Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy

Registration

Registration

NCT02882477

Study type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Phase

PHASE2; PHASE3

Masking

NONE

Status

UNKNOWN

Enrollment

20

Start

2016-12

Primary completion

2018-05

Lead sponsor

Hadassah Medical Organization

Conditions

Diabetes Mellitus; Iron Metabolism Disorders; Gastroduodenal Ulcer; Optic Atrophy; Sensorineural Hearing Loss; Platelet Dysfunction

Interventions

DRUG:Deferiprone; DRUG:Acetylcysteine; DRUG:Sitagliptin and Metformin

Results posted

FALSE

Individual participant data sharing

NO

Registered primary outcomes

• Insulin levels in blood in response to Glucose Challenge - Oral Glucose tolerance test (OGTT ) and Intra venous glucose tolerance test (IVGTT) [5 months]
• Platelet aggregation to Adenosine diphosphate (ADP) and Collagen - blood test for Platelet function test [5 months]
• Nerve conduction velocity in VEP [5 months]
• HBA1C level [5 months]
• daily glucose level measurement in the blood using Glucometer two to 5 times daily [5 months]
• C-Peptide levels in blood in response to Glucose Challenge - Oral Glucose tolerance test ( OGTT ) and Intra venous glucose tolerance test IVGTT [5 months]