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Safety & Efficacy of AlloRx SC® in PTHS Patients

Primary Purpose

Pitt Hopkins Syndrome

Status

Not yet recruiting

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

AlloRx Stem Cells®

Placebo control

About this trial

This is an interventional treatment trial for Pitt Hopkins Syndrome

Eligibility Criteria

2 Years - 45 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Intervention Arm 1

Intervention Arm 2

Arm Description

AlloRx Stem Cells IV infusion treatment

IV infusion of normal saline

Outcomes

Primary Outcome Measures

Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS

Collection of all adverse events (AEs)

Safety: Incidence of serious adverse events

Collection of all serious adverse events

Secondary Outcome Measures

Change in motor function in individuals with PTHS

Ambulation measures by video and wearable device

Change in communication abilities in individuals with PTHS.

Observer-Reported Communication Ability Measure (ORCA)

Change in sleep habits

Sleep diary

Change in gastrointestinal health

Gastrointestinal Health Questionnaire

Change in breath holding spells

Diary of breath holding spells

Change in cognition

Bayley Scales of Infant Development (BSID-4)

Change in adaptive function

Vineland Adaptive Behavioral Scale-3

Change in autistic features

Childhood Autism Rating Scale (CARS)

Change in parent assessment of quality of life

Pediatric Quality of Life Questionnaire

Change in global clinical status

PTHS-specific Clinical Global Impression Scale

Full Information

NCT ID

NCT05165017

First Posted

October 29, 2021

Last Updated

December 6, 2021

Sponsor

Vitro Biopharma Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05165017

Brief Title

Safety & Efficacy of AlloRx SC® in PTHS Patients

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)

Study Type

Interventional

2. Study Status

Record Verification Date

December 2021

Overall Recruitment Status

Not yet recruiting

Study Start Date

December 2021 (Anticipated)

Primary Completion Date

December 2022 (Anticipated)

Study Completion Date

December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vitro Biopharma Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief 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. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pitt Hopkins Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Model Description

Randomized Placebo Controlled Double Blinded

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

26 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Intervention Arm 1

Arm Type

Active Comparator

Arm Description

AlloRx Stem Cells IV infusion treatment

Arm Title

Intervention Arm 2

Arm Type

Placebo Comparator

Arm Description

IV infusion of normal saline

Intervention Type

Biological

Intervention Name(s)

AlloRx Stem Cells®

Intervention Description

Umbilical cord-derived allogeneic mesenchymal stem cells

Intervention Type

Other

Intervention Name(s)

Placebo control

Intervention Description

Placebo infusion without mesenchymal stem cells

Primary Outcome Measure Information:

Title

Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS

Description

Collection of all adverse events (AEs)

Time Frame

Change from baseline to day 456 (end of study)

Title

Safety: Incidence of serious adverse events

Description

Collection of all serious adverse events

Time Frame

Change from baseline to day 456 (end of study)

Secondary Outcome Measure Information:

Title

Change in motor function in individuals with PTHS

Description

Ambulation measures by video and wearable device

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in communication abilities in individuals with PTHS.

Description

Observer-Reported Communication Ability Measure (ORCA)

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in sleep habits

Description

Sleep diary

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in gastrointestinal health

Description

Gastrointestinal Health Questionnaire

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in breath holding spells

Description

Diary of breath holding spells

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in cognition

Description

Bayley Scales of Infant Development (BSID-4)

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in adaptive function

Description

Vineland Adaptive Behavioral Scale-3

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in autistic features

Description

Childhood Autism Rating Scale (CARS)

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in parent assessment of quality of life

Description

Pediatric Quality of Life Questionnaire

Time Frame

Change from baseline to day 456 (end of study)

Title

Change in global clinical status

Description

PTHS-specific Clinical Global Impression Scale

Time Frame

Change from baseline to day 456 (end of study)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

12. IPD Sharing Statement

Plan to Share IPD

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Safety & Efficacy of AlloRx SC® in PTHS Patients

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