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New treatment option for Adenosine Deaminase Severe Combined Immune Deficiency
Official title Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
ClinicalTrials.gov ID: NCT05432310
What this study is testing
What is 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?
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 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for adenosine deaminase severe combined immune deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 and older
You may be able to join if
- All people must fulfill the following criteria to be included in the study:
- Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians...
- people ≥30 days of age,
- With a diagnosis of ADA-SCID based on: Evidence of ADA deficiency, defined as: i. Decreased ADA enzymatic activity in erythrocytes, leukocytes, skin...
- Lymphopenia (absolute lymphocyte count (ALC) \<400 cells/mL) OR absence or low number of T cells (absolute CD3+ count \< 300 cells/mL), or
You likely can't join if
- people will not be eligible for the study if any of the following criteria is fulfilled:
- Ineligible for autologous HSCT as per clinical site criteria
- Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the mobilization of peripheral blood or...
- Hematologic abnormality, defined as:
- Anemia (Hb \<8.0 g/dl).
- Neutropenia (ANC \<500/mm3). Note: ANC \<500 with absence of myelodysplastic syndrome on bone marrow aspirate and biopsy and normal marrow...
See the full eligibility criteria
- All people must fulfill the following criteria to be included in the study:
- Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the...
- people ≥30 days of age,
- With a diagnosis of ADA-SCID based on: Evidence of ADA deficiency, defined as: i. Decreased ADA enzymatic activity in erythrocytes, leukocytes, skin fibroblasts, or in cultured fetal cells to levels consistent with...
- Lymphopenia (absolute lymphocyte count (ALC) \<400 cells/mL) OR absence or low number of T cells (absolute CD3+ count \< 300 cells/mL), or
- Severely decreased T lymphocyte blastogenic responses to phytohemagglutinin (either \<10% of lower limit of normal controls for the diagnostic laboratory, or \<10% of the response of the normal control of the day, or...
- Identification of SCID by neonatal screening revealing low T Cell Receptor Excision Circles (TREC) levels.
- Ineligible for matched family allogeneic bone marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an...
- Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.
- people and their parents/legal guardians must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period and willing to return to the clinic for...
- people will not be eligible for the study if any of the following criteria is fulfilled:
- Ineligible for autologous HSCT as per clinical site criteria
- Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the mobilization of peripheral blood or the leukapheresis process, the administration of busulfan and the...
- Hematologic abnormality, defined as:
- Anemia (Hb \<8.0 g/dl).
- Neutropenia (ANC \<500/mm3). Note: ANC \<500 with absence of myelodysplastic syndrome on bone marrow aspirate and biopsy and normal marrow cytogenetics are acceptable for eligibility.
- Thrombocytopenia (platelet count \<50,000/mm3, at any age).
- Prothrombin time or international normalized ratio (INR) and partial thromboplastin time (PTT) \>2 x upper limit of normal (ULN) (people with a correctable deficiency controlled on medication will not be excluded).
- Cytogenetic abnormalities on peripheral blood or bone marrow or amniotic fluid (if available).
- Prior allogeneic HSCT with cytoreductive conditioning.
- Pulmonary abnormality, defined as:
- Resting O2 saturation by pulse oximetry \<90% on room air.
- Chest X-ray indicating active or progressive pulmonary disease. Note: Chest X ray indicating residual signs of treated pneumonitis is acceptable for eligibility.
- Cardiac abnormality, defined as:
- Abnormal ECG indicating cardiac pathology.
- Uncorrected congenital cardiac malformation with clinical symptoms.
- Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, hypotension.
- Poor cardiac function as evidenced by left ventricular ejection fraction \<40% on echocardiogram.
- Neurologic abnormality, defined as:
- Significant neurologic abnormality revealed by examination.
- Uncontrolled seizure disorder.
- Renal abnormality, defined as:
- Renal insufficiency: serum creatinine ≥1.2 mg/dl (106 µmol/L), or ≥3+ proteinuria.
- Abnormal serum sodium, potassium, calcium, magnesium or phosphate levels at \>2 x ULN.
- Hepatic/gastrointestinal abnormality, defined as:
- Serum transaminases \>5 x ULN.
- Serum bilirubin \>2 x ULN.
- Serum glucose \>1.5 x ULN.
- Oncologic disease, defined as:
- Evidence of active malignant disease other than dermatofibrosarcoma protuberans (DFSP).
- Evidence of DFSP expected to require anti-neoplastic therapy within the 5 years following the infusion of genetically corrected cells (if anti-neoplastic therapy has been completed, a subject with a history of DFSP can...
- Evidence of DFSP expected to be life limiting within the 5 years following the infusion of genetically corrected cells.
- Known sensitivity to Busulfan.
- Confirmation of an infectious disease by deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) positive at time of assessment for the following:
- HIV-1,
- Hepatitis B,
- Parvovirus B19.
- The subject is pregnant or has a major congenital anomaly.
- Is likely to require treatment during the study with drugs that are not permitted by the study protocol.
- The subject has previously received another form of gene therapy.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 month and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.