ADA-SCID

Clinical trial pipeline · Data from ClinicalTrials.gov

See which ADA-SCID trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

RecruitingPhase 1/2

NCT05432310

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blo…

United States20 participantsStarted 2023-01-04

Treatment: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

RecruitingNot Applicable

NCT03645460

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactiva…

China10 participantsStarted 2024-06-30

Treatment: Direct intravenous injection of ivlv-ADA lentiviral vector

All recent trials (31 shown)

By InvitationNot Applicable

NCT04049084

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo g…

United States, United Kingdom70 participantsStarted 2019-09-26

Treatment: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

By InvitationNot Applicable

NCT03655223

Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…

United States30,000 participantsStarted 2018-10-15

Treatment:

ADA-SCID

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

All recent trials (31 shown)

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Early Check: Expanded Screening in Newborns

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

All recent trials (31 shown)

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Early Check: Expanded Screening in Newborns

Registry Study of Revcovi Treatment in Patients With ADA-SCID

Molecular and Clinical Studies of Primary Immunodeficiency Diseases

Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

Retroviral Insertion Site Methodology Study

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral V…

Investigation of ADA Enzyme Deficiency

Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E

Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

Patients Treated for SCID (1968-Present)