Autosomal Dominant Polycystic KidneyPhase 1NCT04908462

To Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AL01211 in Healthy Volunteers

A Phase 1 study of Autosomal Dominant Polycystic Kidney, run by AceLink Therapeutics, Inc. at 1 site in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Oral AL01211 in healthy volunteers Read more about this study

Who is it for?: Ages 18 to 55 yearsHealthy volunteers can take part
Conditions the study is for: Autosomal Dominant Polycystic Kidney
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 69 participants
Where: 1 siteAustralia
Study dates: 8 Jun 2021to 20 Jun 2022
Phase: Phase 1
Are there results?: None posted in the registry

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 55 years
All sexes
Healthy volunteers accepted
Adult

Who may be able to join

For Part A (SAD) and Part B (MAD)
To be eligible for the study, participants must meet all of the following inclusion criteria:
Healthy male or female volunteers, between 18 and 55 years of age
Participants in good health as determined by medical history, physical examination, vital signs, ECG, and clinical laboratory tests.
Body Mass Index (BMI) between 20.0 and 34.9 kg/m2 (inclusive).
Participants who smoke no more than 2 cigarettes per day or equivalent per week (includes e-cigarettes) can be included in the study but must be willing to abstain from smoking during confinement periods.

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Participants must have no relevant dietary restrictions,
Females must be non-pregnant and non-lactating, and must use an acceptable, highly effective double contraception from Screening until at least 30 days have passed since study drug administration , including the follow-up period. Double contraception is defined as a condom AND one other form of the following:
Established hormonal contraception (oral contraceptive pills [OCPs], long-acting implantable hormones, and injectable hormones) for at least 1 month prior to Screening
A vaginal ring or an intrauterine device [IUD]
Documented evidence of surgical sterilization at least 6 months prior to Screening (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) for women or vasectomy at least 90 days prior to Screening for men (with appropriate post-vasectomy documentation of the absence of sperm in semen), provided the male partner is a sole partner.
Women not of childbearing potential must be postmenopausal for ≥ 12 months. Postmenopausal status will be confirmed through testing of follicle-stimulating hormone (FSH) levels ≥ 40 IU/mL at Screening for amenorrhoeic female participants. Females who are abstinent from heterosexual intercourse will also be eligible.
Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not considered highly effective methods of birth control. Participant complete abstinence for the duration of the study and for 1 month after the last study treatment is acceptable.
Female participants who exclusively are in same sex relationships are not required to use contraception.
Males must be surgically sterile (> 90 days since vasectomy with no viable sperm), abstinent, or if engaged in sexual relations with a woman of childbearing potential (WOCBP), the participant and his partner must be surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy) or using an acceptable, highly effective contraceptive method from Screening until at least 90 days have passed since study drug administration, including the follow-up period. Acceptable methods of contraception include the use of condoms and the use of an effective contraceptive for the female partner that includes: OCPs, long-acting implantable hormones, injectable hormones, a vaginal ring, or an IUD. Participants with same sex partners (abstinence from penile-vaginal intercourse) are eligible when this is their preferred and usual lifestyle.
WOCBP must have a negative pregnancy test at Screening and Day -1 and be willing to have additional pregnancy tests as required throughout the study.
Males must not donate sperm for at least 90 days after the last dose of AL01211.
Participants must have the ability and willingness to attend the necessary visits to the CRU.
Must sign an informed consent form (ICF) indicating that they understand the purpose of, and procedures required for the study and are willing to participate in the study.

Who cannot join

For Part A (SAD) and Part B (MAD)
A participant who meets any of the following exclusion criteria must be excluded from the study:
Any concomitant disease, condition, or treatment that could interfere with the conduct of the study,.
History or symptoms of significant psychiatric disease,
History or evidence of significant hepatic or renal disease or impairment, including clinically significant abnormalities in laboratory test results (including complete blood count, chemistry panel including kidney panel and liver function tests, and urinalysis).
Evidence of an active or suspected cancer or a history of malignancy for at least 5 years, except for: nonmelanoma skin cancer considered cured, curatively treated localized prostate cancer, or other in situ cancer.

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Known hypersensitivity or allergy to AL01211 or excipient contained in the drug formulation.
Uncontrolled hypertension of > 140/90 mm Hg despite optimal therapy.
Any of the following abnormal ECG findings at Screening:
PR interval > 210 ms or < 120 ms
QRS interval > 120 ms
QTcF interval > 450 ms
ST segment elevation or depression considered to be clinically significant
Any hepatic laboratory abnormality > 1.5 times the upper limit of the normal range (ULN), including alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP).
Impaired renal function as determined by the Investigator, based on an estimated glomerular filtration rate (eGFR) < 90mL/min/1.73 m2 at Screening.
Pregnant or lactating at Screening or planning to become pregnant (self or partner) at any time during the study, including up to 30 days after study drug administration (for female participants) or up to 90 days after study drug administration (for female partners of male participants).
Fever or symptomatic viral or bacterial infection at time of Screening. Testing for SARS-CoV-2 infection will be performed in accordance with local guidelines (health authorities, Institutional Review Boards/Independent Ethics Committees, and study centre policies) and at the discretion of the Investigator, if required.
Participants who have received live vaccines or attenuated vaccines within 1 month before dosing. Participants may receive vaccination for SARS-CoV-2 at the discretion of the Investigator as soon as they are eligible, and a vaccine is available.
The participant has, according to World Health Organization (WHO) Grading, a cortical cataract greater than one-quarter of the lens circumference (Grade cortical cataract-2 [COR-2]) or a posterior subcapsular cataract > 2mm (Grade posterior subcapsular cataract-2 [PSC-2]). Participants with nuclear cataracts will not be excluded.
The participant is currently receiving, or has received within the past month, potentially cataractogenic medications, including a chronic regimen (more frequently than every 2 weeks) of any route of corticosteroids (limited to medium and high-potency topical steroids; intranasal steroids are acceptable) or any medication that may cause cataract (such as phenothiazines and miotics, amiodarone, allopurinol, and phenytoin), according to the Prescribing Information.
Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), or human immunodeficiency virus (HIV) antibody at Screening.
Participants with a positive toxicology screening panel (urine test including qualitative identification of barbiturates, tetrahydrocannabinol (THC), amphetamines, benzodiazepines, opiates and cocaine), or alcohol breath test.
Participants with a history of substance abuse or dependency or history of recreational intravenous (IV) drug use over the last 5 years (by self-declaration)
Regular alcohol consumption defined as >21 alcohol units per week (where 1 unit = 284mL of beer, 25mL of 40% spirit, or a 125mL glass of wine). Participant is unwilling to abstain from alcohol beginning 48 hours prior to admission to the CRU until completion of the primary Follow-up visit. (Part A [except Cohort A3]: Day 7; Part A [Cohort A3]: Day 35; Part B: Day 21).
Use of any IP or investigational medical device within 30 days prior to Screening, or 5 half-lives of the product (whichever is the longest) or participation in more than four investigational drug studies within 1 year prior to screening.
Use of any prescription medications (other than hormonal contraception: OCPs, long-acting implantable hormones, injectable hormones, a vaginal ring or an IUD), over the-counter (OTC) medication, herbal remedies, supplements or vitamins 2 weeks prior to dosing and during the course of the study without prior approval of the Investigator and MM. Simple analgesia (paracetamol, nonsteroidal anti-inflammatory drug [NSAID]) may be permitted at the discretion of the Investigator.
History of anaphylaxis or other severe allergy to any drug, food, toxin, or other exposure.

Trial locations and contacts

1 site in 1 country.

Australia1 site

Nucleus Network
Melbourne, Victoria, 3004

Other trials in Australia

About this study

Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose in Healthy Volunteers and Autosomal Dominant Polycystic Kidney Disease Subjects Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AL01211

The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Oral AL01211 in healthy volunteers

Read the detailed description

This study is a Phase 1, first in human (FIH), randomized, double-blind, placebo-controlled study of AL01211 in healthy adult participants

The study consists of two parts:

Part A will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL01211 in a single ascending dose escalation study in approximately 40 healthy adult participants.

Part B will investigate the safety and tolerability, pharmacokinetics, and pharmacodynamics of AL01211 in a multiple ascending dose escalation study in approximately 40 healthy adult volunteers.

Conditions: Autosomal Dominant Polycystic Kidney
Being tested: AL01211 or Placebo (Part A), AL01211 or Placebo (Part B)
Medical subject headings: Polycystic Kidney, Autosomal Dominant

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AL01211 or Placebo (Part A)Drug
Five dose groups with doses ranging from 2mg to 60 mg
AL01211 or Placebo (Part B)Drug
Five dose groups with doses ranging from 2-60 mg daily. Each separate dose given for 14 days

Study groups in NCT04908462
Study group	Role	What they receive
Part A Healthy volunteers: Single ascending doses	Experimental	Not described Receives: Drug: AL01211 or Placebo (Part A)
Part B Healthy Volunteers Multiple ascending doses	Experimental	Not described Receives: Drug: AL01211 or Placebo (Part B)

What is being measured

The primary outcome is the main question the study is designed to answer.

To assess the safety and tolerability measures of AL01211 through Adverse Events/Serious Adverse Events in healthy adult participants
Number of participants with treatment related adverse events as assessed through CTCAE v5.0
Measured: Baseline to End of the Treatment assessed up to an average of 90 days

Secondary outcomes (3)

To assess the pharmacokinetics of AL01211 in healthy adult participants
The following parameters are used for pharmacokinetics: AUC0-last, AUC0-24h
Measured: Baseline to End of the Treatment assessed up to an average of 56 days
Measurement of glucosylceramide in plasma and urine following oral dosing of AL01211
Change in glucosylceramide levels
Measured: Baseline to End of the Treatment assessed up to an average of 56 days
Measurement of monosialodihexosylganglioside in plasma and urine following oral dosing of AL01211
Change in monosialodihexosylganglioside levels
Measured: Baseline to End of the Treatment assessed up to an average of 56 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Jun 202120 Jun 2022

First posted1 Jun 2021
Study start8 Jun 2021
Primary completion20 Jun 2022
Study completion20 Jun 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Autosomal Dominant Polycystic KidneyStudies of the same condition, open and under way.
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
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 69 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04908462
Sponsor's protocol ID: AL01211-101
Status last verified: Oct 2022
First submitted: 18 May 2021
Last updated: 14 Oct 2022
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Active, not recruitingObservational study
Role of ROS and cAMP-PKA Biomarkers in ADPKD
Autosomal Dominant Polycystic Kidney Disease
Not yet recruitingPhase 1
A Study to Investigate the Pharmacokinetics of JMKX003142 in Healthy Participants and Participants With Hepatic Impairment
ADPKD (Autosomal Dominant Polycystic Kidney Disease), Renal Edema
RecruitingPhase 1
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
RecruitingObservational study
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All Autosomal Dominant Polycystic Kidney trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose in Healthy Volunteers and Autosomal Dominant Polycystic Kidney Disease Subjects Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AL01211
ClinicalTrials.gov ID: NCT04908462
Acronym / sponsor ID: AL01211-101
Conditions: Autosomal Dominant Polycystic Kidney
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 69 participants (actual)
Lead sponsor: AceLink Therapeutics, Inc.Industry (pharma, biotech, devices)
Collaborators: Novotech (Australia) Pty Limited
Study start: 8 Jun 2021 (actual)
Primary completion: 20 Jun 2022 (actual)
Study completion: 20 Jun 2022 (actual)
First posted: 1 Jun 2021 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Oral AL01211 in healthy volunteers

Detailed description

This study is a Phase 1, first in human (FIH), randomized, double-blind, placebo-controlled study of AL01211 in healthy adult participants

The study consists of two parts:

Part A will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL01211 in a single ascending dose escalation study in approximately 40 healthy adult participants.

