Status:

COMPLETED

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

Lead Sponsor:

Ionis Pharmaceuticals, Inc.

Conditions:

Acromegaly

Eligibility:

All Genders

18-75 years

Phase:

PHASE2

Brief 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.

Detailed Description

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-L...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

January 4 2021

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

May 4 2023

Estimated Enrollment :

34 Patients enrolled

Trial Details

Trial ID

NCT04522180

Start Date

January 4 2021

End Date

May 4 2023

Last Update

October 3 2024

Active Locations (22)

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Page 1 of 6 (22 locations)

1

Cedars-Sinai Medical Center

Los Angeles, California, United States, 90048

2

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

3

Palm Research Center Inc.

Las Vegas, Nevada, United States, 89148

4

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

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