Registry Study of Revcovi Treatment in Patients With ADA-SCID
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03878069 |
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Recruitment Status :
Recruiting
First Posted : March 18, 2019
Last Update Posted : July 9, 2021
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| Condition or disease | Intervention/treatment |
|---|---|
| Adenosine Deaminase Deficiency Severe Combined Immunodeficiency | Biological: elapegademase-lvlr |
Patients with ADA-SCID who require treatment with Revcovi as Enzyme Replacement Therapy (ERT) will be enrolled during a 24-month period and each will be followed for 24 months after starting Revcovi or until undergoing hematopoietic stem cell transplant (HSCT) or hematopoietic stem cell gene therapy (HSC-GT), whichever occurs first. Patients undergoing HSCT or HSC-GT will be followed one month after last Revcovi dose and again at six months to assess adverse events (AEs) and survival. Throughout the duration of the study, patients will be assessed continually for AEs.
Patients/Parents/Caregivers will self-administer weekly intramuscular (IM) dose(s) of Revcovi and will be followed according to the Suggested Schedule of Assessments for trough dAXP and ADA activity. Treatment dosing and monitoring will be individualized per provider and patient characteristics in adherence with each study sites' standards of care.
Participants in the STP-2279-002 trial will be given the opportunity to enroll in this registry study and proceed to the Treatment Month 6 Visit per the Suggested Schedule of Assessments for Adagen-Transitioning Patients.
An interim analysis will be performed approximately two years after study initiation.
| Study Type : | Observational [Patient Registry] |
| Estimated Enrollment : | 20 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Target Follow-Up Duration: | 2 Years |
| Official Title: | Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy |
| Actual Study Start Date : | June 25, 2019 |
| Estimated Primary Completion Date : | June 2023 |
| Estimated Study Completion Date : | July 2023 |
| Group/Cohort | Intervention/treatment |
|---|---|
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ERT with ADA
Patients with diagnosis of ADA-SCID treated with ERT with Revcovi or transitioning to Revcovi from Adagen
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Biological: elapegademase-lvlr
Patients transitioning from Adagen: For patients currently receiving Adagen at ≤ 30 U/kg/wk or an unknown Adagen dose, the suggested dosage of Revcovi is 0.2 mg/kg/wk IM. For patients currently receiving Adagen at > 30 U/kg/wk the suggested equivalent Revcovi dosage (mg/kg/wk) is the Adagen dosage in U/kg/wk divided by 150. At the investigator's discretion, the total weekly dose may be divided and administered in multiple IM injections, increased by 0.033 mg/kg/wk if trough ADA activity is < 30 mmol/hr/L, dAXP is > 0.02 mmol/L, and/or the immune reconstitution is inadequate. Adagen-naïve patients: The suggested starting Revcovi dosage is 0.2 mg/kg twice weekly IM based on ideal body weight, for a minimum of 12 to 24 weeks until immune reconstitution is achieved. At the investigator's discretion, the dosage may be gradually increased to maintain trough ADA activity > 30 mmol/hr/L, dAXP < 0.02 mmol/L, and/or to maintain adequate immune reconstitution. Other Name: Revcovi |
- Deoxyadenosine nucleotides (dAXP) activity [ Time Frame: Month 24 ]Total trough erythrocyte dAXP activity
- ADA activity [ Time Frame: Month 24 ]Trough plasma ADA activity
- Immune status (SSA/PI) [ Time Frame: Month 24 ]
Absolute lymphocyte count and subset B, T, and NK analysis.
Immunoglobulin (Ig) concentrations (IgG, IgA, and IgM).
Measurement of immune response at Investigator discretion.
- Clinical status [ Time Frame: Month 24 ]Infections (clinically or microbiologically documented) Incidence and duration of hospitalizations Growth for patients < 18 years old Overall survival through the end of study
- Safety assessed by determining adverse events (AEs), serious adverse events (SAEs) [ Time Frame: Month 24 ]Assessed by determining adverse events (AEs), serious adverse events (SAEs), clinical signs and symptoms from physical examinations, and laboratory examinations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | up to 65 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- Patients with diagnosis of ADA-SCID who require ERT with ADA as judged by the treating physicians, based on the medical history, biochemical test or genotyping.
