A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 and Severe Renal Impairment, With or Without Dialysis

Who is this study for? Patients with primary hyperoxaluria type 1 or 2 and severe renal impairment
What treatments are being studied? DCR-PHXC
Status: Recruiting
Location: See all (18) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Four age groups of participants will be enrolled:

‣ adults and adolescents (aged ≥ 12 years)

⁃ children 6 to 11 years of age

⁃ infants and newborns from birth to \< 2 years of age

• . Documented diagnosis of PH1, confirmed by genotyping

• Estimated GFR at Screening \<30mL/min normalized to 1.73m\^2 BSA

• Mean of 2 Plasma Oxalate \>20μmol/L during screening

• For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.

• Male or Female

‣ Male participants:

• A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.

⁃ Female participants:

• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:

‣ Not a woman of childbearing potential (WOCBP).

∙ OR

∙ A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study intervention and agrees to refrain from harvesting/freezing eggs during this period.

⁃ Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

• Participant (and/or participant's parent or legal guardian if participant is a minor \[defined as patient \<18 years of age, or younger than the age of majority according to local regulations\]) is capable of giving signed informed consent, which includes compliance with the requirement and restrictions listed in the informed consent form (ICF) and in the protocol.

‣ Adolescents (12 to \< 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.

⁃ For children younger than 12 years of age, assent will be based on local regulations

• Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)

Locations
United States
California
Clinical Trial Site
RECRUITING
San Francisco
Massachusetts
Clinical Trial Site
RECRUITING
Boston
Minnesota
Clinical Trial Site
RECRUITING
Rochester
New York
Clinical Trial Site
RECRUITING
New York
Other Locations
France
Clinical Trial Site
WITHDRAWN
Bron
Clinical Trial Site
WITHDRAWN
Paris
Germany
Clinical Trial Site
RECRUITING
Bonn
Clinical Trial Site
RECRUITING
Heidelberg
Italy
Clinical Trial Site
RECRUITING
Roma
Lebanon
Clinical Trial Site
RECRUITING
Beirut
Morocco
Clinical Trial Site
NOT_YET_RECRUITING
Casablanca
Romania
Clinical Trial Site
WITHDRAWN
Oradea
Clinical Trial Site
WITHDRAWN
Oradea
Spain
Clinical Trial Site
RECRUITING
Barcelona
Clinical Trial Site
WITHDRAWN
Santa Cruz De Tenerife
United Arab Emirates
Clinical Trial Site
NOT_YET_RECRUITING
Dubai
United Kingdom
Clinical Trial Site
RECRUITING
London
Clinical Trial Site
RECRUITING
London
Contact Information
Primary
Novo Nordisk
clinicaltrials@novonordisk.com
(+1) 866-867-7178
Time Frame
Start Date: 2021-04-15
Estimated Completion Date: 2032-01-30
Participants
Target number of participants: 28
Treatments
Experimental: Open-Label DCR-PHXC
Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.
Sponsors
Leads: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

This content was sourced from clinicaltrials.gov