OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

  • STATUS
    Recruiting
  • End date
    Jan 29, 2032
  • participants needed
    6
  • sponsor
    Orchard Therapeutics
Send
Updated on 19 February 2024
See if I qualify

Summary

OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients.

Details
Condition Metachromatic leukodystrophy, Lysosomal Storage Disease
Age 100years or below
Treatment OTL-200
Clinical Study IdentifierNCT04283227
SponsorOrchard Therapeutics
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

All the following criteria need to be met
Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two disease-causing ARSA alleles
R or R/R genotype or a genotype recognized as associated with the LJ variant of MLD
a) If symptomatic: age at disease onset between 7 and <17 years of age (i.e. before their 17th birthday). OR
b) If pre-symptomatic: participant must be <17 years of age at treatment (i.e. before their 17th birthday) AND must have a sibling with a diagnosis of late-juvenile MLD variant based on age at disease onset (7 and <17 years of age i.e. before sibling's 17th birthday), with biochemical and molecular diagnosis
Normal cognitive function as defined by an IQ85 on age appropriate cognitive scales
a) If the participant is <7 years (i.e. before their 7th birthday): normal motor milestones achievement, normal gross motor function according to chronological age and normal neurological examination (if applicable based on the age of the subject, GMFC-MLD = 0) OR b) If participant is 7 years: normal gross motor function or mild gross motor function impairment, defined by a GMFC-MLD 0 or 1 (i.e. patient is able to walk independently)
If applicable, participant willing and capable of compliance with contraceptive use requirements
Participant (or if applicable, parent/legal guardian) providing signed informed consent

Exclusion Criteria

Documented HIV infection (positive HIV RNA and/or anti-p24 antibodies)
Malignant neoplasia (except localised skin cancer) or a documented history of hereditary cancer syndrome
Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML) or other serious haematological disorders
Patients currently enrolled in other interventional trials
Has previously undergone allogeneic HSPC gene therapy (HSPC-GT) and has evidence of residual cells of donor origin
Previous gene therapy
Has symptomatic herpes zoster, not responsive to specific treatment
Evidence of active tuberculosis (TB) based upon medical examination, chest imaging and TB testing
Acute or chronic stable Hepatitis B (HBV) as evidenced by positive Hepatitis B surface antigen (HBsAg) test result at screening or within 3 months prior to onset of conditioning and/or positive HBV DNA
Presence of positive Hepatitis C RNA test result at screening
End-organ dysfunction, severe active infection not responsive to treatment, or other severe disease or clinical condition which, in the judgment of the investigator, would make the participant inappropriate for entry into this study
In addition to the potential infections tested per protocol, the PI should consider testing for other transmissible infectious agents listed in the European Union (EU) Cell and Tissue Directive as clinically appropriate and results must be discussed with the Orchard medical monitor prior to stem cell harvest
Participants with alanine transferase (ALT) >2x upper limit of normal (ULN) or total bilirubin >1.5xULN may be included only after discussed and agreed with the Orchard medical monitor and considered in the context of the criterion for excluding participants with other severe disease. Isolated elevation of total bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35% of total
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

Study AnnotationsStudy Notes

Notes added here are public and can be viewed by anyone. Notes added here are only available to you and those who you share with.

user name
Edit Delete

Added by • 

 • 

Private

Edit Delete

Reply by • • Private
Edit Delete

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.