Rett SyndromePhase 2NCT03633058

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

A Phase 2 study testing Ketamine in people with Rett Syndrome, run by Rett Syndrome Research Trust at 7 sites in United States.

Completed

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

Official record

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Trial summary

This 2 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Read more about this study

Who is it for?: Ages 6 to 12 years, women only
Conditions the study is for: Rett Syndrome
What is being tested?: Ketamine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 23 participants
Where: 7 sitesUnited States
Study dates: 12 Mar 2019to 22 Nov 2021
Phase: Phase 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 6 to 12 years
Women only
No healthy volunteers
Child

Who may be able to join

Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
between the ages of 6 and 12, inclusive, who have not achieved menarche
ability to take oral medications
are generally healthy.

Who cannot join

Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,
are taking medications that may interact with ketamine,
have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.

Trial locations and contacts

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

United States7 sites

Children's Hospital Colorado
Aurora, Colorado, 80045
University of Alabama Birmingham School of Medicine
Birmingham, Alabama, 35294
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Texas Children's Hospital
Houston, Texas, 77030
Vanderbilt University Medical Center
Nashville, Tennessee, 37203
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome

Read the detailed description

This study is designed to assess oral ketamine for the treatment of Rett Syndrome and consists of up to 4 ascending dose cohorts, each assessing 1 dose level of ketamine vs placebo. Patients will receive in either order, a 5-day BID regimen of both placebo and the cohort-specified dose level of oral ketamine. Patients may only participate in 1 cohort. Safety and tolerability will be assessed via patient disposition, vital signs, physical examination, adverse events and concomitant medication use. Efficacy will be assessed via physician and caregiver questionnaires and assessments, and continuous, wearable, at-home biosensor data collection. An independent safety committee will review safety data from each cohort to determine if the subsequent ascending dose cohort is warranted. A total of 12 patients per cohort is anticipated at approximately 7 sites. The screening period will last between 2 and 4 weeks, the cross-over treatment period will last 4 weeks, and the safety follow-up period will last 2 weeks. Total patient participation is approximately 8-10 weeks.

Conditions: Rett Syndrome
Being tested: Ketamine
Keywords: MECP2, RTT, Retts, ketamine
Medical subject headings: Rett Syndrome

What participants receive

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

KetamineDrug
Also called Ketalar
oral ketamine dosed twice daily for 5 days

Study groups in NCT03633058
Study group	Role	What they receive
0.75 mg/kg	Experimental	ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo Receives: Drug: Ketamine
1.5 mg/kg	Experimental	ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo Receives: Drug: Ketamine

What is being measured

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

Dose-Limiting Adverse Events
The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized
Measured: 6 weeks

Timeline

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

12 Mar 201922 Nov 2021

First posted16 Aug 2018
Study start12 Mar 2019
Primary completion8 Nov 2021
Study completion22 Nov 2021

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 Rett SyndromeStudies 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Model description: Patients will receive placebo and ketamine for 5 days BID each, in a double-blind treatment order, and will be assessed for 2 weeks after each treatment.
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 23 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03633058
Sponsor's protocol ID: Ket-101-RSRT
Status last verified: May 2024
First submitted: 8 Aug 2018
Last updated: 28 May 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Campbell K, Neul JL, Lieberman DN, Berry-Kravis E, Benke TA, Fu C, Percy A, Suter B, Morris D, Carpenter RL, Marsh ED, von Hehn J. A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome. J Neurodev Disord. 2025 Jan 24;17(1):4. doi: 10.1186/s11689-025-09591-y.
PubMed 39856538

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

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All Rett Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 28 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome
ClinicalTrials.gov ID: NCT03633058
Acronym / sponsor ID: Ket-101-RSRT
Conditions: Rett Syndrome
Keywords: MECP2, RTT, Retts, ketamine
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 23 participants (actual)
Lead sponsor: Rett Syndrome Research TrustUniversity, hospital or other
Collaborators: Vanderbilt University Medical Center
Study start: 12 Mar 2019 (actual)
Primary completion: 8 Nov 2021 (actual)
Study completion: 22 Nov 2021 (actual)
First posted: 16 Aug 2018 (actual)
Results: Posted 27 Mar 2024

Clinical summary

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

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2
Model description: Patients will receive placebo and ketamine for 5 days BID each, in a double-blind treatment order, and will be assessed for 2 weeks after each treatment.

Arm	Type	Receives
0.75 mg/kg	Experimental	Drug: Ketamineketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo
1.5 mg/kg	Experimental	Drug: Ketamineketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo

Who may qualify

Ages: 6 to 12 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (4 listed)

Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
between the ages of 6 and 12, inclusive, who have not achieved menarche
ability to take oral medications
are generally healthy.

Key exclusion criteria (3 listed)

Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,
are taking medications that may interact with ketamine,
have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.

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
KetamineAlso: Ketalaroral ketamine dosed twice daily for 5 days	Drug	0.75 mg/kg, 1.5 mg/kg

Primary outcome

Dose-Limiting Adverse Events
Time frame: 6 weeks
The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized

Results and safety

Results posted to the registry on 27 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
0.75 mg/kg (Placebo First, Then 0.75 mg/kg Ketamine)	5	5
0.75 mg/kg (0.75 mg/kg Ketamine First, Then Placebo)	6	6
1.5 mg/kg (Placebo First, Then 1.5 mg/kg Ketamine)	6	5
1.5 mg/kg (1.5 mg/kg Ketamine First, Then Placebo)	6	6

Primary outcome: Dose-Limiting Adverse Events

Time frame: 6 weeks. Count of participants. Unit: Participants

Measure	0.75 mg/kg Ketamine n = 11	0.75 mg/kg Placebo n = 11	1.5 mg/kg Ketamine n = 12	1.5 mg/kg Placebo n = 12
TEAEs	2	4	6	3
SAEs	0	0	0	0
AEs leading to discontinuation	0	0	1	0
AEs leading to dose interruption	0	0	0	0
No TEAEs reported	9	7	5	9

Population analysed: The Safety Population consisted of all patients who received at least 1 dose of study treatment.

Adverse events

Time frame: 6 weeks. Other adverse events reported at 4.3% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
0.75 mg/kg Ketamine	0 of 11 (0%)	2 of 11 (18.2%)	0 of 11
0.75 mg/kg Placebo	0 of 11 (0%)	4 of 11 (36.4%)	0 of 11
1.5 mg/kg Ketamine	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12
1.5 mg/kg Placebo	0 of 12 (0%)	3 of 12 (25%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 39856538Campbell K, Neul JL, Lieberman DN, Berry-Kravis E, Benke TA, Fu C, Percy A, Suter B, Morris D, Carpenter RL, Marsh ED, von Hehn J. A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome. J Neurodev Disord. 2025 Jan 24;17(1):4. doi: 10.1186/s11689-025-09591-y.
Individual participant dataSharing planned: No

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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.

Jeffrey Neul, MD, PhD principal investigator

Vanderbilt University Medical Center

Jana von Hehn, PhD study director

Rett Syndrome Research Trust

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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ketamine in people with Rett Syndrome, run by Rett Syndrome Research Trust at 7 sites 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 4 inclusion and 3 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 28 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites 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 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 27 Mar 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?

Dose-Limiting Adverse Events (time frame: 6 weeks). 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 NCT03633058, 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 28 May 2024.