A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
The purpose of this study is to determine the safety, tolerability, and efficacy of
 IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.
This is a multi-center, open-label, randomized, Phase 2 study of IONIS-GHR-LRx in up to 40
 participants with acromegaly. Participants will be randomized to 1 of 3 treatment groups to
 receive a single dose of IONIS GHR-LRx monthly for 73 weeks. At the end of 73 weeks,
 participants will enter a 14-week post-treatment (PT) evaluation period.
Inclusion Criteria:

 1. Males or females with a documented diagnosis of Acromegaly* who are 18 to 75 years old
 (inclusive) at the time of informed consent.

 2. Have had pituitary surgery (e.g. transsphenoidal) unless there was a contraindication
 to surgery and are either acromegaly medical treatment naïve, or who had not taken any
 other acromegaly medications prior to the screening visit as outlined below

 -  bromocriptine: 2 weeks

 -  cabergoline: 4 weeks

 -  quinagolide: 4 weeks

 -  octreotide daily injection (SC) or oral formulation: 4 weeks

 -  pegvisomant: 4 weeks

 -  octreotide LAR: 4 months

 -  pasireotide LAR: 4 months

 -  lanreotide (all formulations): 4 months

 3. At Screening, serum IGF-1 (performed at the central lab) between 1.3 to 5 × ULN,
 inclusive, adjusted for age and sex.

 4. Females must be non-pregnant and non-lactating, and either surgically sterile,
 post-menopausal, abstinent, or using 1 highly effective method of birth control

 Exclusion Criteria:

 1. Participants who received surgery for pituitary adenoma within the last 6 months
 before the trial, and/or planning to receive surgery during the trial

 2. Participants who received radiotherapy for pituitary adenoma within the last 3 years
 before the trial, and/or planning to receive radiotherapy during the trial

 3. Participants with a pituitary tumor that, per Investigator judgment, is worsening
 (e.g., either growing or at risk of compressing or abutting the optic chiasm or other
 vital structures) as assessed by pituitary/sellar MRI protocol at Screening or within
 3 months of Screening. CT scan is allowed if MRI is contraindicated

 4. Evidence of decompensated cardiac function per medical judgement and/or New York Heart
 Association (NYHA) Class 3 or 4

 5. Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment

 6. Symptomatic cholelithiasis, and/or choledocholithiasis