Gene therapy study with autologous hemapoietic stem cells for patients affected by MPSIH

Phase 1

• Conditions: Mucopolysaccharidosis type I Hurler; MedDRA version: 20.1Level: PTClassification code 10056886Term: Mucopolysaccharidosis ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2017-002430-23-IT
• Lead Sponsor: OSPEDALE SAN RAFFAELE

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-03-09
• Last Update Posted: 7 September 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

1.Written informed consent by parent/legal guardian
2. Sex: Males or Females
3. Age: = 28 days and = 11 years old
4. Biochemically and molecularly proven MPS IH
5.Lansky Index > 80 %
6.Indication to HSCT
7.Lack of a non-heterozygous (for mutated IDUA) HLA-matched sibling donor or a =7/8 (4 digits high-resolution typing) HLA-matched cord blood donor with a cellularity =5 x 107 Total Nucleated Cells (TNC)/Kg after 1-month search
8.Adequate cardiac, renal, hepatic and pulmonary functions
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
2.Severe, active viral, bacterial, or fungal infection at eligibility evaluation
3.Patients affected by malignant neoplasia or family history of familial cancer syndromes
4.Cytogenetic alterations associated with high risk of developing hematological malignancies
5.History of uncontrolled seizures
6.Patients with end-organ damage or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study
7.Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA and/or Treponema Pallidum or Mycoplasma active infection
8.Patients with DQ/IQ <70
9.Previous allogeneic HSCT or gene therapy with a different product
10. Controindications to PeIMP (G-CSF, Plerixafor, Busulfan, Fludarabine, Rituximab)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified