A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH

Brief Title

A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH

Official Title

A Phase III Extension Study of Efficacy, Safety and Tolerability of Chronocort® in the Treatment of Congenital Adrenal Hyperplasia

Brief Summary

      Subjects completing study DIUR-005 and those who have already completed study DIUR-003 will
      be offered the opportunity either to continue Chronocort® therapy or to switch from their
      current glucocorticoid therapy to Chronocort® in this open-label study.
    

Detailed Description

      All subjects will have a screening visit prior to the baseline assessment to allow DIUR-006
      procedures to be fully explained and informed consent to be given by the subject. For
      subjects from DIUR-003 this screening visit will include safety blood tests. Any subjects not
      meeting the inclusion/exclusion criteria following these blood tests will be not be entered
      into the study.

      All subjects will then return for the baseline visit. For subjects entering from study
      DIUR-003 the full set of baseline assessments will be completed, including 2 blood samples
      (one at 09:00 and one at 13:00 hours) for 17-OHP and A4. For subjects entering from DIUR-005,
      test results from their last visit in the feeder study (Visit 4) will be used for this
      baseline assessment, with the 09:00 and 13:00 hour results taken from the 24-hour hormone
      profiles conducted at the visit. Any subjects not meeting the inclusion/exclusion criteria
      following these blood tests will be withdrawn from this study.

      Once the baseline assessments are completed, the subjects will be given sufficient
      Chronocort® to use until the next visit at Week 4. Subjects from study DIUR-005 who were
      previously on Chronocort® will continue on the same dose of Chronocort® that they were
      receiving at the end of the feeder study. Subjects from study DIUR-005 on standard therapy
      and subjects from study DIUR-003 will have their initial dose of Chronocort® determined using
      the hydrocortisone equivalent of baseline therapy.

      All subjects will return to the study centre at 4, 12 and 24 weeks after starting study
      DIUR-006 for additional blood tests and dose titration, if necessary. Visits thereafter will
      take place at 6-monthly intervals. If there is a change of dose, an interim visit or phone
      call will be needed inbetween the 6-monthly visits.

      All subjects will receive telephone calls at 3 monthly intervals, and unscheduled visits will
      be arranged if necessary. Subjects will also be provided with Chronocort® supplies from the
      study pharmacy at 3-monthly or 6-monthly intervals.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Safety and tolerability of Chronocort over time, as assessed by signs and symptoms of adrenal insufficiency.

Secondary Outcome

 Long-term efficacy of Chronocort, as assessed by total daily dose of Chronocort in mg/day of hydrocortisone and by Body Surface Area (BSA).

Condition

Congenital Adrenal Hyperplasia

Intervention

Hydrocortisone

Study Arms / Comparison Groups

 Chronocort®
Description:  Chronocort® modified release hydrocortisone

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

92

Start Date

August 18, 2016

Completion Date

March 2022

Primary Completion Date

March 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Subjects with CAH who have successfully completed a clinical trial with the current
             formulation of Chronocort®.

          2. Provision of signed written informed consent.

        Exclusion Criteria:

          1. Co-morbid condition requiring daily administration of a medication (or use of any
             medications/supplements) that interferes with the metabolism of glucocorticoids.

          2. Clinical or biochemical evidence of hepatic or renal disease. Creatinine over twice
             the ULN or elevated liver function tests (ALT or AST >2 times ULN]).

          3. Females who are pregnant or lactating.

          4. Subjects on regular daily inhaled, topical, nasal or oral steroids for any indication
             other than CAH.

          5. History of malignancy (other than basal cell carcinoma successfully treated >6 months
             prior to entry into the study).

          6. Subjects with a history of bilateral adrenalectomy.

          7. Participation in another clinical trial of an investigational or licensed drug or
             device within the 3 months prior to inclusion in this study, except for another
             clinical trial with the current formulation of Chronocort®.

          8. Subjects unable to comply with the requirements of the protocol.

          9. Subjects who routinely work night shifts and so do not sleep during the usual
             nighttime hours.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Debbie Merke, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03062280

Organization ID

DIUR-006


Responsible Party

Sponsor

Study Sponsor

Diurnal Limited


Study Sponsor

Debbie Merke, MD, Principal Investigator, National Institutes of Health (NIH)


Verification Date

October 2020