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Clinical Trials/NCT05044611
NCT05044611
Active, not recruiting
Phase 4

AMIloride for the Treatment of Nephrogenic Diabetes Insipidus for Patients With Bipolar Disorder Treated With Lithium: a Randomized Controlled Trial

Assistance Publique - Hôpitaux de Paris2 sites in 1 country148 target enrollmentStarted: January 11, 2023Last updated:

Overview

Phase
Phase 4
Status
Active, not recruiting
Enrollment
148
Locations
2
Primary Endpoint
The main objective of this study is demonstrating the efficacy of amiloride to reduce the urine concentration defect in patients treated by lithium and presenting a nephrogenic diabetes insipidus after 2 months of treatment.

Overview

Brief Summary

Lithium (Li) is the leading treatment for BD, protecting against both maniac and depressive relapse, and reducing the risk of suicide and mortality. However, despite this major clinical efficacy, the use of lithium is limited by its narrow therapeutic index and by its side effects. Li induces a vasopressin-resistant urinary concentration defect, with resulting nephrogenic diabetes insipidus (NDI) in 12-50 % of patients. This feature is more frequent after 5 years of treatment with lithium. Polyuria and subsequent thirst might affect patients' quality of life, but also cause potentially life-threatening hypernatremia if free access to water is impaired. Thus, we aim at evaluating the efficacy of amiloride on urine concentrating ability in patients with nephrogenic diabetes insipidus due to chronic lithium treatment.

Detailed Description

Patients will be referred to the nephrology or the renal physiology department for the usual follow-up of the lithium treatment. After verification of eligilibity criteria, information and collection of consent, patient will be randomized.

During the first phase, patients will be randomized in two parallel groups: the experimental arm will receive 5mg of amiloride twice daily during 2 months and the control arm will receive a placebo twice daily during 2 months.

Measures of fasting urine osmolality will be performed at baseline, 2 months, at 6 months and at 12 months, in order to compare the difference of urine osmolality before and after treatment between the two-randomization arms. Other baseline explorations are as follows: mean number of nocturnal voids, SF-36 questionnaire, thirst intensity and distress scales, YMRS/MADRS mood scale, GAD7 anxiety scale, PSQI sleep scale, GFR measurement and estimation, 24h urine for the quantification of the polyuria and osmolality, plasma and erythrocyte lithium level, serum osmolality, natremia, kaliemia, urea, chlore level, complete blood count, plasma copeptine and vasopressin.

A nephrologist visit will take place 15 days after the initiation of the treatment along with a new measure of plasma lithium level.

Patients will be evaluated at 1 month only if a change in posology is required after the first measurement at day 15 and then at 2, 6 and 12 months.

In parallel, patients will be evaluated by at the psychiatry clinic at 1 month, 2, 6 and 12 months, and in any condition requiring additional visit as usual in standard care (follow-up of anxiety, sleepiness, suicidal ideation, depression).

After the completion of this first phase, the open label second phase will begin. Unblinding the trial will allow the treatment allocation being available for the participants and health care professionals. Amiloride will be continued in participants in the experimental group, and the remaining participants will be followed-up without treatment. This phase will last for 10 months (total trial duration: 12 months).

At one year, renal functions (GFR, urine concentration and 24h urine production) will be assessed along with report of events including hospital admission.

The safety of the experimental treatment will be assessed by regular evaluations of plasma lithium and potassium level, beginning at 2 weeks after treatment initiation and after 2 months. The main risk of amiloride is hyperkalemia, which occurs in patients with severe renal insufficiency. These patients will not be included in our study. Otherwise, the treatment is generally safe and well-tolerated. Plasma lithium level will be measured at the first month clinical evaluation if a change in posology is required after the first measurement at day 15.

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Eligibility Criteria

Ages
18 Years to 70 Years (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Adults between 18 and 70 years (age ≥ 18 years and \<70 years)
  • Patient with bipolar disorder
  • Patient treated with lithium for at least 5 years
  • Patient with a urine concentration defect defined by a maximal urine osmolality \< 600 mOsm/kg
  • Woman of childbearing age agreeing to use an efficient contraceptive method for 12 months

Exclusion Criteria

  • Renal failure defined as eGFR \< 30 ml/min/1.73m² estimated by the CKD-EPI equation
  • Kalemia \> 5 mmol/l
  • Hypersensitivity or known allergy to amiloride
  • Hypersensitivity to lactose
  • Known adrenal insufficiency
  • Concomitant use of other potassium-sparing treatment (e.g. spironolactone, angiotensin converting enzyme inhibitors (ACE), angiotensin II receptor (AT2R) antagonists, calcineurin inhibitors tacrolimus and ciclosporin)
  • Acute ongoing infection (less than 3 days before inclusion)
  • Severe heart failure (NYHA \> II)
  • Rhythm, conduction or repolarisation disorder present on an ECG done within 12 months prior to inclusion
  • Acute phase of mood disorder

Arms & Interventions

Amiloride

Experimental

the experimental arm will receive 5mg of amiloride twice daily during 2 months

Intervention: Anhydrous Amiloride Hydrochloride (Drug)

Placebo

Placebo Comparator

the control arm will receive a placebo twice daily during 2 months

Intervention: Placebo (Drug)

Outcomes

Primary Outcomes

The main objective of this study is demonstrating the efficacy of amiloride to reduce the urine concentration defect in patients treated by lithium and presenting a nephrogenic diabetes insipidus after 2 months of treatment.

Time Frame: 2 month after randomization

The primary endpoint is the percentage change in maximal urine osmolality before and after 2 months of treatment

Secondary Outcomes

  • Demonstrate the efficacy of amiloride to reduce the sensation of thirst(2 months after randomization and 12 months after randomization)
  • Demonstrate the efficacy of amiloride to reduce polyuria(2 months after randomization and 12 months after randomization)
  • Evaluate the effect of amiloride in mood stability(2 months after randomization and 12 months after randomization)
  • Demonstrate the efficacy of amiloride to reduce nocturia(2 months after randomization and 12 months after randomization)
  • Demonstrate the efficacy of amiloride to increase quality of life(2 months after randomization and 12 months after randomization)
  • Evaluate the effect of amiloride in circulating lithium levels stability(2 months after randomization)
  • Demonstrate the efficacy of amiloride to reduce the decline of eGFR after one year of treatment(12 months after randomization)

Investigators

Sponsor Class
Other
Responsible Party
Sponsor

Study Sites (2)

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