Acromegaly| IONIS-GHR-LRx Dose 1
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered 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.
Purpose of Study
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.
Age:18 - 75
Key Inclusion / Exclusion Criteria
- Males or females with a documented diagnosis of Acromegaly* who are 18 to 75 years old (inclusive) at the time of informed consent.
- 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: 3 months
- pasireotide LAR: 4 months
- lanreotide (all formulations): 3 months
- At Screening, serum IGF-1 (performed at the central lab) between 1.3 to 5 × ULN, inclusive, adjusted for age and sex.
- Females must be non-pregnant and non-lactating, and either surgically sterile, post-menopausal, abstinent, or using 1 highly effective method of birth control
- Participants who received surgery for pituitary adenoma within the last 3 months before the trial, and/or planning to receive surgery during the trial
- Participants who received radiotherapy for pituitary adenoma within the last 2 years before the trial, and/or planning to receive radiotherapy during the trial
- 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
- Evidence of decompensated cardiac function per medical judgement and/or New York Heart Association (NYHA) Class 3 or 4
- Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment
- Symptomatic cholelithiasis, and/or choledocholithiasis
Clinical Trials ID
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Start Date / End Date:
01 2021 - 08 2023
IONIS-GHR-LRx Dose 1
19 Study Locations
Fondazione Policlinico Universitario A. Gemelli IRCCS – Universita Cattolica del Sacro Cuore