AZP-3601 SAD and MAD Study in Healthy Subjects and Patients With Hypoparathyroidism

NCT05239221 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: AZP-3601-CLI-001
• Study Type: interventional
• Target: 132
• Locations: 2 countries
• Sites: 2

Brief Summary

This study is investigating the safety, tolerability, pharmacodynamics and pharmacokinetics of AZP-3601 following single and repeated administration in both healthy volunteers and patients with chronic hypoparathyroidism (cHP) The protocol includes 3 parts:

• Part A: first-in-human single ascending dose (SAD) study in healthy volunteers
• Part B: multiple ascending dose (MAD) study with 2 weeks of treatment in healthy volunteers
• Part C: open-label MAD study with a total treatment duration of 3 months in patients with cHP.

Conditions

Chronic Hypoparathyroidism

Outcome Measures

Primary Outcomes (1)

• Treatment Emergent Adverse Events (TEAEs)

  Number of Treatment Emergent Adverse Events (TEAEs), as assessed by medDRA (v25).

  Up to 2 weeks in Part A and Part B, and up to 3 months in Part C

Secondary Outcomes (13)

• Observed Maximum Concentration (Cmax) - Part A

  24 hours

• Observed Maximum Concentration (Cmax) - Part B

  Day 1, Day 14

• Observed Maximum Concentration (Cmax) - Part C

  Day 1, Day 14, Day 28, Day 84

• Area Under the Plasma-drug Concentration Time Curve (AUC) - Part A

  24 hours

• Area Under the Plasma-drug Concentration Time Curve (AUC) - Part B

  Day 1, Day 14

Other Outcomes (1)

• Calcium Excretion Rate 24h- Part C

  Day 1, Day 14, Day 28 and Day 84

Study Arms (2)

AZP-3601

EXPERIMENTAL

subcutaneous (sc) administration once daily

Drug: AZP-3601

Placebo (Parts A and B)

PLACEBO COMPARATOR

subcutaneous (sc) administration once daily

Drug: Placebo

Interventions

AZP-3601 DRUG

Lyophilized powder of AZP-3601 to be reconstituted with water for injection before injection

Also known as: eneboparatide

AZP-3601

Placebo DRUG

Saline solution visually matching active medication

Placebo (Parts A and B)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Part A: healthy male volunteers aged 18 to 60 years old inclusive with a body mass index of 19 to 28 kg/m2
• Part B: healthy male and female volunteers (non-child bearing potential) aged 18 to 60 years inclusive with a Body mass index of 19 to 28 kg/m2
• Part C:
• Male and female patients aged 18 to 75 years inclusive
• History of cHP for ≥12 months at the time of screening with documentation of two measurements of serum calcium and parathyroid hormone (PTH).
• Requirement for therapy with calcitriol ≥0.25 μg per day or alphacalcidol ≥0.50 μg per day (both are active vitamin D supplements), and requirement for supplemental oral calcium treatment ≥1000 mg per day over and above normal dietary calcium intake at baseline assessments.

You may not qualify if:

• Parts A and B:
• Clinically significant abnormal lab values, as judged by the investigator
• Using tobacco products with 3 months prior to first drug administration
• History of alcohol abuse or drug addiction
• Part C:
• Known history of autosomal-dominant hypocalcemia (ADH resulting from gain-of-function calcium-sensing receptor \[CaSR\] or GNA11 mutations) or pseudohypoparathyroidism (impaired responsiveness to PTH)
• Any current disease that might affect calcium metabolism or calcium phosphate homeostasis other than HP
• Use of medications such as loop and thiazide diuretics, raloxifene hydrochloride, lithium, methotrexate, cardiac glycosides (e.g., digoxin or digitoxin) or systemic corticosteroids within 4 weeks prior to start of treatment.
• Previous treatment with PTH-like drugs, including PTH(1-84), PTH(1-34), or abaloparatide, within 3 months prior to screening.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Amolyt Pharma: collaborator

Study Sites (2)

Amolyt Pharma Investigational Site Hungary

Budapest, Hungary

Location

PRA-EDS

Groningen, 9728, Netherlands

Location

Related Publications (1)

• Ovize M, Allas S, Culler MD, Milano S, Ouldrouis T, Sumeray M, van de Wetering de Rooij J, Mannstadt M. Phase 1 clinical trial of eneboparatide, a novel PTH receptor 1 agonist. Endocr Connect. 2025 Jun 19;14(6):e240464. doi: 10.1530/EC-24-0464. Print 2025 Jun 1.

  PMID: 40423237 DERIVED

Results Point of Contact

• Title: Senior VP of Clinical Development and Regulatory Affairs
• Organization: Amolyt Pharma

contact@amolyt.com

+33 428 01 21 54

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part C is open label
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2022
• First Posted: February 14, 2022
• Study Start: September 7, 2020
• Primary Completion: August 23, 2022
• Study Completion: August 23, 2022
• Last Updated: September 8, 2025
• Results First Posted: January 31, 2024

Record last verified: 2025-09

Locations

HU (1); NL (1)