Trial Outcomes & Findings for A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002] (NCT NCT01022424)
NCT ID: NCT01022424
Last Updated: 2018-09-11
Results Overview
Individual subject data on the volumes of the total kidney volume (sum of the volumes of the left and right kidneys) measured by magnetic resonance imaging or computed tomography during trial period. Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
13 participants
Primary outcome timeframe
Baseline, Week 48, 96, 144, and 192
Results posted on
2018-09-11
Participant Flow
Participant milestones
| Measure |
OPC-41061
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
OPC-41061
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
eGFR under the withdrawal criteria
|
2
|
Baseline Characteristics
A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002]
Baseline characteristics by cohort
| Measure |
OPC-41061
n=13 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
45.6 years
STANDARD_DEVIATION 9.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
13 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 48, 96, 144, and 192Individual subject data on the volumes of the total kidney volume (sum of the volumes of the left and right kidneys) measured by magnetic resonance imaging or computed tomography during trial period. Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.
Outcome measures
| Measure |
Baseline
n=13 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 48
n=12 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 96
n=12 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 144
n=11 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 192
n=10 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|---|---|---|---|
|
Total Kidney Volume
|
1428 mL
Interval 789.0 to 4774.0
|
1675 mL
Interval 786.0 to 4457.0
|
1728 mL
Interval 791.0 to 4193.0
|
1775 mL
Interval 818.0 to 4125.0
|
1901 mL
Interval 837.0 to 3669.0
|
PRIMARY outcome
Timeframe: Baseline, Week 48, 96, 144, and 192Individual subject data on eGFR (estimated glomerular filtration rate calculated by Japanese eGFR equation) during trial period. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.
Outcome measures
| Measure |
Baseline
n=13 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 48
n=12 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 96
n=12 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 144
n=11 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
Week 192
n=10 Participants
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|---|---|---|---|
|
Renal Function Test (eGFR)
|
63.0 mL/min/1.73 m2
Interval 31.0 to 93.0
|
60.0 mL/min/1.73 m2
Interval 23.0 to 92.0
|
54.0 mL/min/1.73 m2
Interval 22.0 to 113.0
|
50.5 mL/min/1.73 m2
Interval 15.0 to 63.0
|
51.0 mL/min/1.73 m2
Interval 16.0 to 64.0
|
Adverse Events
OPC-41061
Serious events: 6 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OPC-41061
n=13 participants at risk
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|
|
Eye disorders
Retinal vein occlusion
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Abdominal pain
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Large intestine polyp
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
General disorders
Pyrexia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Hepatic cyst infection
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Pyelonephritis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Psychiatric disorders
Alcoholism
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Vascular disorders
Hypertension
|
7.7%
1/13 • Through study completion, an average of 4 years
|
Other adverse events
| Measure |
OPC-41061
n=13 participants at risk
Repeated oral administration at doses of 15 mg twice daily (morning and evening) until approval of the revised protocol (Edition 4.0) by the IRB of each trial site.
Repeated oral administration at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg twice daily (morning and evening) following approval of the revised protocol.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Blood and lymphatic system disorders
Nephrogenic anaemia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Cardiac disorders
Sinus tachycardia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Cardiac disorders
Supraventricular extrasystoles
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Cardiac disorders
Ventricular extrasystoles
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Ear and labyrinth disorders
Tinnitus
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Angle closure glaucoma
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Asthenopia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Cataract
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Conjunctival haemorrhage
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Glaucoma
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Meibomianitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Ocular hyperaemia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Retinal vein occlusion
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Eye disorders
Eyelids pruritus
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Abdominal pain
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Dental caries
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Diarrhoea
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Diverticulum intestinal
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Enterocolitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Gastritis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Hiatus hernia
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Hyperchlorhydria
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Gastrointestinal disorders
Large intestine polyp
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
General disorders
Chest discomfort
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
General disorders
Oedema peripheral
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
General disorders
Pyrexia
|
23.1%
3/13 • Through study completion, an average of 4 years
|
|
General disorders
Thirst
|
46.2%
6/13 • Through study completion, an average of 4 years
|
|
Hepatobiliary disorders
Biliary cirrhosis primary
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Hepatobiliary disorders
Cholelithiasis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Immune system disorders
Seasonal allergy
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Acute tonsillitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Bronchitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Conjunctivitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Cystitis
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Gastroenteritis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Herpes zoster
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Hordeolum
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Influenza
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Nasopharyngitis
|
61.5%
8/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Periodontitis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Pertussis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Pyelonephritis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Hepatic cyst infection
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Infections and infestations
Enterocolitis viral
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Injury, poisoning and procedural complications
Radius fracture
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Injury, poisoning and procedural complications
Rib fracture
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Alanine aminotransferase increased
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Investigations
Aspartate aminotransferase increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood antidiuretic hormone increased
|
53.8%
7/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood bilirubin increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood creatine phosphokinase increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood creatinine increased
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood lactate dehydrogenase increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood osmolarity increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood triglycerides increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood urea increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Electrocardiogram T wave inversion
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Haematocrit increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Haemoglobin increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Intraocular pressure increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Liver function test abnormal
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Red blood cell count increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Blood phosphorus decreased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Cystatin C increased
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Investigations
Urinary casts present
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Metabolism and nutrition disorders
Dehydration
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Metabolism and nutrition disorders
Gout
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
30.8%
4/13 • Through study completion, an average of 4 years
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
38.5%
5/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Exostosis of jaw
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Periarthritis
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Seborrhoeic keratosis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Nervous system disorders
Headache
|
30.8%
4/13 • Through study completion, an average of 4 years
|
|
Nervous system disorders
Hyperaesthesia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Psychiatric disorders
Alcoholism
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Psychiatric disorders
Depression
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Psychiatric disorders
Eating disorder
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Psychiatric disorders
Insomnia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Renal and urinary disorders
Nocturia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Renal and urinary disorders
Pollakiuria
|
30.8%
4/13 • Through study completion, an average of 4 years
|
|
Renal and urinary disorders
Polyuria
|
23.1%
3/13 • Through study completion, an average of 4 years
|
|
Renal and urinary disorders
Renal cyst haemorrhage
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Renal and urinary disorders
Renal impairment
|
15.4%
2/13 • Through study completion, an average of 4 years
|
|
Reproductive system and breast disorders
Endometriosis
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Reproductive system and breast disorders
Menopausal symptoms
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Reproductive system and breast disorders
Metrorrhagia
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar inflammation
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Chloasma
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Eczema
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Haemorrhage subcutaneous
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Skin and subcutaneous tissue disorders
Urticaria cholinergic
|
7.7%
1/13 • Through study completion, an average of 4 years
|
|
Vascular disorders
Hypertension
|
53.8%
7/13 • Through study completion, an average of 4 years
|
Additional Information
Director of Clinical Trials
Otsuka Pharmaceutical Co.,Ltd.
Phone: +81-3-6361-7366
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place