Part B will investigate the safety and tolerability, pharmacokinetics, and pharmacodynamics of AL01211 in a multiple ascending dose escalation study in approximately 40 healthy adult volunteers.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
Part A Healthy volunteers: Single ascending doses	Experimental	Drug: AL01211 or Placebo (Part A)
Part B Healthy Volunteers Multiple ascending doses	Experimental	Drug: AL01211 or Placebo (Part B)

Who may qualify

Ages: 18 to 55 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult

Key inclusion criteria (19 listed)

For Part A (SAD) and Part B (MAD)
To be eligible for the study, participants must meet all of the following inclusion criteria:
Healthy male or female volunteers, between 18 and 55 years of age
Participants in good health as determined by medical history, physical examination, vital signs, ECG, and clinical laboratory tests.
Body Mass Index (BMI) between 20.0 and 34.9 kg/m2 (inclusive).

Key exclusion criteria (27 listed)

For Part A (SAD) and Part B (MAD)
A participant who meets any of the following exclusion criteria must be excluded from the study:
Any concomitant disease, condition, or treatment that could interfere with the conduct of the study,.
History or symptoms of significant psychiatric disease,
History or evidence of significant hepatic or renal disease or impairment, including clinically significant abnormalities in laboratory test results (including complete blood count, chemistry panel including kidney panel and liver function tests, and urinalysis).

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
AL01211 or Placebo (Part A)Five dose groups with doses ranging from 2mg to 60 mg	Drug	Part A Healthy volunteers: Single ascending doses
AL01211 or Placebo (Part B)Five dose groups with doses ranging from 2-60 mg daily. Each separate dose given for 14 days	Drug	Part B Healthy Volunteers Multiple ascending doses

Primary outcome

To assess the safety and tolerability measures of AL01211 through Adverse Events/Serious Adverse Events in healthy adult participants
Time frame: Baseline to End of the Treatment assessed up to an average of 90 days
Number of participants with treatment related adverse events as assessed through CTCAE v5.0

3 secondary outcomes

To assess the pharmacokinetics of AL01211 in healthy adult participants
Time frame: Baseline to End of the Treatment assessed up to an average of 56 days
Measurement of glucosylceramide in plasma and urine following oral dosing of AL01211
Time frame: Baseline to End of the Treatment assessed up to an average of 56 days
Measurement of monosialodihexosylganglioside in plasma and urine following oral dosing of AL01211
Time frame: Baseline to End of the Treatment assessed up to an average of 56 days

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2022.
Individual participant dataSharing planned: No

Related trials for Autosomal Dominant Polycystic Kidney

RecruitingObservational study
Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Active, not recruitingObservational study
Role of ROS and cAMP-PKA Biomarkers in ADPKD
Autosomal Dominant Polycystic Kidney Disease
Not yet recruitingPhase 1
A Study to Investigate the Pharmacokinetics of JMKX003142 in Healthy Participants and Participants With Hepatic Impairment
ADPKD (Autosomal Dominant Polycystic Kidney Disease), Renal Edema
RecruitingPhase 1
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders

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.

Philip Ryan, MBBS, FRACP principal investigator

Nucleus Network Pty Ltd.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study of Autosomal Dominant Polycystic Kidney, run by AceLink Therapeutics, Inc. at 1 site in Australia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 27 exclusion criteria. 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 14 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Australia. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

To assess the safety and tolerability measures of AL01211 through Adverse Events/Serious Adverse Events in healthy adult participants (time frame: Baseline to End of the Treatment assessed up to an average of 90 days). 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 NCT04908462.

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 14 Oct 2022.