Phase 2
N=34
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
Acromegaly
Bottom Line
View on ClinicalTrials.gov: NCT04522180 ↗Enrolled (actual)
34
Serious AEs
8.8%
Results posted
Oct 2024
Primary outcome: Primary: Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27 — -6.20; -7.16; -7.09 Percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- GHR-LRX (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27 |
-6.20; -7.16; -7.09 | — |
| SECONDARY Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27) |
0; 0; 0 | — |
| SECONDARY Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27) |
0; 0; 0 | — |
| SECONDARY Change From Baseline in Serum IGF-1 Over Time |
-30.00; 5.03; 1.74; -50.67; -45.53; -46.01 | — |
| SECONDARY Percent Change From Baseline in Serum IGF-1 Over Time |
-3.73; 2.64; 2.04; -5.67; -8.28; -8.04 | — |
Summary
The purpose of this study was to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.
Eligibility Criteria
Inclusion 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT04522180). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.