- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05165017
Safety & Efficacy of AlloRx SC® in PTHS Patients
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)
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Enrollment (Anticipated)
Phase
- Phase 2
- Phase 1
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Intervention Arm 1
AlloRx Stem Cells IV infusion treatment
|
Umbilical cord-derived allogeneic mesenchymal stem cells
|
Placebo Comparator: Intervention Arm 2
IV infusion of normal saline
|
Placebo infusion without mesenchymal stem cells
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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)
|
Change from baseline to day 456 (end of study)
|
Safety: Incidence of serious adverse events
Time Frame: Change from baseline to day 456 (end of study)
|
Collection of all serious adverse events
|
Change from baseline to day 456 (end of study)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in motor function in individuals with PTHS
Time Frame: Change from baseline to day 456 (end of study)
|
Ambulation measures by video and wearable device
|
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)
|
Observer-Reported Communication Ability Measure (ORCA)
|
Change from baseline to day 456 (end of study)
|
Change in sleep habits
Time Frame: Change from baseline to day 456 (end of study)
|
Sleep diary
|
Change from baseline to day 456 (end of study)
|
Change in gastrointestinal health
Time Frame: Change from baseline to day 456 (end of study)
|
Gastrointestinal Health Questionnaire
|
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)
|
Diary of breath holding spells
|
Change from baseline to day 456 (end of study)
|
Change in cognition
Time Frame: Change from baseline to day 456 (end of study)
|
Bayley Scales of Infant Development (BSID-4)
|
Change from baseline to day 456 (end of study)
|
Change in adaptive function
Time Frame: Change from baseline to day 456 (end of study)
|
Vineland Adaptive Behavioral Scale-3
|
Change from baseline to day 456 (end of study)
|
Change in autistic features
Time Frame: Change from baseline to day 456 (end of study)
|
Childhood Autism Rating Scale (CARS)
|
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)
|
Pediatric Quality of Life Questionnaire
|
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)
|
PTHS-specific Clinical Global Impression Scale
|
Change from baseline to day 456 (end of study)
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Anticipated)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALLORX-PTHS
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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