- Understanding and willing to comply with the Registry recommendations via signed and dated written informed consent/assent.
- ADA-SCID patient requiring Revcovi as an ERT.
Exclusion Criteria:
- Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03878069
| Contact: Chiesi Clinical Trial Info | +39.0521 2791 | clinicaltrials_info@chiesi.com |
| United States, Arizona | |
| Phoenix Children's Hospital | Not yet recruiting |
| Phoenix, Arizona, United States, 85016 | |
| Contact: Holly K Miller, MD 602-933-0920 hmiller2@phoenixchildrens.com | |
| Principal Investigator: Holly K Miller, MD | |
| United States, California | |
| University of California Los Angeles | Recruiting |
| Los Angeles, California, United States, 90095-1752 | |
| Contact: Alexis Stephens, MD 310-210-8771 AVStephens@mednet.ucla.edu | |
| Principal Investigator: Manish Butte, MD, PhD | |
| UCSF - University of California | Not yet recruiting |
| San Francisco, California, United States, 94158 | |
| Contact: Morna Dorsey, MD 415-476-3086 Morna.Dorsey@ucsf.edu | |
| Principal Investigator: Morna Dorsey, MD | |
| United States, Florida | |
| University of South Florida Allergy Immunology Clinic | Recruiting |
| Saint Petersburg, Florida, United States, 33701 | |
| Contact: Jolan Walter, MD, PhD 727-553-1258 jolanwalter@usf.edu | |
| Contact: Maryssa Ellison mellison@usf.edu | |
| Principal Investigator: Jolan Walter | |
| United States, Louisiana | |
| Childrens Hospital of New Orleans | Recruiting |
| New Orleans, Louisiana, United States, 70118 | |
| Contact: Luke Wall, MD 504-896-9589 lwall@lsuhsc.edu | |
| Principal Investigator: Luke Wall, MD | |
| United States, Missouri | |
| St. Louis Children's Hospital - Washington University School of Medicine | Recruiting |
| Saint Louis, Missouri, United States, 63110 | |
| Contact: Jeffrey J. Bednarski, MD 314-286-2825 bednarski_j@wustl.edu | |
| Principal Investigator: Jeffrey J. Bednarski, MD | |
| United States, New York | |
| UBMD Pediatrics Outpatient Center | Recruiting |
| Buffalo, New York, United States, 14203 | |
| Contact: Heather Lehman, MD 716-323-0130 hkm@buffalo.edu | |
| Contact: Helena McClenahan hmcclenahan@upa.chob.edu | |
| Principal Investigator: Heather Lehman, MD | |
| United States, Pennsylvania | |
| Penn State Children's Hospital | Recruiting |
| Hershey, Pennsylvania, United States, 17033 | |
| Contact: Tracy B Fausnight, MD 717-531-1846 tfausnight@pennstatehealth.psu.edu | |
| Principal Investigator: Tracy B Fausnight, MD | |
| UPMC Children's Hospital of Pittsburgh | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15224 | |
| Contact: Hey Chong, MD 412-692-7785 hey.chong@chp.edu | |
| Principal Investigator: Hey Chong, MD | |
| United States, Tennessee | |
| Le Bonheur Children's Hospital | Terminated |
| Memphis, Tennessee, United States, 38105 | |
| Responsible Party: | Chiesi Farmaceutici S.p.A. |
| ClinicalTrials.gov Identifier: | NCT03878069 |
| Other Study ID Numbers: |
LBI-2279-004 |
| First Posted: | March 18, 2019 Key Record Dates |
| Last Update Posted: | July 9, 2021 |
| Last Verified: | July 2021 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
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Severe Combined Immunodeficiency Immunologic Deficiency Syndromes Immune System Diseases Primary Immunodeficiency Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |
DNA Repair-Deficiency Disorders Metabolic Diseases Elapegademase Adenosine Deaminase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |