Skip to main content
MedPathMedPath Home
Clinical Trials/EUCTR2017-003534-89-FR
EUCTR2017-003534-89-FR
Active, not recruiting
Phase 1

A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR PHXC Solution for Injection (subcutaneous use) - 2-part study of DCR-PHXC in NHV and PH patients

Dicerna Pharmaceuticals Inc0 sites41 target enrollmentOctober 4, 2018
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
ConditionsPrimary HyperoxaluriaMedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Overview

Phase
Phase 1
Intervention
Not specified
Conditions
Primary Hyperoxaluria
Sponsor
Dicerna Pharmaceuticals Inc
Enrollment
41
Status
Active, not recruiting
Last Updated
6 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
October 4, 2018
End Date
TBD
Last Updated
6 years ago
Study Type
Interventional clinical trial of medicinal product

Investigators

Eligibility Criteria

Inclusion Criteria

  • •PH patients must meet all of the following criteria to be eligible for participation in this study.
  • •1\. Patient and/or patient's parent or guardian if the patient is a minor (defined as patient \< 18 years of age, or younger than the age of majority, according to local regulations),
  • •a. Understands the full nature and purpose of the study, including possible risks and side effects.
  • •b. Is willing and able to comply with all study procedures including collection of 24\-hr urine samples.
  • •c. Provides informed consent. 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.
  • •2\. Male or female, at least 6 years of age at the time of obtaining informed consent.
  • •3\. Patient must have a minimum body weight of 25 Kg.
  • •4\. Documented diagnosis of PH1 or PH2, confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
  • •5\. 24\-hr urine oxalate excretion \=0\.7 mmol for patients 18 years and older, or \=0\.7 mmol per 1\.73 m2 body surface area (BSA) for patients less than 18 years of age, on at least one of the two assessments conducted in the screening period, with less than 30% variation between both oxalate measurements.
  • •6\. eGFR \=30 mL/min normalized to 1\.73 m2 BSA calculated using the Modification of Diet in Renal Disease (MDRD) formula in adults (age \=18 years), or the formula by Schwartz in patients 6 to \< 18 years old (Levey et al., 1999; Schwartz et al., 2009; National Kidney Foundation, 2002\).

Exclusion Criteria

  • •PH patients meeting any of the following criteria will be excluded from this study:
  • •1\. Prior renal and/or hepatic transplantation.
  • •2\. Currently receiving dialysis.
  • •3\. Documented evidence of clinical manifestations of systemic oxalosis.
  • •4\. Participation in any clinical study where they received an investigational medical product within 4 months before enrollment. For IMPs with the potential to reduce Uox and/or plasma oxalate, these concentrations must have returned to historical baseline levels. a. If patient participated in an earlier cohort in this study (DCR\-PHXC\-101\), a minimum of 8 weeks must have elapsed prior to re\-enrollment and urinary oxalate excretion must have returned to \=80% of baseline.
  • •5\. Presence of any medical condition or co\-morbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to: a. severe intercurrent illness
  • •b. routine vaccination within 30 days prior to dosing and through EOS visit
  • •c. known causes of active liver disease/ injury or transaminase elevation (e.g., alcoholic liver disease, Nonalcoholic fatty liver disease/ steatohepatitis (NAFLD/NASH)
  • •d. physician concerns about excess alcohol consumption
  • •e. routine or chronic use of more than 3 grams of acetaminophen daily.

Outcomes

Primary Outcomes

Not specified

Similar Trials

Active, not recruiting
Phase 1
2-part study of DCR-PHXC in healthy volunteers and in primary hyperoxaluria patients
EUCTR2017-003534-89-NLDicerna Pharmaceuticals Inc43
Active, not recruiting
Phase 1
2-part study of DCR-PHXC in healthy volunteers and in primary hyperoxaluria patientsPrimary HyperoxaluriaMedDRA version: 20.1Level: PTClassification code 10020703Term: HyperoxaluriaSystem Organ Class: 10038359 - Renal and urinary disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
EUCTR2017-003534-89-GBDicerna Pharmaceuticals Inc43
Active, not recruiting
Phase 1
2-part study of DCR-PHXC in healthy volunteers and in primary hyperoxaluria patients
EUCTR2017-003534-89-DEDicerna Pharmaceuticals Inc41
Not yet recruiting
Not Applicable
A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR PHXC Solution for Injection (subcutaneous use)
NL-OMON46435Dicerna Pharmaceutical Inc5
Active, not recruiting
Phase 1
Study Investigating the Dose of NAITgam (a Human Anti-Human Platelet Antigen (HPA)-1a Immune Globulin) Needed to Eliminate Platelets Transfused to Healthy Male Volunteers
EUCTR2019-003459-12-DERallybio, IPA, LLC28