Ignite Creation Date:
2025-12-25 @ 2:17 AM
Ignite Modification Date:
2026-03-04 @ 11:18 PM
Study NCT ID:
NCT02225860
Status:
COMPLETED
Last Update Posted:
2017-03-30
First Post:
2014-08-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Diet as a Potential Treatment for Autosomal Dominant Polycystic Kidney Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016891', 'term': 'Polycystic Kidney, Autosomal Dominant'}, {'id': 'D003919', 'term': 'Diabetes Insipidus'}], 'ancestors': [{'id': 'D007690', 'term': 'Polycystic Kidney Diseases'}, {'id': 'D052177', 'term': 'Kidney Diseases, Cystic'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000072661', 'term': 'Ciliopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D010900', 'term': 'Pituitary Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D004032', 'term': 'Diet'}], 'ancestors': [{'id': 'D009747', 'term': 'Nutritional Physiological Phenomena'}, {'id': 'D000066888', 'term': 'Diet, Food, and Nutrition'}, {'id': 'D010829', 'term': 'Physiological Phenomena'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'rperrone@tuftsmedicalcenter.org', 'phone': '6176362372', 'title': 'Ronald D. Perrone', 'organization': 'Tufts Medical Center'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'Small sample size and short follow up duration.'}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment', 'otherNumAtRisk': 17, 'otherNumAffected': 0, 'seriousNumAtRisk': 17, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Control', 'description': 'Continue with usual diet', 'otherNumAtRisk': 17, 'otherNumAffected': 0, 'seriousNumAtRisk': 17, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Mean Serum Copeptin From Baseline (a Reflection of Endogenous Vasopressin Production) at Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment'}, {'id': 'OG001', 'title': 'Control', 'description': 'Continue with usual diet'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.86', 'spread': '1.3', 'groupId': 'OG000'}, {'value': '0.39', 'spread': '1.2', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.01', 'groupIds': ['OG000', 'OG001'], 'groupDescription': 'The analysis of the primary and secondary outcome was based on an intention to treat principle. Copeptin was measured in each subject at baseline and week 2 and the change from baseline to end point ("delta") was calculated for all subjects. The delta copeptin between subjects in the intervention and control arms was compared with a two sample t test for independent groups. One sample t-tests of the delta were used to examine whether there was any change over time for each group separately.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 2', 'description': 'The copeptin level will reflect the combined effect of low osmolar diet and adjusted water intake at week 2', 'unitOfMeasure': 'pmole/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Total Daily Urinary Solutes From Baseline to Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment'}, {'id': 'OG001', 'title': 'Control', 'description': 'Continue with usual diet'}], 'classes': [{'categories': [{'measurements': [{'value': '-120', 'spread': '212', 'groupId': 'OG000'}, {'value': '27', 'spread': '79', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.01', 'groupIds': ['OG000', 'OG001'], 'groupDescription': 'The analysis of the primary and secondary outcome was based on an intention to treat principle. Copeptin was measured in each subject at baseline and week 2 and the change from baseline to end point ("delta") was calculated for all subjects. The delta copeptin between subjects in the intervention and control arms was compared with a two sample t test for independent groups. One sample t-tests of the delta were used to examine whether there was any change over time for each group separately.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 2', 'description': 'Total daily urinary solutes (this will serve as a surrogate for diet adherence and is known to be associated with lower vasopressin secretion).\n\nTotal daily solutes is the total amount of osmoles detected in 24 hours urine collection.', 'unitOfMeasure': 'mOsm/Day', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Mean Serum Copeptin Level From Baseline to Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment: The dietary intervention consisted of three elements: low sodium (1500 mg/day), low protein (daily protein dietary allowance of 0.8 gram/kg body weight), and low urea (avoidance of preservatives, food additives, bulking agents, and chewing gum). Protein was factored by measured body weight to mirror the estimated average requirement (EAR) of healthy adults which is set on a grams per kilogram basis'}, {'id': 'OG001', 'title': 'Control', 'description': 'Continue with usual diet'}], 'classes': [{'categories': [{'measurements': [{'value': '0.9', 'spread': '1.2', 'groupId': 'OG000'}, {'value': '1.4', 'spread': '1.3', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline to week 1', 'description': 'Mean serum copeptin level at week one which will reflect the effect of low osmolar diet alone.', 'unitOfMeasure': 'pmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'At week 1, there was a nonsignificant (t-test) increase in plasma copeptin to 7.1 ±5.6 in the low osmolar diet group and to 6.1 ±5.5 in the control group. The change in mean plasma copeptin level between baseline and week1 was not statistically significant between groups.