Phase 2 Study of ALXN2420 versus Placebo in Combination with Somatostatin Analogs in Participants with Acromegaly

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Alexion Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

23 Oct 2025 → ongoing

Decision date (initial)

2025-09-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Alexion Pharmaceuticals, Inc

External identifiers

EU CT number

2025-521243-20-00

WHO UTN

U1111-1319-2934

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Dose response, Safety, Therapy, Pharmacokinetic

To evaluate the efficacy of 15 week treatment with ALXN2420 versus placebo for decreasing IGF-1 levels, when administered in combination with SSA therapy to adult participants with acromegaly

Secondary objectives 4

1. 1_To evaluate the efficacy of 15 week treatment with ALXN2420 versus placebo for decreasing IGF-1 levels, when administered in combination with SSA therapy to adult participants with acromegaly
2. 2_To evaluate the efficacy of 15 week treatment with ALXN2420 versus placebo for improving symptoms of acromegaly, when administered in combination with SSA therapy to adult participants with acromegaly
3. 3_To evaluate the effect of 15 week treatment with ALXN2420 versus placebo on HRQoL, when administered in combination with SSA therapy to adult participants with acromegaly
4. 4_To evaluate the effect of 15 week treatment with ALXN2420 versus placebo for improving global impression of severity and global impression of change, when administered in combination with SSA therapy to adult participants with acromegaly

Conditions and MedDRA coding

Acromegaly

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

Yes

IPD plan description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared. AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment https://vivli.org/. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1_Aged 18 to 80 years (inclusive) at Screening
2. 2_Documented diagnosis of acromegaly, that is, historically documented evidence of a GH secreting pituitary adenoma based on MRI or pathology report
3. 3_ Receiving maximum, or maximally tolerated dose per treating physician judgment, of long-acting SSAs and meet both of the following: - Received for ≥ 6 months prior to Screening - Receiving a once-monthly regimen (approximately every 4 weeks)
4. 4_partial responder to SSAs at Screening, defined as > 20% relative IGF-1 reduction during the course of SSA therapy
5. 5_Serum IGF-1 levels ≥ 1.3 to 5 × ULN inclusive, as assessed at a central laboratory and adjusted for age and sex, based on average of 2 consecutive values obtained during the Screening Period and obtained ≥ 7 days apart

Exclusion criteria 4

1. 1_Participants are excluded if they have any of the following medical conditions: 1. Had surgery for pituitary adenoma within the last 6 months before Day 1 or planning to receive surgery for pituitary adenoma during the study 2. Pituitary adenoma that, per Investigator’s judgement, is worsening as assessed by pituitary/sellar MRI or computed tomography scan obtained ≤ 6 months prior to Screening 3. Pituitary adenoma causing compression of the chiasm 4. Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment with dopamine agonists 5. Known hypothyroidism or hypocortisolism not adequately treated with a stable dose of thyroid or glucocorticoid hormone replacement therapy for ≥ 3 months prior to Screening 6. Active, clinically significant cardiac disease as judged by the Investigator 7. History of unstable angina, stroke, or acute myocardial infarction ≤ 3 months prior to Screening 8. Known uncontrolled Type 2 diabetes (HbA1c >10%) 9. Active malignant disease ≤ 2 years prior to Screening with exception of basal and squamous cell carcinoma of the skin
2. 2_Participants are excluded in case of Clinically significant renal or hepatic disease at the time of Screening, as judged by the Investigator or: - eGFR (CKD-EPI formula) < 30 mL/minute/1.73 m2 documented based on recent value (< 3 months prior to randomization) - Clinically significant abnormal values for hematology, biochemistry, coagulation, or urinalysis, as judged by the Investigator, including, but not limited to, total bilirubin > 1.5 × ULN (except if in free bilirubin linked to a known Gilbert Syndrome) or AST, ALT, or alkaline phosphatase > 2 × ULN
3. 3_Participants are excluded if received any of the following: - any type of fractionated radiotherapy or a second surgical adenectomy for pituitary adenoma within the last 3 years (5 years for conventional radiation) before starting treatment and/or are planning to receive radiotherapy or a second surgical adenectomy during the study - pegvisomant ≤ 8 weeks prior to Screening - dopamine agonists ≤ 4 weeks prior to Screening - pasireotide long-acting release ≤ 4 months prior to Screening
4. 4_Known allergy or sensitivity to ALXN2420 or any of the excipients

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percentage change from baseline in serum IGF-1 level at Week 15

Secondary endpoints 12

1. 1_Number of Participants Who Achieve Serum IGF-1 Level ≤1.3 Upper Limit of Normal (ULN) at Week 15
2. 2_Number of Participants Who Achieve Serum IGF-1 Level ≤1.0 ULN at Week 15
3. 3_Change From Baseline in Symptoms, as Assessed by disease specific questionnaire, at Week 15
4. 4_Change From Baseline in Serum IGF-1 Level at Week 15
5. 5_Change From Baseline in 36-item Short Form Survey (SF-36) Summary Scores and Subscores at Week 15
6. 6_Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) at Week 15
7. 7_Change from baseline in Acromegaly Quality of Life (AcroQoL) at Week 15
8. 8_Change From Baseline in Global Impression of Severity at Week 15 as Assessed by Patient Global Impression of Severity (PGIS) Scale
9. 9_Global Impression of Change at Week 15 as Assessed by Patient Global Impression of Change (PGIC) Scale
10. 10_Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Study Intervention Discontinuation or Interruption through Week 15
11. 11_Plasma Concentration of ALXN2420 over time through Week 15
12. 12_Number of Participants With Antidrug Antibodies (ADAs) through Week 15

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Auxiliary 6

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Alexion Pharmaceuticals Inc.

Sponsor organisation

Alexion Pharmaceuticals Inc.

Address

121 Seaport Boulevard

City

Boston

Postcode

02210-2050

Country

United States

Scientific contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial information

Public contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial information

Third parties 9

Locations

7 EU/EEA countries · 24 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 7 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 74 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications