Trial Outcomes & Findings for Medical Safety Follow-up Study for Patients Who Received More Than 28 Days of Total Exposure to BGG492 (NCT NCT02150213)

Results Overview

Incidence of adrenal cortical adenomas as assessed by non-contrast MRI of the abdomen (CT or ultrasound of the abdomen was permitted if MRI was contraindication)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

59 participants

Primary outcome timeframe

Minimum of one year after last dose of BGG492 in study BGG492A2207 or BGG492A2212

Results posted on

2016-12-05

Participant Flow

The purpose of this follow-up safety study was to provide medical follow-up to patients exposed to BGG492 for more than 28 days in Study BGG492A2207 and/or BGG492A2212.

The first period focused on contacting the patients, obtaining consent for follow-up, and conducting screening assessments. The second period of the study focused on performing the follow-up procedures, obtaining and interpreting the results, and referring the patient, if necessary, to an appropriate medical specialist.

Participant milestones

Participant milestones
Measure	BGG492 This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Overall Study STARTED	59
Overall Study COMPLETED	57
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	BGG492 This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Overall Study Lost to Follow-up	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Medical Safety Follow-up Study for Patients Who Received More Than 28 Days of Total Exposure to BGG492

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BGG492 n=59 Participants This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Age, Continuous	43.2 Years STANDARD_DEVIATION 11.58 • n=5 Participants
Gender Female	31 Participants n=5 Participants
Gender Male	28 Participants n=5 Participants

PRIMARY outcome

Timeframe: Minimum of one year after last dose of BGG492 in study BGG492A2207 or BGG492A2212

Population: Full analysis Set: included all patients who signed informed consent to enter the study

Incidence of adrenal cortical adenomas as assessed by non-contrast MRI of the abdomen (CT or ultrasound of the abdomen was permitted if MRI was contraindication)

Outcome measures

Outcome measures
Measure	BGG492 n=59 Participants This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Incidence of Adrenal Cortical Adenomas	3 Particpants

PRIMARY outcome

Timeframe: Minimum of one year after last dose of BGG492 in study BGG492A2207 or BGG492A2212

Population: Full Analysis Set (FAS):included all patients who signed informed consent to enter the study, but this assessment was only done on female patients. Of the 31 female patients, two had a hysterectomy and were not evaluated (N=29)

Incidence of uterine endometrial stromal sarcomas as assessed by sonogram/biopsy (females)

Outcome measures

Outcome measures
Measure	BGG492 n=29 Participants This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Incidence of Uterine Endometrial Stromal Sarcomas	0 Participants

Adverse Events

BGG492

Serious events: 5 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BGG492 n=59 participants at risk This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adrenal Adenoma	5.1% 3/59
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Angiomyolipoma	1.7% 1/59
Reproductive system and breast disorders Ovarian cyst	1.7% 1/59

Other adverse events

Other adverse events
Measure	BGG492 n=59 participants at risk This was a follow-up safety study where study treatment was not administered. Patients came from BGG492 studies where patients were previously exposed to \> 28 days of BGG492 50 mg, 100 mg or 150 mg given orally three times a day
Renal and urinary disorders Renal Cyst	5.1% 3/59

Additional Information

Study Director

Novartis

Phone: 862-778-8300

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER