung infection |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Pneumonia |
1/41 (2.44%) |
0/41 (0.00%) |
1/38 (2.63%) |
Sepsis |
1/41 (2.44%) |
0/41 (0.00%) |
1/38 (2.63%) |
Septic shock |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Urinary tract infection |
1/41 (2.44%) |
1/41 (2.44%) |
0/38 (0.00%) |
Gastroenteritis norovirus |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Gastroenteritis viral |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Graft infection |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Onychomycosis |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Tonsillitis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Injury, poisoning and procedural complications |
|
|
|
Arteriovenous fistula thrombosis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Procedural hypotension |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Arteriovenous fistula aneurysm |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Arteriovenous fistula site complication |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Arteriovenous fistula site haemorrhage |
0/41 (0.00%) |
1/41 (2.44%) |
1/38 (2.63%) |
Metabolism and nutrition disorders |
|
|
|
Dehydration |
0/41 (0.00%) |
0/41 (0.00%) |
4/38 (10.53%) |
Hyperkalemia |
0/41 (0.00%) |
1/41 (2.44%) |
0/38 (0.00%) |
Hypocalcaemia |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Musculoskeletal and connective tissue disorders |
|
|
|
Scoliosis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
|
|
Squamous cell carcinoma |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Nervous system disorders |
|
|
|
Migraine with aura |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Cerebral haemorrhage |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Dizziness |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Headache |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Loss of consciousness |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Syncope |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Psychiatric disorders |
|
|
|
Depression |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Psychotic disorder |
0/41 (0.00%) |
1/41 (2.44%) |
0/38 (0.00%) |
Renal and urinary disorders |
|
|
|
Renal failure acute |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Reproductive system and breast disorders |
|
|
|
Testicular pain |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Respiratory, thoracic and mediastinal disorders |
|
|
|
Acute respiratory failure |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Epistaxis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Pneumonitis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Surgical and medical procedures |
|
|
|
Arteriovenous fistula operation |
1/41 (2.44%) |
0/41 (0.00%) |
0/38 (0.00%) |
Ossiculoplasty |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Renal transplant |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Vascular disorders |
|
|
|
Hypertension |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Peripheral artery thrombosis |
0/41 (0.00%) |
0/41 (0.00%) |
1/38 (2.63%) |
Term from vocabulary, MedDRA 15.1
Indicates events were collected by systematic assessment
|
|
Frequency Threshold for Reporting Other Adverse Events
|
5%
|
|
Cystagon® Phase
|
RP103 Phase
|
Long-Term Phase
|
|
Affected / at Risk (%) |
Affected / at Risk (%) |
Affected / at Risk (%) |
Total |
28/41 (68.29%) |
33/41 (80.49%) |
32/38 (84.21%) |
Blood and lymphatic system disorders |
|
|
|
Anaemia |
0/41 (0.00%) |
1/41 (2.44%) |
4/38 (10.53%) |
Eye disorders |
|
|
|
Conjunctivitis |
0/41 (0.00%) |
1/41 (2.44%) |
3/38 (7.89%) |
Gastrointestinal disorders |
|
|
|
Abdominal pain |
1/41 (2.44%) |
3/41 (7.32%) |
5/38 (13.16%) |
Abdominal pain upper |
2/41 (4.88%) |
6/41 (14.63%) |
3/38 (7.89%) |
Constipation |
0/41 (0.00%) |
2/41 (4.88%) |
3/38 (7.89%) |
Diarrhoea |
4/41 (9.76%) |
9/41 (21.95%) |
7/38 (18.42%) |
Nausea |
4/41 (9.76%) |
15/41 (36.59%) |
5/38 (13.16%) |
Vomiting |
2/41 (4.88%) |
11/41 (26.83%) |
7/38 (18.42%) |
General disorders |
|
|
|
Influenza like illness |
0/41 (0.00%) |
0/41 (0.00%) |
4/38 (10.53%) |
Malaise |
3/41 (7.32%) |
1/41 (2.44%) |
1/38 (2.63%) |
Pyrexia |
2/41 (4.88%) |
2/41 (4.88%) |
2/38 (5.26%) |
Infections and infestations |
|
|
|
Bronchitis |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Candidiasis |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Ear infection |
0/41 (0.00%) |
0/41 (0.00%) |
3/38 (7.89%) |
Gastroenteritis |
1/41 (2.44%) |
4/41 (9.76%) |
4/38 (10.53%) |
Influenza |
0/41 (0.00%) |
0/41 (0.00%) |
4/38 (10.53%) |
Nasopharyngitis |
8/41 (19.51%) |
5/41 (12.20%) |
6/38 (15.79%) |
Respiratory tract infection |
1/41 (2.44%) |
2/41 (4.88%) |
2/38 (5.26%) |
Upper respiratory tract infection |
3/41 (7.32%) |
0/41 (0.00%) |
6/38 (15.79%) |
Urinary tract infection |
2/41 (4.88%) |
3/41 (7.32%) |
4/38 (10.53%) |
Viral infection |
1/41 (2.44%) |
2/41 (4.88%) |
2/38 (5.26%) |
Viral upper respiratory tract infection |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Investigations |
|
|
|
Blood creatinine increased |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Hepatic enzyme increased |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Weight decreased |
1/41 (2.44%) |
1/41 (2.44%) |
3/38 (7.89%) |
Metabolism and nutrition disorders |
|
|
|
Dehydration |
1/41 (2.44%) |
0/41 (0.00%) |
2/38 (5.26%) |
Musculoskeletal and connective tissue disorders |
|
|
|
Arthralgia |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Knee deformity |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Nervous system disorders |
|
|
|
Headache |
8/41 (19.51%) |
6/41 (14.63%) |
7/38 (18.42%) |
Renal and urinary disorders |
|
|
|
Renal impairment |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Reproductive system and breast disorders |
|
|
|
Dysmenorrhoea |
0/41 (0.00%) |
1/41 (2.44%) |
2/38 (5.26%) |
Respiratory, thoracic and mediastinal disorders |
|
|
|
Cough |
2/41 (4.88%) |
1/41 (2.44%) |
3/38 (7.89%) |
Skin and subcutaneous tissue disorders |
|
|
|
Skin lesion |
0/41 (0.00%) |
0/41 (0.00%) |
2/38 (5.26%) |
Term from vocabulary, MedDRA 15.1
Indicates events were collected by systematic assessment
|