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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04522180
Recruitment Status : Active, not recruiting
First Posted : August 21, 2020
Last Update Posted : January 10, 2022
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.

Condition or disease Intervention/treatment Phase
Acromegaly Drug: IONIS-GHR-LRx Phase 2

Detailed Description:
This is a multi-center, open-label, randomized, Phase 2 study of IONIS-GHR-LRx in up to 40 participants with acromegaly. Participants will be randomized to 1 of 3 treatment groups to receive a single dose of IONIS GHR-LRx monthly for 73 weeks. At the end of 73 weeks, participants will enter a 14-week post-treatment (PT) evaluation period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : January 4, 2021
Estimated Primary Completion Date : August 2023
Estimated Study Completion Date : August 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: IONIS-GHR-LRx Dose 1
Single dose of IONIS-GHR-LRx Dose 1 will be administered by SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
Drug: IONIS-GHR-LRx
IONIS-GHR-LRx will be administered by SC injection.

Experimental: IONIS-GHR-LRx Dose 2
Single dose of IONIS-GHR-LRx Dose 2 will be administered by SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
Drug: IONIS-GHR-LRx
IONIS-GHR-LRx will be administered by SC injection.

Experimental: IONIS-GHR-LRx Dose 3
Single dose of IONIS-GHR-LRx Dose 3 will be administered by SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
Drug: IONIS-GHR-LRx
IONIS-GHR-LRx will be administered by SC injection.




Primary Outcome Measures :
  1. Percent Change From Baseline in Insulin-like Growth Factor I (IGF-1) to Week 27 [ Time Frame: Up to Week 27 ]

Secondary Outcome Measures :
  1. Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.2 Times of Gender and Age Limits at Day 183 (Week 27) [ Time Frame: At Week 27 ]
  2. Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.0 Times of Gender and Age Limits at Day 183 (Week 27) [ Time Frame: At Week 27 ]
  3. Change From Baseline in Serum IGF-1 Over Time [ Time Frame: Up to approximately 80 weeks ]
  4. Percent Change From Baseline in Serum IGF-1 Over Time [ Time Frame: Up to approximately 80 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04522180


Locations
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Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04522180    
Other Study ID Numbers: ISIS 766720-CS5
2020-000675-20 ( EudraCT Number )
First Posted: August 21, 2020    Key Record Dates
Last Update Posted: January 10, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ionis Pharmaceuticals, Inc.:
acromegaly
IONIS-GHR-LRx
Additional relevant MeSH terms:
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Acromegaly
Bone Diseases, Endocrine
Bone Diseases
Musculoskeletal Diseases
Hyperpituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Endocrine System Diseases