Menkes DiseasePhase 1/2NCT07398508
NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease
A Phase 1/2 study testing Droxidopa Oral Product in people with Menkes Disease, run by Stephen G. Kaler at 1 site in United States.
Trial summary
This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. Read more about this study
- Who is it for?
- Ages 7 to 17 years
- Conditions the study is for
- Menkes Disease
- What is being tested?
- Droxidopa Oral Product
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Aug 2026to 30 Jun 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 7 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children or adolescents with Menkes disease who survived beyond the expected natural history, attained independent ambulation, and attend school after early CuHis treatment for three years, who manifest clinical signs and symptoms of dysautonomia, e.g., orthostatic hypotension: specifically, a decrease in systolic or diastolic blood pressure of at least 20 or 10 mm Hg, respectively, within three minutes after standing, and/or chronic diarrhea: production of loose stools with or without increased stool frequency for more than four weeks immediately preceding enrollment.
- History of at least thrice weekly occurrence of dizziness/feeling lightheaded while standing upright and/or thrice weekly episodes of diarrhea or an urgent need to defecate after food ingestion for more than four weeks immediately preceding enrollment.
- Documented mutation in ATP7A.
- One parent must sign and date an Informed Consent Form (ICF) and patient must also assent.
- Age 7 to 17 years. (Enrollment will be staggered so that at least the first two children enrolled are aged 12-17 years.)
- Ability to adhere to the prescribed oral Northera (Droxidopa) regimen.
- Willingness to comply with all study visits and procedures.
Who cannot join
- Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease (i.e., calculated glomerular filtration rate <30 ml/min).
- History of age-adjusted stage 1 hypertension (≥ 95th percentile) [1] (Also see Attachment A).
- History of anti-hypertensive therapy, heart failure (or decreased ejection fraction), cardiac arrhythmia, or bleeding diatheses.
- Any disease or condition that, in the opinion of the Investigator, has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements.
- Any alpha-1 adrenoreceptor agonist, beta-blocker, DOPA decarboxylase inhibitor, midodrine, ephedrine, or any triptan medication as a concomitant medication.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Vagelos College of Physicians and Surgeons, Columbia University
New York, New York, 10032
Stephen G Kaler, MD, 212 305-3669, sgk2141@cumc.columbia.edu
Maryann Kaler, MS, mk5169@cumc.columbia.edu
About this study
Official title: Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease: Double-blind Placebo-controlled Randomized Crossover Clinical Trial
This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. The investigator hypothesizes that Northera (Droxidopa) treatment in pediatric Menkes disease survivors with symptoms of dysautonomia (e.g., syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, and bowel or bladder dysfunction) from deficiency of the cuproenzyme, dopamine-beta-hydroxylase, will be safe and will correct or improve blood neurochemical levels, raise systolic blood pressure, and produce symptomatic improvement and a better quality of life. The investigator will test this hypothesis, in six to ten child or adolescent Menkes disease survivors through a placebo-controlled trial to evaluate adverse event rates and whether oral administration of Northera (Droxidopa) at doses established for individual subjects by careful dose titration improves plasma norepinephrine and dihydroxyphenylglycol (DHPG) levels, raises systolic blood pressure, and improves performance on tests of physical exertion. As an exploratory outcome measure, the study will validate the Orthostatic Hypotension Symptom Assessment (OHSA) questionnaire for this population for two four-week periods of either active or placebo treatment.
Aim 1. Determine the safety of Droxidopa in Menkes disease pediatric survivors.
Aim 2. Determine the efficacy of Droxidopa in Menkes disease survivors. The investigator hypothesizes that low-dose Droxidopa treatment in classic Menkes disease survivors aged 7 to 17 will improve orthostatic hypotension and ameliorate other signs and symptoms of dysautonomia. This pilot study will employ an ascending dose paradigm in a double-blind placebo-controlled randomized crossover design to optimize statistical power and rigorously discern treatment effects on 1) tilt table tests of orthostatic hypotension, 2) systolic and diastolic blood pressure, 3) plasma neurochemical levels and 4) tests of physical exertion. The trial will also validate the Orthostatic Hypotension Symptom Assessment (OHSA) questionnaire for this population of children and adolescents. This study addresses an important unmet clinical need for subjects with a rare disease, Menkes disease.