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment'}, {'id': 'FG001', 'title': 'Control', 'description': 'Continue with usual diet'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '34', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Diet and Water Adjustment', 'description': 'Reduction in dietary salt and protein intake\n\nDiet and water adjustment'}, {'id': 'BG001', 'title': 'Control', 'description': 'Continue with usual diet'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '34', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '34', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 34}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-02', 'completionDateStruct': {'date': '2016-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-02-10', 'studyFirstSubmitDate': '2014-08-20', 'resultsFirstSubmitDate': '2016-12-02', 'studyFirstSubmitQcDate': '2014-08-25', 'lastUpdatePostDateStruct': {'date': '2017-03-30', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-02-10', 'studyFirstPostDateStruct': {'date': '2014-08-26', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-03-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Mean Serum Copeptin From Baseline (a Reflection of Endogenous Vasopressin Production) at Week 2', 'timeFrame': 'Baseline to week 2', 'description': 'The copeptin level will reflect the combined effect of low osmolar diet and adjusted water intake at week 2'}], 'secondaryOutcomes': [{'measure': 'Change in Total Daily Urinary Solutes From Baseline to Week 2', 'timeFrame': 'Baseline to week 2', 'description': 'Total daily urinary solutes (this will serve as a surrogate for diet adherence and is known to be associated with lower vasopressin secretion).\n\nTotal daily solutes is the total amount of osmoles detected in 24 hours urine collection.'}, {'measure': 'Change in Mean Serum Copeptin Level From Baseline to Week 1', 'timeFrame': 'baseline to week 1', 'description': 'Mean serum copeptin level at week one which will reflect the effect of low osmolar diet alone.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['ADPKD', 'Copeptin', 'Vasopressin', 'Diet', 'Water'], 'conditions': ['Autosomal Dominant Polycystic Kidney Disease (ADPKD)']}, 'referencesModule': {'references': [{'pmid': '39356039', 'type': 'DERIVED', 'citation': 'St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to learn if dietary habits can affect vasopressin secretion in patients with autosomal dominant polycystic kidney disease. Vasopressin increases the growth of kidney cysts and accelerates disease progression. Understanding how to control secretion of this hormone based on dietary habits may help to develop treatments to control this disease. The study will include about 60 patients from Tufts Medical Center. The study will last for 2 weeks. Blood and urine tests will be done 3 times during the study period. Subjects will be randomly assigned (by chance like flipping a coin), to one of two study groups. Group 1 will be given instructions to adjust their diet. This will include adjusting the amount of water, protein, and salt intake. Group 2 will have no adjustment of diet or water. The project has tremendous public health relevance, given the large numbers of people affected by autosomal dominant polycystic kidney disease and the substantial impact of the disease on morbidity, mortality, hospitalizations,dialysis or transplant, and societal costs of caring for those patients.', 'detailedDescription': 'Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease with an estimated 600,000 persons affected in the United States and 12.5 million persons worldwide. To date, no disease-modifying treatment has been approved for the treatment of ADPKD.\n\nArginine vasopressin (AVP) is a key player in cyst enlargement and disease progression. It has been established that patients with ADPKD have higher levels of AVP as compared to healthy controls. Suppression, blockade or elimination of AVP slows cyst progression. AVP-V2 receptor inhibition controls disease progression in both animal models and humans, as does genetic elimination of vasopressin in the Polycystic Kidney (PCK) rat. This evidence indicates that AVP could be a promising target for therapeutic intervention. Unfortunately, the only clinically tested medication that blocks the AVP-V2 receptor (Tolvaptan) is associated with side effects including hypernatremia, hyperuricemia and elevated liver enzymes. An ideal therapeutic approach to target AVP in patients with ADPKD would be safe, easy to administer and could be adopted early in the disease process to prevent permanent kidney damage. High fluid intake presents one such possible treatment, and has been shown to suppress plasma levels of AVP, and slow cyst progression in an animal model of polycystic kidney disease. However, adherence to a high fluid intake diet is difficult to maintain in clinical practice.