Classic GalactosemiaPhase 3NCT05418829
AT-007 in Adult Subjects With Classic Galactosemia (CG)
A Phase 3 study testing AT-007 in people with Classic Galactosemia, run by Applied Therapeutics, Inc. at 1 site in United States.
Trial summary
This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension. The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Classic Galactosemia
- What is being tested?
- AT-007
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2021to Dec 2022
- Phase
- Phase 3
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or non-pregnant, non-lactating female subject with a CG diagnosis
- Previous participation in Study AT-007-1001 Part D and/or Part D Extension
- No other significant health problems which preclude participation
Who cannot join
- Concomitant use of certain medications or over-the-counter therapies
- Discontinuation from Study AT-007-1001 Part D and/or Part D Extension due an adverse event
Trial locations and contacts
1 site in 1 country.
United States1 site
Hassman Research Institute
Berlin, New Jersey, 08009
About this study
Official title: An Open-Label Study to Evaluate the Long-Term Safety and Pharmacodynamic Efficacy of AT-007 in Adult Subjects With Classic Galactosemia (CG)
This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension.
The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.
Read the detailed description
Primary:
- To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with Classic Galactosemia (CG)
Secondary:
- To evaluate the long-term inhibition of galactitol, a biomarker of aldose reductase (AR) activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
- To evaluate the pharmacokinetic (PK) parameters of 12-month oral administration of AT-007 in adult subjects with CG
Exploratory:
- To evaluate the effect of 12-month oral administration of AT-007 on the levels of galactose and its other metabolites in adult subjects with CG
- To evaluate the burden of illness (BOI) of adult subjects with CG
- To evaluate quality of life (QOL) measures of adult subjects with CG
- To evaluate the effect of 12-month oral administration of AT-007 on the BOI and QOL of adult subjects with CG
- Conditions
- Classic Galactosemia
- Being tested
- AT-007
- Medical subject headings
- Galactosemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AT-007Drug
AT-007 20 mg/kg once daily
| Study group | Role | What they receive |
|---|---|---|
| AT-007 | Experimental | Open-label AT-007 20 mg/kg once daily Receives: Drug: AT-007 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
Number of participants with treatment-emergent adverse events and broken down by organ class and relatedness.
Measured: 12 months
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by clinical laboratory test results.
Number of participants with clinically-significant changes in clinical laboratory test results.
Measured: 12 months
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.
Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.
Measured: 12 months
Secondary outcomes (3)
To evaluate the long-term change of galactitol, a biomarker of AR activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
Measured: 12 months
To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG
Maximum concentration will be determined by individual concentration time-data (mg/L)
Measured: 12 months
To evaluate the pharmacokinetic (PK) parameter Area-Under-the-Curve (AUC) of 12-month oral administration of AT-007 in adult subjects with CG (mg*h/L)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2022
- Study start1 Oct 2021
- Primary completionNov 2022 (estimated)
- Study completionDec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Classic GalactosemiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Applied Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05418829
- Sponsor's protocol ID
- AT-007-1006
- Status last verified
- Jun 2022
- First submitted
- 16 Feb 2022
- Last updated
- 14 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Long-Term Safety and Pharmacodynamic Efficacy of AT-007 in Adult Subjects With Classic Galactosemia (CG)
- ClinicalTrials.gov ID
- NCT05418829
- Acronym / sponsor ID
- AT-007-1006
- Conditions
- Classic Galactosemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Applied Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Oct 2021 (actual)
- Primary completion
- Nov 2022 (estimated)
- Study completion
- Dec 2022 (estimated)
- First posted
- 14 Jun 2022 (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.
This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension.
The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.
Detailed description
Primary:
- To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with Classic Galactosemia (CG)
Secondary:
- To evaluate the long-term inhibition of galactitol, a biomarker of aldose reductase (AR) activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
- To evaluate the pharmacokinetic (PK) parameters of 12-month oral administration of AT-007 in adult subjects with CG
Exploratory:
- To evaluate the effect of 12-month oral administration of AT-007 on the levels of galactose and its other metabolites in adult subjects with CG
- To evaluate the burden of illness (BOI) of adult subjects with CG
- To evaluate quality of life (QOL) measures of adult subjects with CG
- To evaluate the effect of 12-month oral administration of AT-007 on the BOI and QOL of adult subjects with CG
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AT-007 | Experimental | Drug: AT-007Open-label AT-007 20 mg/kg once daily |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or non-pregnant, non-lactating female subject with a CG diagnosis
- Previous participation in Study AT-007-1001 Part D and/or Part D Extension
- No other significant health problems which preclude participation
Key exclusion criteria (2 listed)
- Concomitant use of certain medications or over-the-counter therapies
- Discontinuation from Study AT-007-1001 Part D and/or Part D Extension due an adverse event
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 |
|---|---|---|
| AT-007AT-007 20 mg/kg once daily | Drug | AT-007 |
Primary outcome
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
Time frame: 12 months
Number of participants with treatment-emergent adverse events and broken down by organ class and relatedness.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by clinical laboratory test results.
Time frame: 12 months
Number of participants with clinically-significant changes in clinical laboratory test results.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.
Time frame: 12 months
Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.
3 secondary outcomes
To evaluate the long-term change of galactitol, a biomarker of AR activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
Time frame: 12 months
To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG
Time frame: 12 months
To evaluate the pharmacokinetic (PK) parameter Area-Under-the-Curve (AUC) of 12-month oral administration of AT-007 in adult subjects with CG (mg*h/L)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2022.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AT-007 in people with Classic Galactosemia, run by Applied Therapeutics, Inc. at 1 site in United States.
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 3 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 14 Jun 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 United States. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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 evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events. (time frame: 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Jun 2022.