Read the detailed description
This proposal outlines a concerted effort to evaluate the safety and efficacy of the synthetic amino acid L-threo-3,4-dihydroxyphenylserine (Droxidopa) for the symptoms of dysautonomia that are common in pediatric survivors of Menkes disease. Classic Menkes disease is a fatal X-linked recessive disorder of copper homeostasis that results from variants in a copper transporter gene, ATP7A, and has an estimated birth prevalence of 1 in 35,000 males. The tragedy of Menkes disease involves the apparent good health of affected infants both prenatally and during the first 6-8 weeks of life, after which signs and symptoms of central nervous system (CNS) degeneration emerge. Our prior clinical research demonstrated that early diagnosis (within approximately 28 days of birth) and three years of daily treatment with subcutaneous injections of Copper Histidinate (NDA #34,166) can extend survival and markedly improve neurodevelopmental and neurocognitive outcomes. However, survivors of the illness's severe CNS effects often develop symptoms of dysautonomia, such as syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, nocturnal bradycardia, and bowel or bladder dysfunction beginning in middle childhood. These problems are caused by partial deficiency of dopamine-beta-hydroxylase (DBH), which requires ATP7A to deliver Cu to intracellular compartments for incorporation as its enzymatic cofactor. DBH normally converts dopamine to norepinephrine, and similar symptoms are reported in individuals with congenital absence of DBH, an autosomal recessive disorder. The drug Droxidopa is a synthetic amino acid L-threo-3,4-dihydroxyphenylserine and can be metabolized by the non-copper dependent enzyme DOPA decarboxylase to produce norepinephrine, bypassing the DBH enzymatic defect. The investigator has demonstrated neurochemical improvements in a mouse model of Menkes disease with this compound, as well as in adult Menkes disease survivors over age 18 in a detailed trial (NCT04977388). The investigator recently developed a new liquid suspension of Droxidopa to test smaller doses in pediatric survivors of Menkes disease in a clinical trial.
- Conditions
- Menkes Disease
- Being tested
- Droxidopa Oral Product, Placebo Control
- Keywords
- Menkes disease, dysautonomia, orthostatic hypotension
- Medical subject headings
- Menkes Kinky Hair Syndrome, Autonomic Nervous System Diseases, Hypotension, Orthostatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Droxidopa Oral ProductDrug
Also called NORTHERA
1 month supply will contain pre-prepared capsules containing either 20mg, 40mg, or 60mg Droxidopa, plus 300 ml of simple syrup. For each dose, add the contents of 1 capsule and 10 ml of simple syrup to a plastic 1 ounce medication cup. Simple syrup will be dispensed from a larger bottle using a 10mL syringe. Mix by swirling briefly and consume the entire contents. Prepare the suspension freshly for each dose and take the prescribed dose twice daily, at approximately 8am and 2pm.
Placebo ControlOther
Also called Placebo pill
1 month supply will contain pre-prepared capsules containing cellulose microcrystalline placebo, plus 300 ml of simple syrup. For each dose, add the contents of 1 capsule and 10 ml of simple syrup to a plastic 1 ounce medication cup. Simple syrup will be dispensed from a larger bottle using a 10mL syringe. Mix by swirling briefly and consume the entire contents. Prepare the suspension freshly for each dose and take the prescribed dose twice daily, at approximately 8am and 2pm.
| Study group | Role | What they receive |
|---|---|---|
| Droxidopa Treatment Arm | Experimental | Oral administration of droxidopa study drug. Receives: Drug: Droxidopa Oral Product |
| Placebo Control Arm | Placebo comparator | Oral administration of placebo control capsules that are indistinguishable from study drug. Receives: Other: Placebo Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of SAEs
This is to measure safety and tolerability of study drug in children and adolescents with Menkes disease. The study will determine the adverse event profile of the formulation by comparing the time-to-event of serious adverse events (SAEs) and adverse events (AEs) between treatment and placebo arms as defined in the protocol.
Measured: 10 weeks
Secondary outcomes (5)
Change in level of plasma norepinephrine and dihydroxyphenylglycol after Northera (Droxidopa)
Plasma L-DOPS levels: Levels of L-threo-3,4-dihydroxyphenylserine (L-DOPS, droxidopa) may be measured for comparison to the known pharmacokinetic (PK) profile of Northera (Droxidopa) (peak level at approximately 3 hrs). The assay for plasma LDOPS and plasma catecholamines (High Performance Liquid Chromatography with electrochemical detection) is the same. Measurements of L-DOPS may permit correlation between plasma L-DOPS and plasma norepinephrine levels.
Measured: Baseline and post intervention at 4 weeks
Change in systolic blood pressure
Blood pressure (BP) will be measured using a standard blood pressure monitor while subjects are standing, and between supine and head-up tilt table positions after Northera (Droxidopa). Unit: mm Hg. Tilt Table testing involves positioning a patient onto the tilt table with feet resting on footplates. Continuous blood pressure monitors are then attached to the subject. The entire table can be tilted to move from 0˚ to 60˚angle in approximately 45s, allowing determination of changes in pulse and blood pressure based on supine (0˚) and standing (60˚) positions.
Measured: Baseline and post intervention at 4 weeks
Average number of daily bowel movements
Gastrointestinal symptoms will be monitored using irritable bowel syndrome-diarrhea report. Decreased daily bowel movements counts as an improvement.
Measured: Baseline, 4 weeks
Average time standing
This is to measure any improvement in physicality using performance of physical exertion tests. The longer the time standing, the greater the improvement. Unit: minutes.
Measured: Baseline and post intervention at 4 weeks
Average 6-minute walk distance
This is to measure any improvement in physicality using performance of physical exertion tests. The further the distance, the greater the improvement. The primary measurement taken from a 6-minute walk test (6MWT) is the final distance someone manages to walk, which we refer to as the 6MWD (6-minute walk distance), which can be used to assess a patient's functional status. For everyday healthy people, the average distance walked during this test is between 400 and 700 meters.
Measured: Baseline and post intervention at 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2026
- Study start10 Aug 2026
- Primary completion30 Jun 2030 (estimated)
- Study completion30 Jun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Menkes DiseaseStudies 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 1/2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Double-blind Placebo-controlled Randomized Crossover Clinical Trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stephen G. Kaler
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Stephen G. Kaler, Columbia University
- Investigators
- Stephen G Kaler, MD, MPH, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07398508
- Sponsor's protocol ID
- ACYY0326
- Status last verified
- Oct 2026
- First submitted
- 4 Feb 2026
- Last updated
- 5 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease: Double-blind Placebo-controlled Randomized Crossover Clinical Trial
- ClinicalTrials.gov ID
- NCT07398508
- Acronym / sponsor ID
- ACYY0326
- Conditions
- Menkes Disease
- Keywords
- Menkes disease, dysautonomia, orthostatic hypotension
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Stephen G. KalerUniversity, hospital or other
- Study start
- 10 Aug 2026 (actual)
- Primary completion
- 30 Jun 2030 (estimated)
- Study completion
- 30 Jun 2030 (estimated)
- First posted
- 10 Feb 2026 (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 clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. The investigator hypothesizes that Northera (Droxidopa) treatment in pediatric Menkes disease survivors with symptoms of dysautonomia (e.g., syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, and bowel or bladder dysfunction) from deficiency of the cuproenzyme, dopamine-beta-hydroxylase, will be safe and will correct or improve blood neurochemical levels, raise systolic blood pressure, and produce symptomatic improvement and a better quality of life. The investigator will test this hypothesis, in six to ten child or adolescent Menkes disease survivors through a placebo-controlled trial to evaluate adverse event rates and whether oral administration of Northera (Droxidopa) at doses established for individual subjects by careful dose titration improves plasma norepinephrine and dihydroxyphenylglycol (DHPG) levels, raises systolic blood pressure, and improves performance on tests of physical exertion. As an exploratory outcome measure, the study will validate the Orthostatic Hypotension Symptom Assessment (OHSA) questionnaire for this population for two four-week periods of either active or placebo treatment.
Aim 1. Determine the safety of Droxidopa in Menkes disease pediatric survivors.
Aim 2. Determine the efficacy of Droxidopa in Menkes disease survivors. The investigator hypothesizes that low-dose Droxidopa treatment in classic Menkes disease survivors aged 7 to 17 will improve orthostatic hypotension and ameliorate other signs and symptoms of dysautonomia. This pilot study will employ an ascending dose paradigm in a double-blind placebo-controlled randomized crossover design to optimize statistical power and rigorously discern treatment effects on 1) tilt table tests of orthostatic hypotension, 2) systolic and diastolic blood pressure, 3) plasma neurochemical levels and 4) tests of physical exertion. The trial will also validate the Orthostatic Hypotension Symptom Assessment (OHSA) questionnaire for this population of children and adolescents. This study addresses an important unmet clinical need for subjects with a rare disease, Menkes disease.
Detailed description
This proposal outlines a concerted effort to evaluate the safety and efficacy of the synthetic amino acid L-threo-3,4-dihydroxyphenylserine (Droxidopa) for the symptoms of dysautonomia that are common in pediatric survivors of Menkes disease. Classic Menkes disease is a fatal X-linked recessive disorder of copper homeostasis that results from variants in a copper transporter gene, ATP7A, and has an estimated birth prevalence of 1 in 35,000 males. The tragedy of Menkes disease involves the apparent good health of affected infants both prenatally and during the first 6-8 weeks of life, after which signs and symptoms of central nervous system (CNS) degeneration emerge. Our prior clinical research demonstrated that early diagnosis (within approximately 28 days of birth) and three years of daily treatment with subcutaneous injections of Copper Histidinate (NDA #34,166) can extend survival and markedly improve neurodevelopmental and neurocognitive outcomes. However, survivors of the illness's severe CNS effects often develop symptoms of dysautonomia, such as syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, nocturnal bradycardia, and bowel or bladder dysfunction beginning in middle childhood. These problems are caused by partial deficiency of dopamine-beta-hydroxylase (DBH), which requires ATP7A to deliver Cu to intracellular compartments for incorporation as its enzymatic cofactor. DBH normally converts dopamine to norepinephrine, and similar symptoms are reported in individuals with congenital absence of DBH, an autosomal recessive disorder. The drug Droxidopa is a synthetic amino acid L-threo-3,4-dihydroxyphenylserine and can be metabolized by the non-copper dependent enzyme DOPA decarboxylase to produce norepinephrine, bypassing the DBH enzymatic defect. The investigator has demonstrated neurochemical improvements in a mouse model of Menkes disease with this compound, as well as in adult Menkes disease survivors over age 18 in a detailed trial (NCT04977388). The investigator recently developed a new liquid suspension of Droxidopa to test smaller doses in pediatric survivors of Menkes disease in a clinical trial.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double-blind Placebo-controlled Randomized Crossover Clinical Trial
| Arm | Type | Receives |
|---|---|---|
| Droxidopa Treatment Arm | Experimental | Drug: Droxidopa Oral ProductOral administration of droxidopa study drug. |
| Placebo Control Arm | Placebo comparator | Other: Placebo ControlOral administration of placebo control capsules that are indistinguishable from study drug. |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Children or adolescents with Menkes disease who survived beyond the expected natural history, attained independent ambulation, and attend school after early CuHis treatment for three years, who manifest clinical signs and symptoms of dysautonomia, e.g., orthostatic hypotension: specifically, a decrease in systolic or diastolic blood pressure of at least 20 or 10 mm Hg, respectively, within three minutes after standing, and/or chronic diarrhea: production of loose stools with or without increased stool frequency for more than four weeks immediately preceding enrollment.
- History of at least thrice weekly occurrence of dizziness/feeling lightheaded while standing upright and/or thrice weekly episodes of diarrhea or an urgent need to defecate after food ingestion for more than four weeks immediately preceding enrollment.
- Documented mutation in ATP7A.
- One parent must sign and date an Informed Consent Form (ICF) and patient must also assent.
- Age 7 to 17 years. (Enrollment will be staggered so that at least the first two children enrolled are aged 12-17 years.)
Key exclusion criteria (5 listed)
- Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease (i.e., calculated glomerular filtration rate <30 ml/min).
- History of age-adjusted stage 1 hypertension (≥ 95th percentile) [1] (Also see Attachment A).
- History of anti-hypertensive therapy, heart failure (or decreased ejection fraction), cardiac arrhythmia, or bleeding diatheses.
- Any disease or condition that, in the opinion of the Investigator, has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements.
- Any alpha-1 adrenoreceptor agonist, beta-blocker, DOPA decarboxylase inhibitor, midodrine, ephedrine, or any triptan medication as a concomitant medication.
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 |
|---|---|---|
| Droxidopa Oral ProductAlso: NORTHERA1 month supply will contain pre-prepared capsules containing either 20mg, 40mg, or 60mg Droxidopa, plus 300 ml of simple syrup. For each dose, add the contents of 1 capsule and 10 ml of simple syrup to a plastic 1 ounce medication cup. Simple syrup will be dispensed from a larger bottle using a 10mL syringe. Mix by swirling briefly and consume the entire contents. Prepare the suspension freshly for each dose and take the prescribed dose twice daily, at approximately 8am and 2pm. | Drug | Droxidopa Treatment Arm |
| Placebo ControlAlso: Placebo pill1 month supply will contain pre-prepared capsules containing cellulose microcrystalline placebo, plus 300 ml of simple syrup. For each dose, add the contents of 1 capsule and 10 ml of simple syrup to a plastic 1 ounce medication cup. Simple syrup will be dispensed from a larger bottle using a 10mL syringe. Mix by swirling briefly and consume the entire contents. Prepare the suspension freshly for each dose and take the prescribed dose twice daily, at approximately 8am and 2pm. | Other | Placebo Control Arm |
Primary outcome
Incidence of SAEs
Time frame: 10 weeks
This is to measure safety and tolerability of study drug in children and adolescents with Menkes disease. The study will determine the adverse event profile of the formulation by comparing the time-to-event of serious adverse events (SAEs) and adverse events (AEs) between treatment and placebo arms as defined in the protocol.
5 secondary outcomes
Change in level of plasma norepinephrine and dihydroxyphenylglycol after Northera (Droxidopa)
Time frame: Baseline and post intervention at 4 weeks
Change in systolic blood pressure
Time frame: Baseline and post intervention at 4 weeks
Average number of daily bowel movements
Time frame: Baseline, 4 weeks
Average time standing
Time frame: Baseline and post intervention at 4 weeks
Average 6-minute walk distance
Time frame: Baseline and post intervention at 4 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Menkes Disease
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Active, not recruitingObservational study
Long Term Follow-up on Menkes Disease Patients
Menkes Disease
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.
Stephen G Kaler, MD, MPH principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Droxidopa Oral Product in people with Menkes Disease, run by Stephen G. Kaler 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 7 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 5 Oct 2026. 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. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Stephen G Kaler, MD, MPH (212 305-3669, sgk2141@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of SAEs (time frame: 10 weeks). 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 5 Oct 2026.