HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

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ClinicalTrials.gov Identifier: NCT05154851

Expanded Access Status : No longer available

First Posted : December 13, 2021

Last Update Posted : August 1, 2022

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborator:

Hope Biosciences

Information provided by (Responsible Party):

Hope Biosciences ( Hope Biosciences Stem Cell Research Foundation )

Study Description

Brief Summary:

This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded access, the patient will receive the investigational product through 14 intravenous infusions, followed by Follow-Up visit and an End of Study.

Condition or disease	Intervention/treatment
Congenital Muscular Dystrophy Due to Lamin A/C Mutation	Biological: HB-adMSCs

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Detailed Description:

This Expanded Access includes an up to 28 days screening period, a 44-week Treatment Period, and a 52-week Safety Follow-up Period. All periods are associated with evaluations and procedures that must be performed at specific time points.

-Screening

At the Screening Visit (Visit 1), the study's participant information will be collected by delegated personnel to evaluate trial eligibility. The following information is required to determine eligibility:

Parent Permission before any trial-related activities.
Collection of demographic information, such as age, race, ethnicity, date of birth, gender, and relevant medical and surgical history.
Collection of Medical History and concomitant medications, including relevant information about study participants' past and present health.
Inclusion and Exclusion criteria evaluation.
Measurement of vital signs includes respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight and height measurement.
Blood chemistry, Coagulation panel, Inflammatory Markers, & Hematology.
Physical examination by Principal Investigator.

Within 28 days of the Visit 1 - Screening, the principal investigator must decide the participant's eligibility. Once the principal investigator has confirmed the subject's eligibility, the subject will be scheduled to receive Infusion 1.

Infusion 1 - Baseline

The following evaluations will be performed during this visit:

Update medical history and concomitant medications if any change occurred since the last visit.
Measure vital signs, including respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight measurement.
Physical examination by Principal Investigator. As this 2-year-old is unable to walk or hold his head up without physical support, assessment for neurologic sequelae will include the following observations and discharge instructions for observations to continue at home by the primary caregivers:
- Unusual /new weakness or numbness on one side of the body
- New or different slurred speech or new language difficulty
- New vision problems
- Sudden lethargy or drowsiness
- Seizure
CHOP INTEND, PDMS-2 & PEDI-CAT by delegated personnel.
Investigational product administration by delegated study personnel. HB-adMSCs should only be administered intravenously, with vital sign monitoring of two hours.
Assess the incidence of any adverse event.
Twenty-four hours after administration of the investigational product, study participants' parents or guardians will be contacted by telephone call to assess the incidence of adverse events.
- Infusion 2 through Infusion 13.

The following assessments are necessary during these visits:

Update medical history and concomitant medications if any change occurred since the last visit.
Measure vital signs, including respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight measurement.
Physical examination by Principal Investigator. As this 2-year-old is unable to walk or hold his head up without physical support, assessment for neurologic sequelae will include the following observations and discharge instructions for observations to continue at home by the primary caregivers:
- Unusual /new weakness or numbness on one side of the body
- New or different slurred speech or new language difficulty
- New vision problems
- Sudden lethargy or drowsiness
- Seizure
CHOP INTEND, PDMS-2 & PEDI-CAT by delegated personnel (at Inf. 1, 7, and EOS).
Coagulation panel & Hematology. - (at Infusions 2,4,7,10, and EOS).
Inflammatory Markers & Blood chemistry at Infusions 2, 7 and EOS.
Investigational product administration by delegated study personnel. HB-adMSCs should only be administered intravenously, with vital sign monitoring of two hours.
Assess the incidence of any adverse event.
Twenty-four hours after administration of the investigational product, study participants' parents or guardians will be contacted by telephone call to assess the incidence of adverse events. The child's skin and eyes will be checked for symptoms of yellowing at each visit, and caregivers will be asked whether they have seen any vomiting, nausea, or the subject looking to be more tired than usual. Discharge instructions will include monitoring for these symptoms. Liver enzymes will be assessed at Screening, Infusion 2, 4, 7, 10, and End of Study per-protocol; however, these laboratory samples can be collected more often if the principal investigator indicates.
- Safety Follow Up

The following evaluations will need to be completed during this visit:

Update medical history and concomitant medications if any change occurred since the last visit.
Measure vital signs, including respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight measurement.
Physical examination by Principal Investigator.

-End of Study Visit
Update medical history and concomitant medications if any change occurred since the last visit.
Measure vital signs, including respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight measurement.
Collect laboratory samples, including Comprehensive Metabolic Panel (CMP), Complete Blood Count (CBC), Inflammatory Markers, and Coagulation panel.
Physical examination by Principal Investigator.
CHOP INTEND, PDMS-2 & PEDI-CAT by delegated personnel.

Study Design

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Study Type :	Expanded Access
Expanded Access Type :	Individual Patients
Official Title:	An Expanded Access IND to Evaluate the Safety and Efficacy of Autologous HBadMSCs for the Treatment of a Single Pediatric Patient With Congenital Muscular Dystrophy.

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscular Dystrophy

Genetic and Rare Diseases Information Center resources: Muscular Dystrophy Congenital Muscular Dystrophy

U.S. FDA: Expanded Access (Compassionate Use)

U.S. FDA Resources

Interventions

Intervention Details:

Biological: HB-adMSCs
HB-adMSCs (Hope Biosciences autologous adipose-derived mesenchymal stem cells) is manufactured by Hope Biosciences, L.L.C., a biotechnology company headquartered in Sugar Land, Texas.

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:	Child, Adult, Older Adult
Sexes Eligible for Study:	All

Criteria

Inclusion Criteria:

Subject diagnosed with LCMNA congenital muscular dystrophy.
Subject must have banked his stem cells at Hope Biosciences LLC.

Exclusion Criteria:

Subject has any active infection requiring medications.
The subject has any known coagulation anomalies.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05154851

Locations

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United States, Texas
Hope Biosciences Stem Cell Research Foundation
Sugar Land, Texas, United States, 77478

Sponsors and Collaborators

Hope Biosciences Stem Cell Research Foundation

Hope Biosciences

Investigators

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Principal Investigator:	Djamchid Lotfi, MD	Investigator

More Information

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Responsible Party:	Hope Biosciences Stem Cell Research Foundation
ClinicalTrials.gov Identifier:	NCT05154851
Other Study ID Numbers:	HBCMD01
First Posted:	December 13, 2021 Key Record Dates
Last Update Posted:	August 1, 2022
Last Verified:	July 2022

Keywords provided by Hope Biosciences ( Hope Biosciences Stem Cell Research Foundation ):


CMD Muscular Dystrophy Congenital Stem cells L-CMD

Additional relevant MeSH terms:

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Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases	Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn

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