\n\nTo address this adherence challenge, The investigators have developed a stepwise approach of combining a low osmolar diet (low protein and salt) with adjusted water intake, with the goal of lowering the amount of water intake needed to suppress AVP secretion. The major objective of this proposal is to evaluate whether this intervention can suppress vasopressin secretion in patients with early ADPKD. Vasopressin suppression will be assessed by measuring copeptin levels, which have been shown to be a reliable surrogate marker for the circulating AVP concentration.\n\nThe rationale for this proposal is based on the fact that part of the difficulty in sustaining a low AVP level with daily water ingestion is the consumption of a diet that generates a large number of osmoles; high osmolar load stimulates vasopressin secretion to maintain water homeostasis. Hence, combining a low osmolar diet with adjusted water intake might prove to be sufficient to suppress vasopressin secretion in the clinical setting. The investigators propose the following:\n\nSpecific Aim: To conduct a randomized controlled trial to evaluate the effect of a low osmolar diet and high water intake intervention on vasopressin secretion, urine osmolality, and daily solute excretion in adult patients with ADPKD. The investigators hypothesize that a low osmolar diet combined with adjusted water intake will decrease serum copeptin level and total daily solute excretion in patients with ADPKD as compared to the control arm.\n\nTo accomplish the research goals, the current proposal builds upon existing expertise at Tufts Medical Center in conducting controlled clinical trials in patients with ADPKD.\n\nThe expected outcomes include the identification of a relevant, safe, easily tolerated and affordable intervention that can suppress vasopressin secretion in ADPKD patients early in the disease process; the proposed stepwise approach of combining a low osmolar diet and adjusted water intake carries the premise of lowering the amount of water needed to suppress AVP secretion and potentially slow the progression of this devastating disorder.\n\nThe study long-term goal is to evaluate whether this therapeutic approach could be tolerated by patients over a longer period of time, and could impact clinical outcome measures such as kidney volume and kidney function progression.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '60 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adults 18 to 60 years of age, who have ADPKD with an estimated glomerular filtration rate (eGFR) of 60 ml/min/1.73m2 or above\n\nExclusion Criteria:\n\n1. Patients on chronic use of medications known to affect AVP secretion (Serotonin Specific Reuptake inhibitors (SSRI), Opioids, Tricyclic Antidepressants (TCA) and Tolvaptan)\n2. History of diseases influencing renal concentration capacity, such as, diabetes insipidus, adrenal or thyroid deficiencies, present or prior use of lithium, or kidney diseases other than ADPKD.\n3. Baseline hyponatremia (Na below 135 mEq/l)\n4. Inability to comply with dietary or fluid requirements\n5. Have physical or cognitive impairments which prevent participation\n6. Pregnant women'}, 'identificationModule': {'nctId': 'NCT02225860', 'briefTitle': 'Diet as a Potential Treatment for Autosomal Dominant Polycystic Kidney Disease', 'organization': {'class': 'OTHER', 'fullName': 'Tufts Medical Center'}, 'officialTitle': 'Low Osmolar Diet and Adjusted Water Intake for Vasopressin Suppression in ADPKD', 'orgStudyIdInfo': {'id': '11111'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Diet and Water adjustment', 'description': 'Reduction in dietary salt and protein intake', 'interventionNames': ['Behavioral: Diet and water adjustment']}, {'type': 'NO_INTERVENTION', 'label': 'Control', 'description': 'Continue with usual diet'}], 'interventions': [{'name': 'Diet and water adjustment', 'type': 'BEHAVIORAL', 'description': 'The dietary intervention consisted of three elements: low sodium (1500 mg/day), low protein (daily protein dietary allowance of 0.8 gram/kg body weight), and low urea (avoidance of preservatives, food additives, bulking agents, and chewing gum). Protein was factored by measured body weight to mirror the estimated average requirement (EAR) of healthy adults which is set on a grams per kilogram basis', 'armGroupLabels': ['Diet and Water adjustment']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02111', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Tufts Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'overallOfficials': [{'name': 'Ronald Perrone, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Tufts Medical Center'}, {'name': 'Osama Amro, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Tufts Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Tufts Medical Center', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}