Pitt Hopkins SyndromePhase 1/2NCT05165017
Safety & Efficacy of AlloRx SC® in PTHS Patients
A Phase 1/2 study testing AlloRx Stem Cells® in people with Pitt Hopkins Syndrome, run by Vitro Biopharma Inc..
Trial summary
This study will evaluate the safety of AlloRx Stem Cells® in subjects with Pitt Hopkins syndrome ages 2-45 with a molecularly confirmed pathogenic mutation in TCF4 or 18q deletion including TCF4. Read more about this study
- Who is it for?
- Ages 2 to 45 years
- Conditions the study is for
- Pitt Hopkins Syndrome
- What is being tested?
- AlloRx Stem Cells®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- No sites listed yet
- Study dates
- Dec 2021to Dec 2023
- Phase
- Phase 1/2
- 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 2 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed informed consent from parent(s) or legal guardian(s)/authorized representative(s) (LAR)
- Documented genetic confirmation of mutation in TCF4, with clinical diagnosis of Pitt Hopkins Syndrome (PTHS)
- Stable seizure control (defined as clinically stable with no changes in antiepileptic medications or use of rescue medication over the prior 1 month before the screening visit, other than weight associated dose adjustments)
- Normal renal function with serum creatinine and spot urine protein within normal limits
- Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including intravenous infusion
Who cannot join
- Any change in medications or diet/supplements intended to treat symptoms of PTHS (e.g., sleeping aids, supplements, cannabidiol products) over the prior 3 months before screening
- Inability to ambulate independently or with an assistive device or caregiver handhold
- Any bleeding or platelet disorder
- Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, make the patient unsuitable for participation in, and/or unable to complete the study procedures
- Any laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study result
- Known positive for hepatitis B virus, hepatitis C virus, or human immunodeficiency virus (HIV). Patient is pregnant or lactating
Show all 9Show fewer
- Usage of drugs that increase the risk of bleeding (e.g., heparin, low molecular weight heparin, platelet inhibitors).
- Use of any investigational oligonucleotide and any investigational drugs in the past 6 months
- Any prior use of gene therapy
About this study
Official title: Randomized Double Blind Placebo Controlled Study of the Safety & Efficacy of Therapeutic Treatment With AlloRx Stem Cells® in Patients With Pitt Hopkins Syndrome (Phase 1/2 Study)
This study will evaluate the safety of AlloRx Stem Cells® in subjects with Pitt Hopkins syndrome ages 2-45 with a molecularly confirmed pathogenic mutation in TCF4 or 18q deletion including TCF4. Participants will receive mesenchymal stem cells infusions every 3 months for a year with completion of outcome measures to assess the efficacy of the product.
Read the detailed description
This is a phase 1/2, randomized placebo controlled double blinded standard dose study to evaluate the safety and tolerability of AlloRx® stem cells in pediatric patients with a confirmed diagnosis of a TCF4 mutation consistent with haploinsufficiency causing PTHS. Approximately 26 patients (male and female) ages ≥ 2 and≤ 45 years of age with a genetically confirmed diagnosis of Pitt-Hopkins syndrome caused by molecular findings consistent with a pathogenic mutation in the gene TCF4. Written informed consent will be obtained from the patient's parent or legal guardian/ authorized representative (LAR) prior to participation in the study. The study includes screening, baseline, treatment and safety follow up periods. The procedure for intravenous administration is performed under the supervision of clinician with experience caring for patients with PTHS.
Each patient will remain inpatient for at least 24 hours after the initial dose of AlloRx, for frequent vital sign monitoring, electrocardiogram (ECG), blood and urine safety tests, and neurologic assessments. Investigators will work closely with the parent/legal guardian to ensure that any signs of discomfort/distress are immediately communicated to study staff. For the administration of the initial dose of study drug in each study cohort, patients will be dosed in a sequential fashion with no more than one patient receiving their first dose of study drug on the same day.
An independent data DSMB will review all safety and laboratory data throughout the study on a periodic basis and ad hoc should an SAE occur. The DSMB will also review all available safety data when all patients have received 2 doses of AlloRx and have had at least two weeks of follow-up in order to determine if it is safe to dose the next patient.
- Conditions
- Pitt Hopkins Syndrome
- Being tested
- AlloRx Stem Cells®, Placebo control
- Medical subject headings
- Pitt-Hopkins syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlloRx Stem Cells®Biological
Umbilical cord-derived allogeneic mesenchymal stem cells
Placebo controlOther
Placebo infusion without mesenchymal stem cells
| Study group | Role | What they receive |
|---|---|---|
| Intervention Arm 1 | Active comparator | AlloRx Stem Cells IV infusion treatment Receives: Biological: AlloRx Stem Cells® |
| Intervention Arm 2 | Placebo comparator | IV infusion of normal saline Receives: Other: Placebo control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS
Collection of all adverse events (AEs)
Measured: Change from baseline to day 456 (end of study)
Safety: Incidence of serious adverse events
Collection of all serious adverse events
Measured: Change from baseline to day 456 (end of study)
Secondary outcomes (10)
Change in motor function in individuals with PTHS
Ambulation measures by video and wearable device
Measured: Change from baseline to day 456 (end of study)
Change in communication abilities in individuals with PTHS.
Observer-Reported Communication Ability Measure (ORCA)
Measured: Change from baseline to day 456 (end of study)
Change in sleep habits
Sleep diary
Measured: Change from baseline to day 456 (end of study)
Change in gastrointestinal health
Gastrointestinal Health Questionnaire
Measured: Change from baseline to day 456 (end of study)
Change in breath holding spells
Diary of breath holding spells
Measured: Change from baseline to day 456 (end of study)
Change in cognition
Bayley Scales of Infant Development (BSID-4)
Measured: Change from baseline to day 456 (end of study)
Change in adaptive function
Vineland Adaptive Behavioral Scale-3
Measured: Change from baseline to day 456 (end of study)
Change in autistic features
Childhood Autism Rating Scale (CARS)
Measured: Change from baseline to day 456 (end of study)
Change in parent assessment of quality of life
Pediatric Quality of Life Questionnaire
Measured: Change from baseline to day 456 (end of study)
Change in global clinical status
PTHS-specific Clinical Global Impression Scale
Measured: Change from baseline to day 456 (end of study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2021
- Study startDec 2021 (estimated)
- Primary completionDec 2022 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pitt Hopkins 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized Placebo Controlled Double Blinded
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vitro Biopharma Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05165017
- Sponsor's protocol ID
- ALLORX-PTHS
- Status last verified
- Dec 2021
- First submitted
- 29 Oct 2021
- Last updated
- 21 Dec 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Double Blind Placebo Controlled Study of the Safety & Efficacy of Therapeutic Treatment With AlloRx Stem Cells® in Patients With Pitt Hopkins Syndrome (Phase 1/2 Study)
- ClinicalTrials.gov ID
- NCT05165017
- Acronym / sponsor ID
- ALLORX-PTHS
- Conditions
- Pitt Hopkins Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Vitro Biopharma Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2021 (estimated)
- Primary completion
- Dec 2022 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 21 Dec 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.
This study will evaluate the safety of AlloRx Stem Cells® in subjects with Pitt Hopkins syndrome ages 2-45 with a molecularly confirmed pathogenic mutation in TCF4 or 18q deletion including TCF4. Participants will receive mesenchymal stem cells infusions every 3 months for a year with completion of outcome measures to assess the efficacy of the product.
Detailed description
This is a phase 1/2, randomized placebo controlled double blinded standard dose study to evaluate the safety and tolerability of AlloRx® stem cells in pediatric patients with a confirmed diagnosis of a TCF4 mutation consistent with haploinsufficiency causing PTHS. Approximately 26 patients (male and female) ages ≥ 2 and≤ 45 years of age with a genetically confirmed diagnosis of Pitt-Hopkins syndrome caused by molecular findings consistent with a pathogenic mutation in the gene TCF4. Written informed consent will be obtained from the patient's parent or legal guardian/ authorized representative (LAR) prior to participation in the study. The study includes screening, baseline, treatment and safety follow up periods. The procedure for intravenous administration is performed under the supervision of clinician with experience caring for patients with PTHS.
Each patient will remain inpatient for at least 24 hours after the initial dose of AlloRx, for frequent vital sign monitoring, electrocardiogram (ECG), blood and urine safety tests, and neurologic assessments. Investigators will work closely with the parent/legal guardian to ensure that any signs of discomfort/distress are immediately communicated to study staff. For the administration of the initial dose of study drug in each study cohort, patients will be dosed in a sequential fashion with no more than one patient receiving their first dose of study drug on the same day.
An independent data DSMB will review all safety and laboratory data throughout the study on a periodic basis and ad hoc should an SAE occur. The DSMB will also review all available safety data when all patients have received 2 doses of AlloRx and have had at least two weeks of follow-up in order to determine if it is safe to dose the next patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized Placebo Controlled Double Blinded
| Arm | Type | Receives |
|---|---|---|
| Intervention Arm 1 | Active comparator | Biological: AlloRx Stem Cells®AlloRx Stem Cells IV infusion treatment |
| Intervention Arm 2 | Placebo comparator | Other: Placebo controlIV infusion of normal saline |
Who may qualify
- Ages
- 2 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Signed informed consent from parent(s) or legal guardian(s)/authorized representative(s) (LAR)
- Documented genetic confirmation of mutation in TCF4, with clinical diagnosis of Pitt Hopkins Syndrome (PTHS)
- Stable seizure control (defined as clinically stable with no changes in antiepileptic medications or use of rescue medication over the prior 1 month before the screening visit, other than weight associated dose adjustments)
- Normal renal function with serum creatinine and spot urine protein within normal limits
- Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including intravenous infusion
Key exclusion criteria (9 listed)
- Any change in medications or diet/supplements intended to treat symptoms of PTHS (e.g., sleeping aids, supplements, cannabidiol products) over the prior 3 months before screening
- Inability to ambulate independently or with an assistive device or caregiver handhold
- Any bleeding or platelet disorder
- Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, make the patient unsuitable for participation in, and/or unable to complete the study procedures
- Any laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study result
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 |
|---|---|---|
| AlloRx Stem Cells®Umbilical cord-derived allogeneic mesenchymal stem cells | Biological | Intervention Arm 1 |
| Placebo controlPlacebo infusion without mesenchymal stem cells | Other | Intervention Arm 2 |
Primary outcome
Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS
Time frame: Change from baseline to day 456 (end of study)
Collection of all adverse events (AEs)
Safety: Incidence of serious adverse events
Time frame: Change from baseline to day 456 (end of study)
Collection of all serious adverse events
10 secondary outcomes
Change in motor function in individuals with PTHS
Time frame: Change from baseline to day 456 (end of study)
Change in communication abilities in individuals with PTHS.
Time frame: Change from baseline to day 456 (end of study)
Change in sleep habits
Time frame: Change from baseline to day 456 (end of study)
Change in gastrointestinal health
Time frame: Change from baseline to day 456 (end of study)
Change in breath holding spells
Time frame: Change from baseline to day 456 (end of study)
Change in cognition
Time frame: Change from baseline to day 456 (end of study)
Change in adaptive function
Time frame: Change from baseline to day 456 (end of study)
Change in autistic features
Time frame: Change from baseline to day 456 (end of study)
Change in parent assessment of quality of life
Time frame: Change from baseline to day 456 (end of study)
Change in global clinical status
Time frame: Change from baseline to day 456 (end of study)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2021.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AlloRx Stem Cells® in people with Pitt Hopkins Syndrome, run by Vitro Biopharma Inc..
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 5 inclusion and 9 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 21 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
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?
Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS (time frame: Change from baseline to day 456 (end of study)). The study lists 2 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 21 Dec 2021.