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GHR-LRX

Phase 2

Acromegaly | Small molecule | Endocrine |Ionis Pharmaceuticals, Inc.|Last Updated: Oct 3, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment34

FDA Designations

No designations recorded

Clinical trial landscape

GHR-LRX · 1 trial · 1 indication

Phase 2 1
NCT04522180A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyAcromegaly
COMPLETED34 Analytics
PHASE2COMPLETED
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
AcromegalyUnlock trial analytics

Study Endpoints

Primary Endpoints

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

Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27)
At Week 27
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27)
At Week 27
Change From Baseline in Serum IGF-1 Over Time
Up to approximately 80 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GHR-LRX 120 mgEXPERIMENTALParticipants received GHR-LRX 120 mg subcutaneous (SC) injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
GHR-LRX 160 mgEXPERIMENTALParticipants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).

Interventions

NameTypeDescription
GHR-LRXDRUGGHR-LRX was administered by SC injection.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites22

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...

Countries:United StatesEstoniaHungaryItalyLatviaLithuaniaPolandRomaniaRussiaSerbia
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Frequently asked questions about GHR-LRX

What is GHR-LRx used for in acromegaly?

GHR-LRx is an investigational drug being studied for the treatment of acromegaly, a condition caused by excess growth hormone. It is designed to reduce growth hormone receptor activity, potentially lowering hormone levels and alleviating symptoms. The drug is currently in clinical development and has not been approved for use.

What does GHR-LRx target?

GHR-LRx targets the growth hormone receptor. By inhibiting this receptor, the drug aims to block the effects of excess growth hormone in patients with acromegaly. This mechanism is intended to help manage the hormonal imbalance characteristic of the condition.

Who makes GHR-LRx?

GHR-LRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for acromegaly.

What phase is GHR-LRx in?

GHR-LRx is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The Phase 2 trial has been completed, and the drug is being evaluated for safety, tolerability, and efficacy in patients with acromegaly.

What clinical trials is GHR-LRx in?

GHR-LRx has been studied in a Phase 2 clinical trial with the identifier NCT04522180. This completed trial assessed the safety, tolerability, and efficacy of the drug in 34 patients with acromegaly. The study was conducted across multiple countries, including the United States and several European nations.

Is GHR-LRx the same as IONIS-GHR-LRx?

Yes, GHR-LRx is also known as IONIS-GHR-LRx. The clinical trial NCT04522180 used the name IONIS-GHR-LRx in its title. Both names refer to the same investigational drug being developed by Ionis Pharmaceuticals for the treatment of acromegaly.