Sponsor study identifier
interventional
26
0 countries
N/A
Study Start
December 2021
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Primary Completion
December 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
December 2023
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 2023
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
October 29, 2021
Actual (reported)December 2021
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
December 21, 2021
Actual (reported)December 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
December 2023
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS
Collection of all adverse events (AEs)
Change from baseline to day 456 (end of study)
Safety: Incidence of serious adverse events
Collection of all serious adverse events
Change from baseline to day 456 (end of study)
Change in motor function in individuals with PTHS
Change from baseline to day 456 (end of study)
Change in communication abilities in individuals with PTHS.
Change from baseline to day 456 (end of study)
Change in sleep habits
Change from baseline to day 456 (end of study)
Change in gastrointestinal health
Change from baseline to day 456 (end of study)
Change in breath holding spells
Change from baseline to day 456 (end of study)
AlloRx Stem Cells IV infusion treatment
IV infusion of normal saline
Umbilical cord-derived allogeneic mesenchymal stem cells
Placebo infusion without mesenchymal stem cells
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
First Submitted
October 29, 2021
First Posted
December 21, 2021
Study Start
December 2021
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Contact the study team to confirm eligibility.
Primary Completion
December 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
December 2023
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Last Updated
December 21, 2021
Record last verified: 2021-12
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Source: ClinicalTrials.gov.
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