Chronic HypoparathyroidismPhase 1/2NCT05239221
AZP-3601 SAD and MAD Study in Healthy Subjects and Patients With Hypoparathyroidism
A Phase 1/2 study testing AZP-3601 in people with Chronic Hypoparathyroidism, run by Alexion Pharmaceuticals, Inc. at 2 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Chronic Hypoparathyroidism
- What is being tested?
- AZP-3601
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 132 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 7 Sep 2020to 23 Aug 2022
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Main inclusion criteria
- 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.
Main exclusion criteria
- 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.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZP-3601, a Synthetic Parathyroid Hormone Analog, in Healthy Subjects and in Subjects With Hypoparathyroidism
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
- Being tested
- AZP-3601, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZP-3601Drug
Also called eneboparatide
Lyophilized powder of AZP-3601 to be reconstituted with water for injection before injection
PlaceboDrug
Saline solution visually matching active medication
| Study group | Role | What they receive |
|---|---|---|
| AZP-3601 | Experimental | subcutaneous (sc) administration once daily Receives: Drug: AZP-3601 |
| Placebo (Parts A and B) | Placebo comparator | subcutaneous (sc) administration once daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Emergent Adverse Events (TEAEs)
Number of Treatment Emergent Adverse Events (TEAEs), as assessed by medDRA (v25).
Measured: 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
Observed maximum concentration (Cmax) of AZP-3601 (pg/mL) in Part A
Measured: 24 hours
Observed Maximum Concentration (Cmax) - Part B
Observed maximum concentration (Cmax) of AZP-3601 (pg/mL) in Part B
Measured: Day 1, Day 14
Observed Maximum Concentration (Cmax) - Part C
Observed maximum concentration (Cmax) of AZP-3601 (pg/mL) in Part C.
Measured: Day 1, Day 14, Day 28, Day 84
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part A
Area under the plasma-drug concentration time curve (AUC) (pg*h/mL) in Part A
Measured: 24 hours
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part B
Area Under the Plasma-drug Concentration Time Curve (AUC) (pg*h/mL) in Part B
Measured: Day 1, Day 14
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part C
Area Under the Plasma-drug Concentration Time Curve (AUC) (pg*h/mL) in Part C.
Measured: Day 1, Day 14, Day 28, Day 84
Calcium Corrected for Albumin - Part A
Levels of calcium corrected for albumin (mg/dL) in Part A
Measured: 24 hours
Calcium Corrected for Albumin - Part B
Levels of calcium corrected for albumin (mg/dL) in Part B
Measured: 24 hours, Day 14
Calcium Corrected for Albumin - Part C
Levels of calcium corrected for albumin (mg/dL) in Part C.
Measured: Day 1, Day 14, Day 28 and Day 84
Serum Phosphate - Part A
Serum phosphate levels (mg/dL) in Part A
Measured: 24 hours
Serum Phosphate - Part B
Serum phosphate levels (mg/dL) in Part B
Measured: Day 1, Day 14
Serum Phosphate - Part C
Serum phosphate levels (mg/dL) in Part C.
Measured: Day 1 (H0), Day 14 (H2), Day 28 (H2) and Day 84 (H2)
Daily Dose of Oral Calcium and Active Vitamin D - Part C
Daily dose of oral calcium and active vitamin D for patients treated in Part C.
Measured: Day 28 and Day 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2022
- Study start7 Sep 2020
- Primary completion23 Aug 2022
- Study completion23 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic HypoparathyroidismStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 132 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amolyt Pharma
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05239221
- Sponsor's protocol ID
- AZP-3601-CLI-001
- Status last verified
- Sep 2025
- First submitted
- 7 Jan 2022
- Last updated
- 8 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40423237
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZP-3601, a Synthetic Parathyroid Hormone Analog, in Healthy Subjects and in Subjects With Hypoparathyroidism
- ClinicalTrials.gov ID
- NCT05239221
- Acronym / sponsor ID
- AZP-3601-CLI-001
- Conditions
- Chronic Hypoparathyroidism
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 132 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Amolyt Pharma
- Study start
- 7 Sep 2020 (actual)
- Primary completion
- 23 Aug 2022 (actual)
- Study completion
- 23 Aug 2022 (actual)
- First posted
- 14 Feb 2022 (actual)
- Results
- Posted 31 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AZP-3601 | Experimental | Drug: AZP-3601subcutaneous (sc) administration once daily |
| Placebo (Parts A and B) | Placebo comparator | Drug: Placebosubcutaneous (sc) administration once daily |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AZP-3601Also: eneboparatideLyophilized powder of AZP-3601 to be reconstituted with water for injection before injection | Drug | AZP-3601 |
| PlaceboSaline solution visually matching active medication | Drug | Placebo (Parts A and B) |
Primary outcome
Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 2 weeks in Part A and Part B, and up to 3 months in Part C
Number of Treatment Emergent Adverse Events (TEAEs), as assessed by medDRA (v25).
13 secondary outcomes
Observed Maximum Concentration (Cmax) - Part A
Time frame: 24 hours
Observed Maximum Concentration (Cmax) - Part B
Time frame: Day 1, Day 14
Observed Maximum Concentration (Cmax) - Part C
Time frame: Day 1, Day 14, Day 28, Day 84
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part A
Time frame: 24 hours
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part B
Time frame: Day 1, Day 14
Area Under the Plasma-drug Concentration Time Curve (AUC) - Part C
Time frame: Day 1, Day 14, Day 28, Day 84
Calcium Corrected for Albumin - Part A
Time frame: 24 hours
Calcium Corrected for Albumin - Part B
Time frame: 24 hours, Day 14
Calcium Corrected for Albumin - Part C
Time frame: Day 1, Day 14, Day 28 and Day 84
Serum Phosphate - Part A
Time frame: 24 hours
Serum Phosphate - Part B
Time frame: Day 1, Day 14
Serum Phosphate - Part C
Time frame: Day 1 (H0), Day 14 (H2), Day 28 (H2) and Day 84 (H2)
Daily Dose of Oral Calcium and Active Vitamin D - Part C
Time frame: Day 28 and Day 43
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40423237Ovize 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AZP-3601 in people with Chronic Hypoparathyroidism, run by Alexion Pharmaceuticals, Inc. at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Hungary, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Emergent Adverse Events (TEAEs) (time frame: Up to 2 weeks in Part A and Part B, and up to 3 months in Part C). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05239221, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2025.