Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Ended
Participants planned
64
Countries
1
Sites
1
Healthy - R7999 being developed for the treatment of diseases associated with iron overload.
To evaluate the safety and tolerability of single ascending intravenous (IV) and subcutaneous (SC) doses of REGN7999 in healthy adult participants.
Key facts
- Sponsor
- Regeneron Pharmaceuticals Inc.
- Participant type
- Healthy volunteers
- Age range
- 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 24 Oct 2022 → 24 Aug 2023
- Decision date (initial)
- 2022-10-03
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- Regeneron Pharmaceuticals Inc
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Safety, Pharmacodynamic
To evaluate the safety and tolerability of single ascending intravenous (IV) and subcutaneous (SC) doses of REGN7999 in healthy adult participants.
Secondary objectives 2
- To characterize the drug concentration profile of single doses of Intravenous or Subcutaneous REGN7999.
- To assess the immunogenicity of single ascending SC or IV doses of REGN7999.
Conditions and MedDRA coding
Healthy - R7999 being developed for the treatment of diseases associated with iron overload.
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | PT | 10065973 | Iron overload | 100000004861 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Method of Treatment Assignment Up to 64 participants will be randomized in a 3:1 ratio to receive either REGN7999 or placebo,
according to a central randomization scheme provided by an interactive voice response system
(IVRS)/interactive web response system (IWRS) to the designated study pharmacist (or qualified
designee).
|
Randomised Controlled | Double | [{"id":4300,"code":1,"name":"Subject"},{"id":4298,"code":2,"name":"Investigator"},{"id":4299,"code":3,"name":"Monitor"},{"id":4301,"code":4,"name":"Analyst"}] | Ascending IV and subcutaneous Cohorts: 5 IV dose administration (doses of 10, 30, 100, 300, and 900 mg) and 3 SC cohorts (doses of 100, 300, and 900 mg) |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Is a male or female from 18 to 60 years of age (inclusive) at the screening visit.
- Is judged by the investigator to be in good health based on medical history, physical examination, vital sign measurements, and ECGs performed at screening and/or prior to administration of initial dose of study drug.
- Hemoglobin, serum iron, transferrin, serum ferritin, and transferrin saturation, equal to or above the lower limit of the reference range for the participant’s age and sex at the local labs, at screening, repeatable once during screening period.
- WBC count, platelet count, RBC count, hematocrit, and RBC hemoglobin not clinically significantly outside of the reference range in the judgment of the investigator at screening and baseline visits.
- Provide informed consent signed by study participant.
Exclusion criteria 8
- Pregnant or breastfeeding women.
- Consistent with Clinical Trial Facilitation Group (CTFG) guidance, women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception , during the study through the EOS visit. Highly effective contraceptive measures include: a. stable use of combined (estrogen and progestogen containing) hormonal contraception (oral, intravaginal, transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation initiated 2 or more menstrual cycles prior to screening; b. intra-uterine device (IUD); intra-uterine hormone-releasing system; c. bilateral tubal ligation or tubal occlusion; d. vasectomized partner (provided that the male vasectomized partner is the sole sexual partner of the WOCBP study participant and that the vasectomized partner has obtained medical assessment of surgical success for the procedure); and/or e. sexual abstinence as described in the protocol.
- Premenopausal women whose method(s) of birth control is/are associated with ongoing menstruation (eg, combined hormonal contraceptive regimens associated with withdrawal bleeding, non-hormone-releasing IUD, bilateral tubal ligation, bilateral salpingectomy, vasectomized partner, sexual abstinence). Female participants must not be menstruating during the trial, due to being postmenopausal or due to permanent sterilization via hysterectomy, and/or bilateral oophorectomy, or amenorrheic due to use of hormone-releasing IUD, implantable device, or intake of continuous hormonal contraception.
- History of clinically significant cardiovascular (including congestive heart failure and angina), respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, infectious, autoimmune, oncologic, psychiatric or neurological disease, as assessed by the investigator, that may confound the results of the study or poses an additional risk to the participant by study participation.
- History of chronic anemia, at any time in the past.
- Whole blood donation within the previous 56 days or plasma donation within the previous 7 days prior to screening. Planning on whole blood or plasma donation at any time point during the study.
- Participated in any clinical research study evaluating another investigational drug including biologics or therapy, including specific immunotherapy, within 90 days or at least 5 half-lives (whichever is longer) of an investigational biologic drug, or at least 4 weeks for other investigational drug, prior to the screening visit.
- Has received a COVID-19 vaccination within 1 week of planned start of study medication or for which the planned COVID-19 vaccinations would not be completed 1 week prior to start of study drug.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Incidence and severity of treatment emergent adverse events (TEAEs) in participants treated with REGN7999 or placebo through the end of study visit (week 20 for IV cohorts 1 to 4 and for SC cohorts 1 and 2, and week 26 for IV cohort 5 and SC cohort 3)
Secondary endpoints 2
- Concentrations of REGN7999 in serum through the end of study visit (week 20 for IV cohorts 1 to 4 and for SC cohorts 1 and 2 and week 26 for IV cohort 5 and SC cohort 3)
- Incidences of anti-drug antibodies (ADA) to REGN7999 over time through the end of study visit (week 20 for IV Cohorts 1 to 4 and SC Cohort 1 and 2 and week 26 for IV Cohort 5 and SC cohort 3).
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
PRD9797026 · Product
- Active substance
- REGN7999
- Pharmaceutical form
- POWDER FOR SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 900 mg milligram(s)
- Max total dose
- 900 mg milligram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- REGENERON PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD9797025 · Product
- Active substance
- REGN7999
- Pharmaceutical form
- POWDER FOR SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 900 mg milligram(s)
- Max total dose
- 900 mg milligram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- REGENERON PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Regeneron Pharmaceuticals Inc.
- Sponsor organisation
- Regeneron Pharmaceuticals Inc.
- Address
- 777 Old Saw Mill River Road
- City
- Tarrytown
- Postcode
- 10591-6717
- Country
- United States
Scientific contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Public contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Third parties 4
| Organisation | City, country | Duties |
|---|---|---|
| Fisher Clinical Services Inc. ORG-100014726
|
Allentown, United States | Other |
| Transperfect Translations International Inc. ORG-100043494
|
New York, United States | Other |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | Laboratory analysis |
| Yprime LLC ORG-100042888
|
Malvern, United States | Interactive response technologies (IRT) |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ended | 64 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2022-10-24 | 2023-08-24 | 2022-10-24 | 2023-02-10 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Summary of results R7999-HV-2154 SUM-42066
|
2024-08-21T19:18:18 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay person summary of results R7999-HV-2154 | 2024-08-21T19:22:52 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | R7999-HV-2154_TRS-EN-US | 1 |
| Summary of results (for publication) | 2022-500398-15-00_Results_2024-08-21 | 1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2022-07-28 | Belgium | Acceptable 2022-09-29
|
2022-10-03 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2022-10-26 | Belgium | Acceptable 2022-09-29
|
2022-10-26 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2022-12-13 | Belgium | Acceptable 2022-09-29
|
2022-12-13 |