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

NCT ID: NCT04522180

Last Updated: 2024-10-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-01-04
• Study Completion Date: 2023-05-04

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-LRx monthly for 73 weeks. At the end of 73 weeks, participants entered a 14-week post-treatment (PT) evaluation period.

Conditions

Acromegaly

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

GHR-LRX 120 mg

Participants received GHR-LRX 120 mg subcutaneous (SC) injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).

Group Type: EXPERIMENTAL

GHR-LRX

Intervention Type: DRUG

GHR-LRX was administered by SC injection.

GHR-LRX 160 mg

Participants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).

Group Type: EXPERIMENTAL

GHR-LRX

Intervention Type: DRUG

GHR-LRX was administered by SC injection.

Interventions

GHR-LRX

GHR-LRX was administered by SC injection.

Intervention Type: DRUG

Other Intervention Names

ISIS 766720

Eligibility Criteria

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: 3 months
• pasireotide LAR: 4 months
• lanreotide (all formulations): 3 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 3 months before the trial, and/or planning to receive surgery during the trial

2. Participants who received radiotherapy for pituitary adenoma within the last 2 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Cedars-Sinai Medical Center

Los Angeles, California, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Palm Research Center Inc.

Las Vegas, Nevada, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

Endocrinology Associates, Inc

Columbus, Ohio, United States

Oregon Health & Science University (OHSU)

Portland, Oregon, United States

East-Tallinn Central Hospital

Tallinn, , Estonia

Tartu University Hospital

Tartu, , Estonia

Debreceni Egyetem Klinikai Kozpont

Debrecen, , Hungary

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, , Italy

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Roma, , Italy

Pauls Stradins Clinical University Hospital

Riga, , Latvia

Vaidoto Urbanaviciaus Individuali imone

Alytus, , Lithuania

Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos

Kaunas, , Lithuania

Zespol Oddzialow Chorob Wewnetrznych, Endokrynologii i Diabetologii

Warsaw, , Poland

Uniwersytecki Szpital Kliniczny, im. Jana Mikulicza-Radeckiego we Wroclawiu,

Wroclaw, , Poland

Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.

Wroclaw, , Poland

Centrul Medical Unirea Bucuresti, Endocrinologie

Bucharest, , Romania

I.M. Sechenov Moscow First State Medical University

Moscow, , Russia

Clinical Center of Serbia

Belgrade, , Serbia

Clinical Center of Vojvodina

Novi Sad, , Serbia

Countries

United States; Estonia; Hungary; Italy; Latvia; Lithuania; Poland; Romania; Russia; Serbia

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2020-000675-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NCT04522180

Identifier Type: OTHER

Identifier Source: secondary_id

ISIS 766720-CS5

Identifier Type: -

Identifier Source: org_study_id