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Completed

NCT Number: NCT04522180

A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly

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.

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Key information

Conditions

Acromegaly Bone Diseases Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Endocrine System Diseases Hyperpituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pituitary Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

East-Tallinn Central Hospital, Tallinn, Estonia

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About this study

This was a multi-center, open-label, randomized, Phase 2 study of IONIS-GHR-LRx in up to 40 participants with acromegaly. Participants were randomized to 1 of 2 treatment groups to receive IONIS GHR-LRx monthly for 73 weeks. At the end of 73 weeks, participants entered a 14-week post-treatment (PT) evaluation period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

GHR-LRX

Drug

GHR-LRX was administered by SC injection.

Other names: ISIS 766720

Primary outcomes

  1. Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27

    Time frame: Baseline to Week 27

    IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. Baseline of IGF-1 is defined as the average value of Screening and Day 1. A negative percent change from Baseline indicated improvement.

Secondary outcomes

  1. Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27)

    Time frame: At Week 27

    Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's upper limit of normal (ULN).

  2. Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27)

    Time frame: At Week 27

    Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's ULN.

  3. Change From Baseline in Serum IGF-1 Over Time

    Time frame: Up to approximately 80 weeks

    IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative change from baseline indicated improvement.

  4. Percent Change From Baseline in Serum IGF-1 Over Time

    Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 21, 25, 27, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73

    IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative percent change from Baseline indicated improvement.

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Registry information

Official study title

An Open Label, Randomized, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor, Administered Monthly as Monotherapy in Patients With Acromegaly

Important dates

Study start
2021
Primary completion
2022
Study completion
2023
First posted
Aug 21, 2020
Registry last updated
Oct 3, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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