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Gene transfer    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
65Primary immunodeficiency14

65. Primary immunodeficiency    [ 413 clinical trials,   581 drugs,   (DrugBank: 97 drugs),   68 drug target genes,   202 drug target pathways]
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
14 / 413 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03601286
(ClinicalTrials.gov)		December 21, 201822/2/2018Lentiviral Gene Therapy for X-linked Severe Combined ImmunodeficiencyPhase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted BusulfanSevere Combined Immunodeficiency, X-LinkedDrug: Lentiviral vector transduced CD34+ cellsGreat Ormond Street Hospital for Children NHS Foundation TrustNULLRecruiting8 Weeks5 YearsMale5Phase 1United Kingdom
2EUCTR2018-000673-68-GB
(EUCTR)		09/10/201827/07/2018 A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined ImmunodeficiencyPhase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening.
MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]		Great Ormond Street Hospital for Children NHS TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes		5Phase 1United Kingdom
3NCT03538899
(ClinicalTrials.gov)		May 31, 20183/5/2018Autologous Gene Therapy for Artemis-Deficient SCIDA Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic CellsSevere Combined ImmunodeficiencyDrug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: BusulfanUniversity of California, San FranciscoNULLRecruiting2 MonthsN/AAll15Phase 1;Phase 2United States
4NCT03311503
(ClinicalTrials.gov)		January 19, 201812/10/2017Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningPhase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningSevere Combined Immunodeficiency, X Linked;Gene TherapyBiological: autologous CD34+ cell transduced with G2SCID vectorDavid WilliamsNULLRecruitingN/A5 YearsMale10Phase 1;Phase 2United States;United Kingdom
5NCT03217617
(ClinicalTrials.gov)		July 15, 20173/7/2017Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)SCID, X LinkedBiological: TYF-IL-2Rg gene-modified autologous stem cellsShenzhen Geno-Immune Medical InstituteNULLRecruiting1 Month10 YearsMale10Phase 1;Phase 2China
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT01512888
(ClinicalTrials.gov)		August 17, 201613/1/2012Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed InfantsA Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic CellsSevere Combined Immunodeficiency Disease, X-linkedGenetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacsSt. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM)RecruitingN/A24 MonthsMale28Phase 1;Phase 2United States
7NCT01306019
(ClinicalTrials.gov)		September 25, 201226/2/2011Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyX-Linked Severe Combined Immune Deficiency (XSCID)Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1a-hyc-OPT vectorNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting2 Years40 YearsMale30Phase 1;Phase 2United States
8NCT03315078
(ClinicalTrials.gov)		April 201216/10/2017Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyLentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyX-Linked Combined Immunodeficiency DiseasesBiological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting2 Years40 YearsAll13Phase 1;Phase 2United States
9NCT01410825
(ClinicalTrials.gov)		July 20114/8/2011Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromePilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeBiological: Retrovirus-mediated gene transferDavid WilliamsNULLActive, not recruiting3 Months35 YearsMale5Phase 1;Phase 2United States
10NCT01129544
(ClinicalTrials.gov)		April 201021/5/2010Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral VectorMulti-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene TransferSevere Combined ImmunodeficiencyBiological: Gene transferDavid WilliamsBoston Children's Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los AngelesActive, not recruitingN/AN/AMale8Phase 1;Phase 2United States
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
11NCT00794508
(ClinicalTrials.gov)		November 200819/11/2008MND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDMND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With BusulfanSevere Combined ImmunodeficiencyBiological: ADA gene transferDonald B. Kohn, M.D.FDA Office of Orphan Products Development;National Institutes of Health (NIH)Completed1 Month18 YearsAll10Phase 2United States
12NCT00028236
(ClinicalTrials.gov)		December 10, 200117/12/2001Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)Ex Vivo Retroviral Gene Transfer For Treatment of X-Linked Severe Combined Immunodeficiency (XSCID)Severe Combined ImmunodeficiencyDrug: Gene-Transduced Autologous CD34+ Stem CellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted18 Months20 YearsAll3Phase 1United States
13NCT00018018
(ClinicalTrials.gov)		June 20, 200127/6/2001Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) DeficiencyTreatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA GeneSevere Combined Immunodeficiency SyndromeDrug: CD34+ cells transduced with ADA retrovirNational Human Genome Research Institute (NHGRI)NULLCompleted1 MonthN/AAll8Phase 1United States
14NCT00001255
(ClinicalTrials.gov)		September 19903/11/1999Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History StudyTreatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History StudySevere Combined ImmunodeficiencyDrug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced LymphocytesNational Human Genome Research Institute (NHGRI)NULLCompletedN/AN/ABoth10N/AUnited States

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